Orphan Drug Production: Production of drugs or biologicals which are unlikely to be manufactured by private industry unless special incentives are provided by others.Rare Diseases: A large group of diseases which are characterized by a low prevalence in the population. They frequently are associated with problems in diagnosis and treatment.Drug Approval: Process that is gone through in order for a drug to receive approval by a government regulatory agency. This includes any required pre-clinical or clinical testing, review, submission, and evaluation of the applications and test results, and post-marketing surveillance of the drug.Economics, Pharmaceutical: Economic aspects of the fields of pharmacy and pharmacology as they apply to the development and study of medical economics in rational drug therapy and the impact of pharmaceuticals on the cost of medical care. Pharmaceutical economics also includes the economic considerations of the pharmaceutical care delivery system and in drug prescribing, particularly of cost-benefit values. (From J Res Pharm Econ 1989;1(1); PharmacoEcon 1992;1(1))Legislation, Drug: Laws concerned with manufacturing, dispensing, and marketing of drugs.United States Food and Drug Administration: An agency of the PUBLIC HEALTH SERVICE concerned with the overall planning, promoting, and administering of programs pertaining to maintaining standards of quality of foods, drugs, therapeutic devices, etc.Tropical Medicine: The branch of medicine concerned with diseases, mainly of parasitic origin, common in tropical and subtropical regions.Ethics, Pharmacy: The principles of proper professional conduct concerning the rights and duties of the pharmacist, relations with patients and fellow practitioners, as well as actions of the pharmacist in health care and interpersonal relations with patient families. (From Stedman, 25th ed)Drug Industry: That segment of commercial enterprise devoted to the design, development, and manufacture of chemical products for use in the diagnosis and treatment of disease, disability, or other dysfunction, or to improve function.Off-Label Use: The practice of prescribing or using a drug outside the scope of the drug's official approved label as designated by a regulatory agency concerning the treatment of a particular disease or condition.Patents as Topic: Exclusive legal rights or privileges applied to inventions, plants, etc.Patient Advocacy: Promotion and protection of the rights of patients, frequently through a legal process.Consumer Advocacy: The promotion and support of consumers' rights and interests.European Union: The collective designation of three organizations with common membership: the European Economic Community (Common Market), the European Coal and Steel Community, and the European Atomic Energy Community (Euratom). It was known as the European Community until 1994. It is primarily an economic union with the principal objectives of free movement of goods, capital, and labor. Professional services, social, medical and paramedical, are subsumed under labor. The constituent countries are Austria, Belgium, Denmark, Finland, France, Germany, Greece, Ireland, Italy, Luxembourg, Netherlands, Portugal, Spain, Sweden, and the United Kingdom. (The World Almanac and Book of Facts 1997, p842)Webcasts as Topic: Transmission of live or pre-recorded audio or video content via connection or download from the INTERNET.United StatesInvestments: Use for articles on the investing of funds for income or profit.Dental Staff: Personnel who provide dental service to patients in an organized facility, institution or agency.Capital Financing: Institutional funding for facilities and for equipment which becomes a part of the assets of the institution.Newspapers: Publications printed and distributed daily, weekly, or at some other regular and usually short interval, containing news, articles of opinion (as editorials and letters), features, advertising, and announcements of current interest. (Webster's 3d ed)Journalism, Medical: The collection, writing, and editing of current interest material on topics related to biomedicine for presentation through the mass media, including newspapers, magazines, radio, or television, usually for a public audience such as health care consumers.Cerium: An element of the rare earth family of metals. It has the atomic symbol Ce, atomic number 58, and atomic weight 140.12. Cerium is a malleable metal used in industrial applications.Mass Media: Instruments or technological means of communication that reach large numbers of people with a common message: press, radio, television, etc.TexasLaboratories, Dental: Facilities for the performance of services related to dental treatment but not done directly in the patient's mouth.Dental Technicians: Individuals responsible for fabrication of dental appliances.Stem Cell Research: Experimentation on STEM CELLS and on the use of stem cells.Embryo Research: Experimentation on, or using the organs or tissues from, a human or other mammalian conceptus during the prenatal stage of development that is characterized by rapid morphological changes and the differentiation of basic structures. In humans, this includes the period from the time of fertilization to the end of the eighth week after fertilization.Stem Cells: Relatively undifferentiated cells that retain the ability to divide and proliferate throughout postnatal life to provide progenitor cells that can differentiate into specialized cells.Stem Cell Transplantation: The transfer of STEM CELLS from one individual to another within the same species (TRANSPLANTATION, HOMOLOGOUS) or between species (XENOTRANSPLANTATION), or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). The source and location of the stem cells determines their potency or pluripotency to differentiate into various cell types.Cell- and Tissue-Based Therapy: Therapies that involve the TRANSPLANTATION of CELLS or TISSUES developed for the purpose of restoring the function of diseased or dysfunctional cells or tissues.Embryonic Stem Cells: Cells derived from the BLASTOCYST INNER CELL MASS which forms before implantation in the uterine wall. They retain the ability to divide, proliferate and provide progenitor cells that can differentiate into specialized cells.Adult Stem Cells: Cells with high proliferative and self renewal capacities derived from adults.Morale: The prevailing temper or spirit of an individual or group in relation to the tasks or functions which are expected.Waste Management: Disposal, processing, controlling, recycling, and reusing the solid, liquid, and gaseous wastes of plants, animals, humans, and other organisms. It includes control within a closed ecological system to maintain a habitable environment.United States Occupational Safety and Health Administration: An office in the Department of Labor responsible for developing and establishing occupational safety and health standards.Economic Competition: The effort of two or more parties to secure the business of a third party by offering, usually under fair or equitable rules of business practice, the most favorable terms.National Institute for Occupational Safety and Health (U.S.): An institute of the CENTERS FOR DISEASE CONTROL AND PREVENTION which is responsible for assuring safe and healthful working conditions and for developing standards of safety and health. Research activities are carried out pertinent to these goals.Workplace: Place or physical location of work or employment.Occupational Health: The promotion and maintenance of physical and mental health in the work environment.Biological Therapy: Treatment of diseases with biological materials or biological response modifiers, such as the use of GENES; CELLS; TISSUES; organs; SERUM; VACCINES; and humoral agents.Encyclopedias as Topic: Works containing information articles on subjects in every field of knowledge, usually arranged in alphabetical order, or a similar work limited to a special field or subject. (From The ALA Glossary of Library and Information Science, 1983)Biopharmaceutics: The study of the physical and chemical properties of a drug and its dosage form as related to the onset, duration, and intensity of its action.Biological Products: Complex pharmaceutical substances, preparations, or matter derived from organisms usually obtained by biological methods or assay.Northwestern United States: The geographic area of the northwestern region of the United States. The states usually included in this region are Idaho, Montana, Oregon, Washington, and Wyoming.Northwest Territories: A federally administered division of Canada. Its capital is Yellowknife. The former northern and eastern-most parts of the Territory comprise the new territory of Nunavut, effective April 1, 1999.MarylandViola: A plant genus of the family VIOLACEAE. Some species in this genus are called bouncing bet which is a common name more often used with SAPONARIA OFFICINALIS. Members contain macrocyclic peptides.Alicyclobacillus: A genus of GRAM-POSITIVE ENDOSPORE-FORMING RODS, in the family Alicyclobacillaceae, containing a unique lipid in their membranes.SwitzerlandKinesin: A microtubule-associated mechanical adenosine triphosphatase, that uses the energy of ATP hydrolysis to move organelles along microtubules toward the plus end of the microtubule. The protein is found in squid axoplasm, optic lobes, and in bovine brain. Bovine kinesin is a heterotetramer composed of two heavy (120 kDa) and two light (62 kDa) chains. EC 3.6.1.-.Nanotechnology: The development and use of techniques to study physical phenomena and construct structures in the nanoscale size range or smaller.

Is orphan drug status beneficial to tropical disease control? Comparison of the American and future European orphan drug acts. (1/70)

OBJECTIVES To quantify past outcomes of tropical pharmacology research and development (R & D) and to assess past benefits of the American orphan drug act and potential benefits of the future European orphan drug regulation on tropical diseases. METHODS: This paper presents two analyses: a 1983-97 retrospective study of the United States Orphan Drug Act concerning rare diseases and a prospective study of the European Proposal for a Regulation Concerning Orphan Drugs and its possible impact on tropical diseases. RESULTS: Different programmes have in the past tried to stimulate R & D in this area, but results remain limited. Of 1450 new chemical entities marketed between 1972 and 1997, 13 were specifically for tropical diseases and considered as essential drugs. Between 1983 & 1997, the US Orphan Drug Act approved 837 drugs and marketing of 152 new molecular entities (NMEs). Three NMEs have been designated for malaria and human African trypanosomiasis. Seven others, already commonly used in tropical diseases, received either orphan designation or an orphan approval for another indication. Pharmaceutical companies benefit from the US framework only when the US market exclusivity clause was applicable. Future European orphan drug regulation appears to be similar to the US Orphan Drug Act. CONCLUSION The orphan drug programmes relating to rare diseases have met with some success. Considering tropical diseases rare diseases seems inadequate to boost pharmaceutical R & D. However, some provisions of the European text may be relevant to tropical diseases, admitting the need for a more specific rule for evaluations of this kind of drug and recognizing the existence of 'diseases of exception'.  (+info)

Development of orphan vaccines: an industry perspective. (2/70)

The development of vaccines against rare emerging infectious diseases is hampered by many disincentives. In the face of growing in-house expenditures associated with research and development projects in a complex legal and regulatory environment, most pharmaceutical companies prioritize their projects and streamline their product portfolio. Nevertheless, for humanitarian reasons, there is a need to develop niche vaccines for rare diseases not preventable or curable by other means. The U.S. Orphan Drug Act of 1983 and a similar proposal from the European Commission (currently under legislative approval) provide financial and practical incentives for the research and development of drugs to treat rare diseases. In addition, updated epidemiologic information from experts in the field of emerging diseases; increased disease awareness among health professionals, patients, and the general public; a list of priority vaccines; emergence of a dedicated organization with strong leadership; and the long-term pharmacoeconomic viability of orphan products will be key factors in overcoming the complexity of orphan status and the limited need for vaccine.  (+info)

Functional foods: the Food and Drug Administration perspective. (3/70)

Because the Federal Food, Drug, and Cosmetic Act (FFDCA) does not provide a statutory definition of functional foods, the Food and Drug Administration has no authority to establish a formal regulatory category for such foods. The primary determinant of the regulatory status of a food is its intended use, which is determined largely by the label and labeling information accompanying the product. This information includes nutrient information, nutrient content claims, and various types of health claims. In marketing these foods, manufacturers may come under one of several existing regulatory options. The first decision manufacturers will make that will help determine their product's regulatory status is whether the product is a food or a drug. Thus, manufacturers and retailers have a range of legal and regulatory categories in which their products may be classified. This article describes the definitions provided in the FFDCA for a drug and a food, the safety and labeling requirements of various food categories, and types of possible claims for dietary supplements.  (+info)

What are today's orphaned vaccines? (4/70)

Development costs for new biological agents are increasing, and the time span from laboratory research to introduction of a product on the world market is becoming ever longer. Complex regulatory requirements add barriers and additional costs to early introduction abroad. This results in reluctance by manufacturers to undertake development of a vaccine that will be used for a tropical disease in only the public sector of a poor country. The chances of recovery of huge investment costs before patents expire are not good, unless such a new vaccine can also be sold at high cost in North America and Europe. These are some of the reasons that we still do not have a modern Japanese encephalitis vaccine or products against malaria and dengue fever. Many tropical countries must find a way to develop their own vaccine production facilities. Innovative help for technology transfer will have to be forthcoming, or many new life-saving products will never bridge the gap between research unit and production.  (+info)

Initial lessons from public-private partnerships in drug and vaccine development. (5/70)

In recent years, venture capital approaches have delivered impressive results in identifying and funding promising health discoveries and bringing them to market. This success has inspired public sector experiments with "social venture capital" approaches to address the dearth of affordable treatment and prevention for diseases of the developing world. Employing the same focus on well-defined and measurable objectives, and the same type of connections to pool and deploy resources as their for-profit counterparts, social venture capitalists seek to use the tools and incentives of capitalism to solve one of its biggest failures: the lack of drugs and vaccines for diseases endemic to low-income populations. As part of a larger trend of partnerships emerging in health product donation and distribution, public-private partnerships for pharmaceutical development have led research and development (R&D) efforts to generate more accessible and efficacious products for diseases such as malaria, tuberculosis, and AIDS. In this article, three R&D-focused partnerships are explored: the International AIDS Vaccine Initiative; the Medicines for Malaria Venture; and the newly formed Global Alliance for TB Drug Development. The article highlights key elements essential to the success of these ventures.  (+info)

Building local research and development capacity for the prevention and cure of neglected diseases: the case of India. (6/70)

This paper examines the proposal to build research and development (R&D) capabilities for dealing with neglected infectious and tropical diseases in countries where they are endemic, as a potentially cost- and time-effective way to fill the gap between the supply of and need for new medicines. With reference to the situation in India, we consider the competencies and incentives needed by companies so that their strategy can be shifted from reverse engineering of existing products to investment in R&D for new products. This requires complex reforms, of which the intellectual property rights agreement is only one. We also consider whether Indian companies capable of conducting research and development are likely to target neglected diseases. Patterns of patenting and of R&D, together with evidence from interviews we have conducted, suggest that Indian companies, like multinational corporations, are likely to target global diseases because of the prospect of much greater returns. Further studies are required on how Indian companies would respond to push and pull incentives originally designed to persuade multinational corporations to do more R&D on neglected diseases.  (+info)

Gleevec for the treatment of chronic myelogenous leukemia: US. Food and Drug Administration regulatory mechanisms, accelerated approval, and orphan drug status. (7/70)

Gleevec (imatinib mesylate), a highly promising new drug for the treatment of chronic myelogenous leukemia in blast crisis, in accelerated phase, and in chronic phase after interferon failure or intolerance, received orphan drug status from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development on January 31, 2001, and accelerated approval from the FDA for the above three indications on May 10, 2001. The purpose of this report is to summarize FDA regulatory mechanisms, i.e., accelerated approval and orphan drug regulations, that have permitted patients to receive this drug as rapidly as possible.  (+info)

U.S. Food and Drug Administration drug approval summaries: imatinib mesylate, mesna tablets, and zoledronic acid. (8/70)

The purpose of this report is to summarize information on drugs recently approved by the U.S. Food and Drug Administration. Three drugs have recently been approved: Gleevec (imatinib mesylate) at a starting dose of 400 or 600 mg daily for the treatment of malignant unresectable and/or metastatic gastrointestinal stromal tumors; Mesnex (mesna) tablets as a prophylactic agent to reduce the incidence of ifosfamide-induced hemorrhagic cystitis, and Zometa (zoledronic acid) for the treatment of patients with multiple myeloma and for patients with documented bone metastases from solid tumors, in conjunction with standard antineoplastic therapy. Prostate cancer should have progressed after treatment with at least one hormonal therapy. The recommended dose and schedule is 4 mg infused over 15 minutes every 3-4 weeks. These three drugs represent three different types of drug approval: Gleevec is an accelerated approval and supplemental new drug application (NDA); Mesnex tablets represent an oral formulation of a drug approved 14 years ago as an intravenous formulation, and Zometa represents a standard NDA for a noncytotoxic, supportive-care drug. Information provided includes rationale for drug development, study design, efficacy and safety results, and pertinent literature references.  (+info)

CRAIGAVON, Northern Ireland, November 15, 2012 /PRNewswire/ --. Galen announced today that the European Commission has designated liposomal daunorubicin as an orphan medicinal product for the treatment of acute myeloid leukaemia (AML).[1] The designation follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA).[2] Liposomal daunorubicin is currently approved in a number of European countries, the U.S. and Brazil for the treatment of advanced HIV-related Kaposis sarcoma, as the medicinal product DaunoXome®.. Acute myeloid leukaemia is estimated to affect not more than 1.2 in 10,000 people in the European Union. It is chronically debilitating and life threatening due to bone marrow dysfunction. If left untreated, the condition progresses rapidly and is often fatal.[2]. Galen is dedicated to supporting the development and provision of innovative medicines in an effort to improve health worldwide. Speaking about the European ...
... QUEBEC CITY M... Juergen Engel Ph. D. President and CE...About Orphan Medicinal Product Designation ...Orphan medicinal product designation is granted by the European Commis...,Aeterna,Zentaris,Receives,Positive,Opinion,for,Orphan,Medicinal,Product,Designation,for,AEZS-108,for,the,Treatment,of,Ovarian,Cancer,from,the,Committee,for,Orphan,Medicinal,Products,of,the,European,Medicines,Agency,biological,advanced biology technology,biology laboratory technology,biology device technology,latest biology technology
Looking for Orphan drugs? Find out information about Orphan drugs. drug developed under the U.S. Orphan Drug Act to treat a disease that affects fewer than 200,000 people in the United States. The orphan drug law offers tax... Explanation of Orphan drugs
The 8th annual World Orphan Drug Congress is the marketplace for orphan drug professionals looking at the complete value chain of orphan drug development, from clinical development and R&D to corporate development and market access.. Being Europes largest Orphan Drug Congress, this event will provide a platform for you to showcase your thought leadership and expertise pharma, biotechs, payers, regulators and patient advocates.. Join us in November and make sure you are at the forefront of the orphan drug industry. Regardless of whether your interest lies in research, clinical development, patient access, global pricing and reimbursement or just to engage with patient advocacy groups more intimately, we have content, networking and potential partners for you!. Take just three days out of the office to meet with 400+ potential customers who need to find solutions to challenges around the commercial, regulatory and scientific issues in orphan drugs. Through strategic keynote plenaries, themed ...
There is some good news for mesotheliomasufferers in a recent report from Thomson Reuters on the orphan drug market. According to Reuters, rare diseases like mesothelioma are "winning unprecedented attention" from drug manufacturers. More than a quarter of the 39 new drugs approved by the FDA in 2012 were granted orphan drug status because they were designed to treat diseases (such as mesothelioma) that affect fewer than 200,000 people a year.. Mesothelioma is an aggressive asbestos-linked cancer of internal membranes. It is considered an orphan disease because it claims the lives of about 2,500 Americans annually. The Reuters report on orphan drugs is good news for mesothelioma patients because few treatment options currently exist for them and their prognosis is often poor. By forgoing certain regulations and fees, orphan drugs can be brought to market faster and potentially benefit more people with diseases like mesothelioma.. Two of the orphan drugs approved in 2012 were mesothelioma drugs. ...
GARD has information from the Food and Drug Administration (FDA) on treatments approved for rare diseases, known as orphan products/drugs. The Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product. The following information comes from the FDA database of orphan drug designations and approvals. You can output the data into an Excel spreadsheet from the FDA website. More information on orphan drug development is available from Global Genes and Orphanet ...
The objective of the Orphan Drug Act of 1983 was simple: To inspire drug companies to develop new drugs for small populations - under 200,000 - theyd get a seven-year exclusivity deal and protection from any competition, so long as no one else came up with a clinically superior product. To sweeten the deal, the government added a 50-percent tax credit on all research expenses.. The government got what it wanted. As of 2001, there were some 1,000 designated orphan products and 242 had been given official marketing sanction, all approved by a special orphan drug office, which the OIG gives high marks for responsiveness and ease of access. In 1983, by contrast, there were only a handful of orphan products, and the new drugs created since included big advances for some terrible conditions like cystic fibrosis, hemophilia, and multiple sclerosis.. For some, orphan drugs can make the difference between life and death. But that does not change the realities of the drug development business. Orphan ...
FDA Orphan Drug Designation received earlier this month. , EMA and FDA Orphan designations validate odiparcil potential to improve treatment options for MPS VI patients. , iMProveS phase IIa study in MPS VI patients on track to begin recruitment by year-end 2017. Daix (France), August 29, 2017 at 07:30am CEST - Inventiva, a biopharmaceutical company developing innovative therapies, particularly in fibrosis, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to odiparcil (formerly IVA336) for the treatment of MPS VI.. "This decision could accelerate the availability of a much needed new treatment for MPS VI patients,;" said Christine Lavery, President of the UK MPS Society added Professor Chris Hendriksz, of FYMCA Medical Ltd. and University of Pretoria, South Africa.. "We recently received U.S. orphan drug status and with this new EU designation we continue delivering on our regulatory strategy for odiparcil. Clearly the recent preclinical data we ...
The decision has been communicated by the Agency on June 21 and follows the positive opinion issued by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) on November 12, 2015, recommending the designation of the medicinal product containing Sirolimus as an orphan medicinal product for the indication: treatment of beta thalassaemia intermedia and major.. Marco Prosdocimi, Managing Director of Rare Partners, said that "This important result further confirm that the collaboration we established since 2011 with Roberto Gambari (University of Ferrara) and his collaborators is extremely fruitful. Their research, supported for many years by AVLT (Associazione Veneta per la Lotta alla Talassemia) has obtained outstanding results, with potential application in patients within a short time frame. A collaborative grant, obtained by Rare Partners from Wellcome Trust (UK) within the Pathfinder Award scheme, allowed completion of fundamental preclinical work and ...
CPX-351, a drug therapy for acute myeloid leukemia, has been raised by the U.S. Food and Drug Administration, FDA, to orphan drug status. Now with seven years market exclusivity in the U.S, Celator Pharmaceuticals will continue through to phase 2 clinical trials.. According to medcitynews.com, its goals involve a fund of $5 million, and $2.57m of this target has been raised through equity, options and securities so far.. Understand how governments are collaborating to improve the orphan drugs challenges, learn the strategic solutions, and discover the trends of technology investments at the World Orphan Drugs Congress USA 2012.. The congress speakers consist of business and scientific industry leaders from North America and beyond. Register now, and meet decision-makers from big pharma, early stage and mid stage biotechs, government and regulatory bodies.. ...
An experimental eye treatment under development by Advanced Cell Technology Inc. has received orphan drug status from European authorities.
GARD has information from the Food and Drug Administration (FDA) on treatments approved for rare diseases, known as orphan products/drugs. The Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product. The following information comes from the FDA database of orphan drug designations and approvals. You can output the data into an Excel spreadsheet from the FDA website. More information on orphan drug development is available from Global Genes and Orphanet ...
H3 Biomedicine, a member of Eisais global Oncology Business Group, has received orphan drug designation from the US Food and Drug Administration (FDA) for its H3B-8800, a clinical compound used to treat patients with acute myelogenous leukaemia (AML) and chronic myelomonocytic leukaemia (CMML).. H3 Biomedicine is a clinical stage biopharmaceutical company that focuses on the discovery and development of precision medicines for oncology.. H3B-8800 is a potent, selective and orally bioavailable small molecule modulator of wild-type and mutant SF3b complexes.. H3 Biomedicine president and CEO Dr Markus Warmuth said: "Receiving the orphan drug designation for H3B-8800 is a critical milestone for H3s ongoing cancer genomics driven drug discovery programme.. ...
Why a regulation for Orphan Medicines is needed? Some conditions occur so infrequently that the cost of developing a medicinal product would not be recovered by the expected revenues. Therefore the pharmaceutical industry is unwilling to develop these medicines under normal market conditions. Patients suffering from rare conditions should be entitled to the same quality of treatment as other patients (EC Regulation No 141/2000)
Ensuring the development and availability of drugs that are medically important but commercially unprofitable poses a significant problem in our free market economy. Recent attempts by the federal government to develop a policy regarding these "orphan" drugs provides an important first step. The federal Interagency Task Force on Drugs of Limited Commercial Value recommendations propose initiation of a wide spectrum of administrative and legislative changes; these are designed to create incentives for industry to cooperate with government in developing and making orphan drugs available to those who need them. But before the proposed recommendations are accepted or rejected, a clearer understanding is needed of the nature of interrelated problems of orphan drugs. Thereafter, the wide spectrum of "stakeholders" involved in and affected by orphan drug policies or programs should seek means for interactive planning to determine desired ends and to devise means for achieving them. ...
Tuesday - April 18, 2006, WEST HAVEN, CT and EMERYVILLE, CA Bayer Pharmaceuticals Corporation (NYSE: BAY) and Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) announced today that Nexavar (sorafenib) tabl...
Albireo AB, a biopharmaceutical company specializing in gastroenterology, today announced that the European Medicines Agency Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on an application for orphan medicinal product status for the companys lead hepatology candidate, A4250.
The U.S. Food and Drug Administration has granted a second orphan drug designation to Waltham ophthalmologic device and pharmaceutical developer Avedro Inc. for a treatment of corneal ectasia. The designation relates to VibeX, its riboflavin solution intended for use with UVA irradiation, the KXL system for Corneal Cross-linking, which the company said on its website helps strengthen the cornea following refractive surgery.. SOURCE ...
The Food and Drug Administration on Nov. 21 granted orphan drug designation to rofecoxib (TRM-201), a cyclooxygenase 2-selective nonsteroidal anti-inflammatory
The FDA has granted orphan drug designation to Alvocidib (flavopiridol) to treat patients with acute myeloid leukemia. Alvocidib, a potent c...
Hatfield, England (ots/PRNewswire) - The investigational monoclonal antibody amatuximab (development code: MORAb-009), has today been granted orphan drug designation (ODD) for...
The Food and Drug Admnistration (FDA) has granted Orphan Drug designation for PRM-151 (Promedior) for the treatment of myelofibrosis.
Acceleron Pharma announced that its pulmonary arterial hypertension (PAH) therapy candidate sotatercept was granted orphan drug designation by the FDA.
The Orphan Drug Tax Credit (ODTC) is a key provision of the Orphan Drug Act, and promotes research spending on rare diseases by lowering development costs for manufacturers - but this is in jeopardy: the tax reform bill passed in November by the House would eliminate the credit altogether, and the Senate bill, which passed earlier this month, cut it nearly in half.. The EveryLife Foundation for Rare Diseases, NORD and Global Genes feel that without the Orphan Drug Tax Credit, investment in therapies for rare disease patients is at risk! Before the Orphan Drug Act, manufacturers were often hesitant to invest in developing new treatments for rare diseases because the small patient population made it difficult to recover development costs.. TAKE ACTION NOW to preserve the ODTC by telling Congress why YOU are grateful for it! Use #RareGratitude and put it to good use!. Here are 4 things you can do to show your #RareGratitude. It only takes a few minutes to help 30 million Americans waiting for ...
Guildford, UK, 29 August 2013: ReNeuron is pleased to announce that its ReN003 retinal stem cell therapy candidate for retinitis pigmentosa has achieved a significant regulatory milestone. The therapy has been granted Orphan Drug Designation in both Europe and the US by the European Commission and the Food and Drug Administration, respectively.. Orphan Drug Designation is typically granted to treatments that provide significant benefit to patients with rare diseases that are life-threatening or chronically debilitating. Treatments with this designation benefit from significant commercial and regulatory advantages such as market exclusivity for 10 years from approval in the disease concerned, against other treatments offering no greater therapeutic advantage.. ReNeuron is using its proprietary human retinal progenitor cells as the basis of its ReN003 therapeutic candidate targeting retinitis pigmentosa, a group of hereditary diseases of the eye that lead to progressive loss of sight due to cells ...
Growing number of people suffering orphan diseases boosts the orphan drugs market. Know the Orphan drugs market trend by disease type, product type & others.
Orphan drugs are a relatively niche segment because they are used for the treatment of rare or orphan diseases. Numerous trends are shaping up the orphan drugs market and it becomes imperative for stakeholders to learn about these trends and be prepared for change.
The Orphan Drug Act permits the Secretary of Health and Human Services (the "Secretary") to designate a drug as an orphan drug. According to statute, "[t]he manufacturer or the sponsor of a drug may request the Secretary to designate the drug as a drug for a rare disease or condition." 21 U.S.C. § 360bb(a)(1). The statute further instructs that, if the Secretary finds that the drug "is being or will be investigated for a rare disease or condition" and the approval, certification or licensure of that drug "would be for use for such a disease or condition," the Secretary "shall designate the drug as a drug for such disease or condition." Id. The Food and Drug Administration (FDA), an agency under the control of the Secretary, oversees the designation and approval of orphan drugs.. Distinction between orphan drug designation and approval to market the drug. The designation of a drug as an orphan drug is separate from approval to market the drug in the United States. Even though a drug has been ...
Meet Orphan Drug scientists and Experts in Rare Diseases from USA (America) UK, Europe, Middle East and Asia Pacific countries- Baltimore, Dubai, Spain, Brisbane, Bangalore, Philadelphia, Chicago, Boston, New York, Switzerland, San Antonio at Orphan Drugs conferences and Pharmaceutical Events held from May 15-17, 2017 Munich, Germany
...THE WOODLANDS Texas March 20 2012 /- a href ht... The Orphan Drug designation of telotristat etiprate by the FDA provid...In the U.S. Orphan Drug designation is generally granted for drugs in...,FDA,Grants,Lexicon,Orphan,Drug,Designation,for,Telotristat,Etiprate,for,the,Treatment,of,Carcinoid,Syndrome,biological,advanced biology technology,biology laboratory technology,biology device technology,latest biology technology
GW Pharmaceuticals plc (AIM:GWP) (Nasdaq:GWPH) ("GW") announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Epidiolex®, GWs product candidate that contains plant-derived Cannabidiol (CBD) as its active ingredient, for use in treating children with Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood-onset epilepsy. Epidiolex is an oral liquid formulation of a highly purified extract of CBD, a non-psychoactive molecule from the cannabis plant.. In November 2013, GW announced that the FDA had granted orphan drug designation for Epidiolex in the treatment of Dravet syndrome. Following the successful follow-on offering completed in January 2014, GW intends to advance a full clinical development program for Epidiolex for the treatment of both LGS and Dravet syndromes. Working with leading pediatric epilepsy specialists in the United States, GW expects to commence an initial Phase 2 clinical trial for Epidiolex in the second half of ...
The debate over cost and access to drugs has long raged between patients, health advocates, and pharmaceutical companies. For patients with "orphan diseases," or rare diseases which affect fewer than 200,000 people in the United States, this debate becomes particularly acute, as the Orphan Drug Act passed by Congress in 1983 threatens to drive up prices for highly specialized treatments. While the Orphan Drug Act has helped to bring drugs for rare diseases to millions of patients and continues to stimulate research and development of orphan drugs, the law is certainly not without its problems and caveats.. Continue Reading. ...
Orphan Drug Designation (ODD) in the U.S. means that a drug has been designated by the U.S. FDA as an orphan drug pursuant to the Orphan Drug Act of 1983. This law was passed to facilitate the development of drugs for rare diseases that affect small numbers of people living in the U.S. (which is defined as affecting less than 200,000 people in the U.S.). Orphan drug designation does not mean that the drug is safe or effective, and does not mean that it is legal to market or sell the drug in the U.S. Rather, the designation means only that the drugs sponsor qualifies for certain benefits from the U.S. government, including tax incentives and market exclusivity for 7 years. Orphan drugs normally follow the same regulatory approval process as other drugs (including dosing, safety and efficacy). However, some statistical requirements are lessened (such as not requiring the same number of patients in a Phase III clinical trial as would be required for a non-orphan drug), in order to try to provide ...
The US FDA now gives special "orphan drug" status to potential treatments for rare diseases, changing the approach of regulators and pharma companies
The FDAs Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Orphan designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.. About Pracinostat. Pracinostat is an orally available histone deacetylase (HDAC) inhibitor that has been tested in a number of Phase I and Phase II clinical trials in advanced hematologic disorders and solid tumor indications in both adult and pediatric patients. Pracinostat has been generally well tolerated in more than 200 patients to date, with readily manageable side effects that are often associated with drugs of this class, such as fatigue. In a Phase I dose-escalation trial, Pracinostat demonstrated evidence of single-agent ...
WALTHAM, Mass. - July 21, 2014 - Proteon Therapeutics Inc., a privately held biopharmaceutical company developing novel, first-in-class pharmaceuticals to address the critical medical needs of patients with kidney and vascular diseases, announced today that the first patient has been treated in a pivotal Phase 3 clinical study of its lead product, PRT-201, in chronic kidney disease (CKD) patients undergoing surgical placement of an arteriovenous fistula (AVF). PRT-201, a locally-acting recombinant human elastase, is an investigational drug that may prolong the patency and reduce the failure of hemodialysis vascular access in patients with CKD. PRT-201 has received fast track and orphan drug designations from the U.S. Food and Drug Administration (FDA) and orphan medicinal product designation from the European Commission for hemodialysis vascular access indications.. "Hemodialysis is a life-saving treatment for patients with CKD, but many of these patients suffer from vascular access failure, ...
Due to the promising early results seen in patients with primary brain cancer, Reata has begun clinical trials of RTA 744 in patients who have other types of tumors (for example, lung or breast cancer) that have spread to the central nervous system (CNS). Reata Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing novel treatments for cancer, inflammation, and neurodegenerative diseases. Reata is matching its clinical and preclinical drug development programs with a best-of-class drug discovery platform to identify small molecule chaperones that can induce proper folding of p53, SOD, and Tau, misfolded proteins that are involved in cancer and neurodegenerative disease.
BioWorld Online is the news service of record for the biotechnology industry and is updated every business morning. BioWorld Online will keep you up to date on all of the industrys business, science and regulatory news -- mergers and collaborations, FDA hearings and results, breakthroughs in research and much more.
BONITA, CA--(Marketwired - October 09, 2015) - The combination of PharmaCyte Biotechs (OTCQB: PMCB) signature live-cell encapsulation technology, Cell-in-a-Box® with low doses of ifosfamide has already won the FDAs Orphan Drug status here in the U.S
The disease may manifest in patients with symptoms of chronic pain, difficulty swallowing, poor growth, malnutrition, and weight loss.
re|


Approval Provides Seven Years of FDA Marketing Exclusivity





</pre>
<p>HOBOKEN, N.J., <span class=xn-chron>Aug. 2</span> /CNW/ -- FDA Confirms Orphan Drug Exclusivity Approval for wilate®, Octapharma USAs
New Therapy for Common Inherited Bleeding Disorder.
LACHEN, Switzerland, Jan. 13 /PRNewswire/ -- Octapharma Receives Orphan Drug Exclusivity Approval for wilate(R) - A Replacement Therapy Developed...
Copyright ©2020 NORD - National Organization for Rare Disorders, Inc. All rights reserved. NORD is a registered 501(c)(3) charity organization. Please note that NORD provides this information for the benefit of the rare disease community. NORD is not a medical provider or health care facility and thus can neither diagnose any disease or disorder nor endorse or recommend any specific medical treatments. Patients must rely on the personal and individualized medical advice of their qualified health care professionals before seeking any information related to their particular diagnosis, cure or treatment of a condition or disorder. ...
TORONTO, ON--(Marketwired - May 18, 2017) - On Thursday, June 1, 2017, Xtalks will host a complimentary webinar featuring Dr. Carlos Camozzi, Chief Medical Officer and Dr. Fabrice Chartier, Chief Operating Officer, both from Simbec-Orion Group, as the speakers. Discussion topics include:Key challenges to improve cost-efficiency in clinical development of...
Martin A. Makary, MD, MPH, FACS. Professor of Surgery, Johns Hopkins School of Medicine Professor of Health Policy & Management, Johns Hopkins University School of Public Health Chief, Islet Transplant Surgery Johns Hopkins Hospital. "Surgery and the Future of Health Care" - Makary is an advocate for innovation in medicine and physician-led initiatives such as The Surgical Checklist, which he developed at Johns Hopkins, and was later popularized in Atul Gawandes best-selling book Checklist Manifesto.[7] In 2016, Makary and his colleagues exposed loopholes in the Orphan Drug Act accounting for higher drug pricing. His article "The Orphan Drug Act: Restoring the Mission to Rare Diseases",[8] covered by Kaiser Heath News,[9] led Senator Grassleys office to announce an investigation[10] into the problem. Makary has advocated for the need for more transparency in healthcare and argued that professional physician associations are in the best position to define and endorse the validity of quality ...
Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases. They are "orphans" because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.. ...
The U.S. Food and Drug Administration has granted orphan drug designation for San Diego-based Organovos 3D bioprinted tissue treatment of a protein deficiency disease. The designation paves the way for more frequent FDA interactions, tax credits for clinical research costs and the potential for seven years of marketing exclusivity after the drug is approved.. Organovo designs and creates functional, three-dimensional human tissues for use in drug discovery, clinical development and therapeutic applications. The companys NovoTissues is intended to treat alpha-1 antitrypsin deficiency, a condition in which the body does not make enough of a protein that protects the lungs and liver from damage. The condition can lead to, among other things, liver disease. "The FDAs rapid action recognizes the importance of developing regenerative medicine therapeutic applications, and mirrors our own urgency in addressing this devastating disease. With tens of thousands of patients being treated for inborn ...
The Institute for OneWorld Health announced today it has received Orphan Drug Designation from the two leading regulatory agencies in the world, the FDA and the European Agency for the Evaluation of Medicinal Products (EMEA), for paromomycin to treat visceral leishmaniasis (VL).
PHOENIX, AZ--(Marketwired - Sep 29, 2014) - Insys Therapeutics, Inc. (NASDAQ: INSY), a specialty pharmaceutical company that is developing and commercializing innovative drugs and novel drug delivery systems, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its pharmaceutical cannabidiol (CBD) for...
Agios Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the companys first-in-cl
It was awarded orphan drug status. DCVax-Direct is a therapy to treat inoperable solid tumors in Phase 1 trials in the US. ... For each of the three, the production process is identical regardless of the patient, and even regardless of the solid tumor ... This gives NWBO an enormous production cost advantage over these older therapies and over current would-be competitors, in part ... Combined with centralized automatic mfg, this greatly simplifies large scale production, potentially allowing cost efficiency ...
JR-031 was granted orphan drug priority review. If successful, it will be the first allogeneic cell-based product approved in ... Mesoblast plans to file production registration with the U.S. FDA in 2016 and will commercially launch the product in New ... Mesoblast said they were in the process of completing an Investigational New Drug (IND) submission to the United States FDA to ... Drugs may include anti-inflammation, steroids, cortisone, immune system suppressors, Remicade, antibiotics, anti-diarrheal and ...
... has been granted orphan drug status in the United States, Australia, and Japan. Cerezyme was one of the drugs ... manufactured at Genzyme's Allston, Massachusetts plant, for which production was disrupted in 2009 after contamination with ... CS1 maint: Uses authors parameter (link) "Imiglucerase on Orpha.net: The portal for rare diseases and orphan drugs". Erin ... Other drugs for the treatment of Gaucher's disease Afegostat (development terminated) Eliglustat Miglustat Velaglucerase alfa ...
The FDA Office of Orphan Products and Development offers an Orphan Drug Designation program (ODD) that allows drugs aimed to ... Ezutromid is projected to have the potential to treat all patients suffering with DMD as it maintains the production of ... "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 2016-11-14. Commissioner, Office of the. " ... Both the FDA and European Medicines Agency has given ezutromid an orphan drug designation. ...
"Meveol: orphan drug status granted by the FDA for the treatment of cystic fibrosis". United States Food and Drug Administration ... Thiocyanate secretion in cystic fibrosis patients is decreased resulting in a reduced production of the antimicrobial ... Lactoferrin with hypothiocyanite for the treatment of cystic fibrosis has been granted orphan drug status by the EMEA and the ... "Public summary of positive opinion for orphan designation of hypothiocyanite / lactoferrin for the treatment of cystic fibrosis ...
Investigational Sarcoma Drug GPX-150 Gets Orphan Drug Designation. 2016 Archived 2016-01-24 at the Wayback Machine. "Drug ... More efficient production techniques have brought the price down to $1.1 million per kg for the nonliposomal formulation. ... Ravina, Enrique (2011). The Evolution of Drug Discovery: From Traditional Medicines to Modern Drugs. John Wiley & Sons. p. 291 ... Doxorubicin and several chemotherapeutic drugs (including cyclophosphamide) cause dyspigmentation. Other groups of drugs that ...
... "orphan drug" by the US FDA. Leveraging this background, the company has confirmed its commitment to developing drugs with ... Construction began in 1964 of an industrial facility in Pomezia equipped with state-of-the-art automatic production machinery ... Orphan drugs are medicines that are effective in the treatment of certain diseases that are not manufactured or put on the ... Sigma-Tau has always proved to be a groundbreaker in the discovery of drugs for rare and orphan diseases: for instance, ...
... orphan drug development "Norway's sovereign-wealth fund passes the $1trn mark". The Economist. 21 September 2017. Aizenman, ... The exclusion was upheld because of the company's production of nuclear missiles. Energy portal The budgetary rule - concerning ... EADS was initially excluded because it produced cluster munitions components, but the company later stopped such production. ... involved in businesses such as arms production, tobacco and fossil fuels. The Petroleum Fund's Advisory Council on Ethics was ...
In 1991 the FDA approved the first version of Genzyme's orphan drug Alglucerase (brand name Ceredase), the only treatment for ... research and development for other drugs and to allow them to fund programs that distribute a small portion of production for ... Genzyme's added revenue from profits on the highly priced orphan or specialty drugs like imiglucerase, which had no competition ... According to Express Scripts 2007 Drug Trend Report in 2007 there was a 14% increase in specialty drugs. There was a 60.4% ...
It is co-marketed by Merck Serono and Pfizer in the US under an exception to the Orphan Drug Act. It was approved in Europe in ... In vitro, interferon beta reduces production of Th17 cells which are a subset of T lymphocytes believed to have a role in the ... Avonex and Rebif are under the top 10 best-selling multiple sclerosis drugs of 2013: It is an example of a specialty drug that ... originally under competition protection in the US under the Orphan Drug Act. Avonex is sold in three formulations, a ...
... drug packaging MeSH J01.576.318.321.400 --- drug labeling MeSH J01.576.318.600 --- orphan drug production MeSH J01.576.370 --- ... drug packaging MeSH J01.576.761.300.400 --- drug labeling MeSH J01.576.761.400 --- food packaging MeSH J01.576.761.400.450 --- ...
Although not approved for use in any country, mannitol dry inhalation powder, has been granted orphan drug status by the FDA ... Inhaled steroid therapy that is consistently adhered to can reduce sputum production and decrease airway constriction over a ... Waknine, Yael (27 July 2005). "Orphan Drug Approvals: Bronchitol, Prestara, GTI-2040". Medscape today for WebMD. Archived from ... Symptoms typically include a chronic cough with mucus production. Other symptoms include shortness of breath, coughing up blood ...
... is a Norwegian pharmaceutical company specializing in the production and marketing of orphan drugs. These medicines ... in 2007 the company has been chartered by the Norwegian Ministry of Health and Care Services to improve global access to orphan ...
... and whether the ODA has truly stimulated the production of non-profitable drugs; the act also has been criticised for allowing ... The Orphan drugs strategy List of European Orphan Drugs USA Food and Drug Administration: The Orphan Drug Act (as amended) US ... Number of orphan drugs in clinical trials: 600 Number of orphan drugs in Phase-2 Trial: 231 Number of orphan drugs in US ... of orphan drugs were biologicals. The US dominated the development of orphan drugs with more than 300 orphan drugs in clinical ...
"The human orphan nuclear receptor PXR is activated by compounds that regulate CYP3A4 gene expression and cause drug ... Production of Neuroactive Steroids1". The Journal of Clinical Endocrinology & Metabolism. 82 (12): 4064-4068. doi:10.1210/jcem. ... Alexandros Makriyannis; Diane Biegel (4 November 2003). Drug Discovery Strategies and Methods. CRC Press. pp. 190-. ISBN 978-0- ...
The FDA has granted SER-109 Orphan Drug, as well as Breakthrough Therapy, designations. SER-109 is currently in a Phase 3 trial ... Berry founded Indigo to pioneer microbial solutions that naturally promote plant health and improve agricultural production by ... KSQ's approach has allowed it to advance a pipeline of cancer and immune disease drug programs against novel therapeutic nodes ... ". "KSQ Therapeutics Emerges With $76 Million In Financing To Pioneer High-Confidence Drug Development". "HOME". "HOME". " ...
The U.S. Food and Drug Administration and the European Medicines Agency had granted lintuzumab orphan drug status for treatment ... body produces excessive numbers of abnormal white blood cells that accumulate in bone marrow and interfere with the production ... The drug had been developed by Seattle Genetics as a treatment for acute myeloid leukemia (AML), a condition which results in ... "Leukemia Drug Trial Fails", The New York Times, September 13, 2010. Accessed September 13, 2010. Staff. "Disappointing Phase II ...
... and would have had drugs developed regardless of the legislation), and whether the ODA has really stimulated the production of ... The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs - drugs for rare ... While orphan drug status is given to drugs with "no reasonable expectation" of profitability, some orphan drugs have gone on to ... Only thirty-eight orphan drugs had been approved prior to the 1983 Act; by 2014 "468 indication designations covering 373 drugs ...
Aldurazyme enjoys orphan drug status in both the United States and the European Union, though in both its orphan drug ... BioMarin is responsible for the production of Aldurazyme. It sells the finished product to Genzyme, which is a fully owned ... Orphan drug exclusivity, which prevents the FDA or similar European body from approving the same drug proposed by another ... FDA Orphan Drug Designations and Approvals. FDA website. Accessed December 15, 2015. United States Securities and Exchange ...
ERT was not used in clinical practice until 1991, after the FDA gave orphan drug approval for the treatment of Gaucher disease ... These disorders arise because of genetic mutations that prevent the production of certain enzymes used in the lysosomes. The ...
1 million grant from the National Institute on Drug Abuse. Obtained Orphan Drug designation from the FDA for smoked marijuana ... National Institute on Drug Abuse (NIDA) has a government granted monopoly on the production of medical marijuana for research ... to obtain a license from the Drug Enforcement Administration for a marijuana production facility. Sponsored analytical research ... Anticipating that the Drug Enforcement Administration (DEA) would move to criminalize MDMA in light of the drug's increasing ...
The United States Food and Drug Administration granted an orphan drug designation to Sym001. NIAID/NIH: Sym002 developmental ... This technology enables the production of consistent mixtures of polyclonal antibodies with high batch-to-batch consistency at ... Rozrolimupab (Sym001), an orphan drug, is a recombinant polyclonal antibody consisting of twenty-five monoclonal antibodies ... The company was founded in 2000 and has patents on a drug discovery platform called Symplex and a drug manufacturing platform ...
... which are called orphan drugs in the United States, MSF started this campaign in November 1999 to bring more awareness to the ... They are also urging a rapid scale-up in the use and production of Ready-to-Use Foods (RUF) to reduce childhood deaths from ... Sometimes, the only drugs available are highly toxic or ineffective, and they often have to resort to inadequate testing ... It is campaigning for new TB drugs and simple diagnostics to be developed while continuing to call to attention the serious ...
Orphan drugs[edit]. In April 2015, AstraZeneca's drug tremelimumab was approved as an orphan drug for the treatment of ... Unlike Erythropoietin (EPO), which directly stimulates the production of red blood cells, FG-4592 is taken orally, and ... "AstraZeneca immune system drug wins orphan status in rare cancer", Reuters, London, retrieved 13 July 2015. ... where it will work on biotech drugs), and Mölndal (near Gothenburg) in Sweden, for research on traditional chemical drugs.[5] ...
During 2005 and 2006 Solbec was granted orphan drug designation for Coramsine by the U.S. Food and Drug Administration in the ... Coramsine also has the potential to modulate the production of interleukin-6. Cham, Bill (2007). "Solasodine Rhamnosyl ... Coramsine (SBP002) was an experimental cancer drug that was evaluated in preliminary clinical trials, but was abandoned by ... "Developing Products for Rare Diseases & Conditions". U.S. Food and Drug Administration. Retrieved 15 October 2008. " ...
This produces about 70% of the rice crop grown in a given year.[17] Crucially, the (debated) shortfall in rice production in ... People would tie a rope around the necks and drag them over to a ditch."[249] Corpses were stacked along the streets of ... In addition to the tens of thousands of children who were orphaned,[265] many were abandoned by the roadside or at orphanages ... The bodies were picked over by vultures and dragged away by jackals. Sometimes this happened while the victim was still living. ...
The Powerful and Evolving Role of Patient Advocacy Groups in Orphan Drug Development. Life Sciences, Pharmaceutical, ... In recent years, patient advocacy groups have expanded their influence over the drug development continuum from discovery to ... She is a member of the National Organization for Rare Disorders and the Drug Information Association. ... supporting the strategic development of products that address unmet medical needs associated with rare and orphan afflictions. ...
Harley-Davidson is sliding after Trump backs boycott if it moves production overseas (HOG). Business Insider ... has granted Orphan Drug Designation to pracinostat, an investigational drug candidate currently in a Phase 3 study in ... The EMA orphan drug designation is a status assigned to a medicine intended for use against a rare condition in the European ... The orphan drug designation is based on the scarcity of treatments for patients suffering from AML and on positive Phase 2 ...
Orphan Drug Development & Production: Turning Lemons into Lemonade Forming a "win-win-win" paradigm for large pharma companies ... Flu Vaccine Production at Sanofi Pasteur first doses of Fluzone released by the U.S. Food and Drug Administration (FDA) for ... GE Increases Cell Culture Media Production Annual production capacity of powdered cell culture media to increase by tenfold in ... Hovione Expands Production Capacity New commercial scale equipment for blending, tableting and coating will be added for oral ...
Here, several methods were investigated to improve production and activity of rhGALNS. These methods involved the use of ... Together, these results describe advances in the current knowledge on the production of human recombinant enzymes in a ... we demonstrated production of an active recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS) enzyme in Escherichia ... Expert Opinion on Orphan Drugs 3, 1279-1290, doi:10.1517/21678707.2015.1086640 (2015). ...
Prosensa achieves orphan drug status on its entire Duchenne muscular dystrophy portfolio. Prosensa has achieved orphan drug ... New salmon peptone - sustainable marine resources used in the production of high-value biotech products A project has ... Roche and IMI promote use of stem cell technology to enhance drug development. Roche and the Innovative Medicines Initiative ... PA by the Food and Drug Administration (FDA). ... of the use of marine derived peptones in the production of high ...
Production: Dawn Flook, Director of Production Services. Circulation: Jackie Ott, Circulation Manager. Webmaster: Webmaster ... About Orphan Drug Status. Under the Orphan Drug Act, orphan drug designation gives companies access to protocol assistance to ... A drug may also receive orphan drug designation if it is used to treat a disease that affects more than 200,000 people in the ... has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). The U.S. prevalence of MF is estimated at ...
Production: Dawn Flook, Director of Production Services. Circulation: Jackie Ott, Circulation Manager. Webmaster: Webmaster ... The FDAs orphan-drug designation program provides special status and incentives to encourage the development of drugs for ... Food and Drug Administration has granted orphan drug designation to NSI-189 for the treatment of Angelman syndrome. ... "Orphan drug designation is an important regulatory milestone in the development of NSI-189 and we are committed to evaluating ...
Amryt Pharma: Cantor Fitzgerald backs orphan drug group for NASDAQ success 3 days, 10 hours ago ... Noble Capital says Great Panther Minings 2Q production figures... 2 days, 6 hours ago ... Mandalay Resources delivers more strong production and sales in... 3 days, 7 hours ago ... Great Panther Mining releases new production figures that... 3 days, 9 hours ago ...
Amryt Pharma: Cantor Fitzgerald backs orphan drug group for NASDAQ success * Carnival secures US$10bn refinancing as it swings ... Noble Capital says Great Panther Minings 2Q production figures... 2 days, 3 hours ago ... Amryt Pharma: Cantor Fitzgerald backs orphan drug group for NASDAQ success * Carnival secures US$10bn refinancing as it swings ... Amryt Pharma: Cantor Fitzgerald backs orphan drug group for NASDAQ success 3 days, 7 hours ago ...
... global orphan drug conference and expo where orphan drug stakeholders gather to talk about strategies to expedite orphan drug ... Developing commercial manufacturing capabilities during pre-clinical and clinical - ensuring successful vector production, ... World Orphan Drug Congress USA 2019. 10 - 12 April. Gaylord National Harbor Hotel, Oxon Hill, MD ... Janet Maynard, Acting Director,Office of Orphan Product Development, F.D.A. U.S. Food and Drug Administration ...
PLX-200 also activates PPARα, which enhances production of transcription factor EB (TFEB) in brain cells. TFEB then binds to ... Polaryx Therapeutics Receives Orphan Drug Designation From the U.S. FDA for PLX-200 for Rare Pediatric Disease This new drug ... Under the U.S. Orphan Drug Act, the FDAs Office of Orphan Products Development provides sponsors with special status and ... Orphan Drug Designation provides seven years of market exclusivity if the drug candidate receives regulatory approval together ...
Members are companies active in modern biotechnology, such as R&D, Production, Marketing and Sales, Finance, Services and ... Prices of orphan drugs vs. rarity of disease - is there a link?. • Are orphan drug prices increasing or decreasing since the ... What are the criteria for orphan drugs in EU and US?. *What are the incentives available to companies developing orphan drugs? ... He has since 2009 been engaged in the development of Orphan Drugs both in TikoMed and as a board member of Orphan Drug ...
EC grants Orphan Drug Designation to FLT190, a Fabry Disease treatment. By Hannah Balfour (European Pharmaceutical Review) ... Drug delivery method leads to sustained HIV antibody production in study. By Victoria Rees (European Pharmaceutical Review) ... These drugs are frequently prescribed to treat behavioural and psychiatric symptoms of dementia, which can be worsened by other ... a decrease in symptomatic drug use. The study is also the worlds first nationwide study on the subject. The results provide ...
There may be some rules for the most efficient orphan drug development but they should be adapted to challenges presented by ... Despite the multiple attractiveness of orphan drugs cash burn minimisation is one of the major challenges faced by developers. ... The completeness of the non-clinical experience and the adequate product development/production campaign are relevant factors ... However, the median current cost per patient is still 5.5 times higher for orphan drugs compared to non-orphan Drugs. ...
With the development of the medicine proceeding, the company started looking for a new facility suitable for production. The ... Recognising an orphan drug. The next step in developing Plasminogen was to have it officially recognised as an orphan drug in ... Developing orphan drugs for orphan diseases in Tuscany, Italy. Ligneous conjunctivitis is a rare form of chronic conjunctivitis ... Orphan drugs are medicines that are commercially unavailable because of high research costs and a low potential for profit. In ...
Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT February 15th, 2018 ... Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT February 15th, 2018 ... Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT February 15th, 2018 ... Production of Nanofiltration Membranes with High Stability in Non-aqueous Media. Tehran, Iran , Posted on September 14th, 2013 ...
Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT February 15th, 2018 ... Home , News , Nano production lines. August 22nd, 2007 Nano production lines Abstract:. Researchers in Switzerland have built ...
Global Orphan drug industry Report 2015 is a professional and in-depth study on the current state of the Orphan drug industry. ... 4 Production Analysis of Orphan drug by Regions, Technology, and Applications. 4.1 Global Production of Orphan drug by Regions ... 1.2 Classification of Orphan drug. 1.3 Applications of Orphan drug. 1.4 industry Chain Structure of Orphan drug. 1.5 industry ... Regional Overview of Orphan drug. 1.6 industry Policy Analysis of Orphan drug. 1.7 industry News Analysis of Orphan drug. 2 ...
It is an orphan drug. Erdosteine stimulates mucus production and increases mucociliary transport, which improves expectoration ...
Such plans have caused disturbances in the production and supply chain.. Global Orphan Drugs Market Analysis 2020: ... Accounting to the massive potential held by orphan drugs, there are numerous clinical trials associated with the drugs. The ... The global orphan drugs market size is projected to reach USD 340.84 billion by the end of 2027. Increasing number of ... Orphan drugs are used for the treatment of severe and life-threatening diseases. The high emphasis on research and development ...
... orphan drug designation in biliary tract cancer (BTC) - EMA orphan drug designation for M7824 in ... ... All implants are manufactured in the companys own production facilities in Dieburg near ... ... Merck Announces EMA Orphan Drug Designation for Bifunctional Immunotherapy M7824 in Biliary Tract Cancer. Darmstadt, Germany ( ... Merck Announces FDA Orphan Drug Designation for Bifunctional Immunotherapy M7824 in Biliary Tract Cancer. Darmstadt, Germany ( ...
The biotechnology companys stock has been rising since Thursday when it received orphan drug designation from the European ... Talecris received orphan drug designation for Plasmin from the U.S. Food and Drug Administration in 2009. ... Indirect-acting thrombolytics, such as Plasminogen activators, target clots indirectly by first stimulating the production of ... The biotechnology companys stock has been rising since Thursday when it received orphan drug designation from the European ...
... announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug designation to omacetaxine for the ... Due to the insufficient production of red, white, or platelet blood cells, MDS patients are more susceptible to chronic anemia ... has granted Orphan Drug designation to omacetaxine for the treatment of Myelodysplastic Syndromes (MDS). Orphan drug ... "FDA Grants Orphan Drug Designation To ChemGenexs Omacetaxine For The Treatment Of Myelodysplastic Syndromes." Medical News ...
In March of this year ACTs RPE cells were granted Orphan Drug Status by the FDA.. Within the past week ACT received NIH ... Advanced Cell Technology Awarded Broad Patent for Production of Retinal Pigment Epithelial Cells. This broad patent gives ... At the end of last year the Company filed an Investigational New Drug Application with the FDA.. In February of this year the ... "The efficient production of highly pure RPE cell preparations derived from human embryonic stem cells represents the first ...
Cell & Gene Therapy Development & Production. Rare Diseases & Orphan drugs. Stem Cell Research and Regenerative Medicine. ...
  • This diverse range in manufacturing scale coupled with thousands of biologics in the development pipeline, has had significant impact on the design of biologics production models. (bioprocessintl.com)
  • Amarantus BioScience Holdings, Inc. (OTCQB:AMBS) , a biotechnology company focused on developing therapeutic and diagnostic products for neurological disorders and orphan indications, today announced that it has entered into a manufacturing agreement with Catalent Pharma Solutions (NYSE:CTLT) , the leading global provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products, for clinical-grade production of MANF (mesencephalic-astrocyte-derived neurotrophic factor). (biomedreports.com)
  • In 2018, the share of orphan drug sales in the blood therapy category was 31.4 percent compared to a forecasted 27.9 percent in 2024. (statista.com)
  • In 2018, Mammalian cell culture systems submarket represents the largest submarket within the biological drug API manufacturing industry. (visiongain.com)
  • What are the market shares of each main segment of the overall biological drug API manufacturing services market in 2018? (visiongain.com)
  • PARAMUS, N.J. , Aug. 7, 2017 /PRNewswire/ -- Polaryx Therapeutics, Inc, a biotech company developing oral small molecule therapeutics for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) and for other forms of NCL, commonly known as Batten disease, has been granted Orphan Drug Designation from the U.S. FDA for PLX-200 to treat LINCL. (prnewswire.com)
  • Dr. Kalipada Pahan , Ph.D., a professor of neurological sciences, biochemistry, and pharmacology, and the Floyd A. Davis, M.D., Endowed Chair in Neurology at the Rush University Medical Center in Chicago , offered a joint statement that the Orphan Drug Designation was a significant achievement in validating the new therapeutics. (prnewswire.com)
  • Polaryx Therapeutics, Inc. is solely dedicated to developing drug candidates for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) and other forms of NCL. (prnewswire.com)
  • Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Dicerna's DCR-A1AT for the treatment of alpha-1 antitrypsin (A1AT) deficiency. (yahoo.com)
  • PharmAbcine Inc., a clinical-stage biotech company developing novel antibody therapeutics for multiple cancer indications, announced today that U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its leading clinical compound TTAC-0001 for "treatment of Glioblastoma Multiforme. (ntbinfo.no)
  • The advanced technologies of the Company include innovative platforms for the production of protein therapeutics, technology and processes for the purification and formulation of these products, as well as technologies in the field of tissue repair (via its collaboration with Novathera) and DNA-repair (via its acquisition of DNage BV). (webwire.com)
  • • AnalytiCon Discovery GmbH , of Potsdam, Germany, began a multiyear collaboration to identify novel drug candidates in oncology with the Experimental Therapeutics Center at Memorial Sloan-Kettering Cancer Center. (bioworld.com)
  • SMi is proud to announce the return of their 6th annual Orphan Drugs Europe event to Berlin on the 15th -16th May 2017! (smi-online.co.uk)
  • If these results are reproduced in the second Phase 3 study and are supported by the results of a planned interim analysis of the Phase 3 extension study, the company expects to submit a New Drug Application with the U.S. Food and Drug Administration in the first quarter of 2017. (drugs.com)
  • A document tabled recently in the House of Commons in answer to a question from Kmiec shows that Health Canada granted marketing approval to 85 orphan drugs between May 2013 and June 2017. (nationalpost.com)
  • The key problem in the production of human recombinant proteins in this bacterial host is related with the lack of post-translational modifications, as glycosylations, and poor protein folding, leading to loss of enzyme activity and the formation of insoluble protein aggregates 14 . (nature.com)
  • Three of those drugs have been labled potential blockbusters that have several big pharmaceutical companies lining up as interested partners. (biomedreports.com)
  • CAMBRIDGE, MA -- MIT researchers have developed a compact, portable pharmaceutical manufacturing system that can be reconfigured to produce a variety of drugs on demand. (brightsurf.com)
  • Active pharmaceutical ingredients are synthesized in chemical manufacturing plants and then shipped to other sites to be converted into a form that can be given to patients, such as tablets, drug solutions, or suspensions. (brightsurf.com)
  • It could also be useful in regions with few pharmaceutical storage facilities, because drugs can be produced on demand, eliminating the need for long-term storage. (brightsurf.com)
  • Without a framework in Canada, Wong-Rieger said, pharmaceutical companies may not bother to apply for marketing approval for their orphan drugs in this country. (nationalpost.com)
  • The National Pharmaceutical Pricing Authority ( "NPPA" ) is empowered by DPCO to fix ceiling prices of drugs and Notified Medical Devices that are listed in the schedule appended to the DPCO ( "Scheduled Formulations" ). (mondaq.com)
  • Drug products combine active pharmaceutical ingredients with excipients in a final formulation for delivery to patients in liquid or lyophilized (freeze-dried) packaged forms - with the latter requiring reconstitution in the clinical setting. (bioprocessintl.com)
  • Needless to say, if the ITC rules in favor of KV, it would set a terrible precedent for active pharmaceutical ingredients imported for other compounded drugs. (anh-usa.org)
  • It is unlikely other pharmaceutical companies will try to spend the money to manufacture the drug, given its history. (anh-usa.org)
  • Now KV Pharmaceutical has been given the exclusive right of production and sale (not to mention drug trial tax breaks! (anh-usa.org)
  • An orphan drug is a pharmaceutical agent that has been developed specifically to treat a relatively rare medical condition, and the designation gives the manufacturer clinical trial tax incentives. (anh-usa.org)
  • The Indian pharmaceutical industry is the world's third largest drug producer by volume and the country's market manufactures 60 percent of vaccines globally. (europeanpharmaceuticalreview.com)
  • In the present situation, the Indian government should take important steps to remove the technical and financial barriers that will spur the pharmaceutical industry to ramp up API production, reducing the dependency of the pharmaceutical industry as a whole on the heavily impacted Chinese market. (europeanpharmaceuticalreview.com)
  • The market for rare disease products is continuously growing, expecting to reach $176 billion by 2020, with a CGR of 10.5% just for orphan drugs - this is twice the growth rate of the overall prescription drug market (5.3% CGR from 2014-2020). (smi-online.co.uk)
  • Previously, we demonstrated production of an active recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS) enzyme in Escherichia coli as a potential therapeutic alternative for mucopolysaccharidosis IVA. (nature.com)
  • In healthcare, it facilitates the safe implantation of cells, holding them isolated from the immune system and allowing long term production of therapeutic molecules in patients. (advfn.com)
  • The drug has a remarkably low toxicity with a very large therapeutic window. (globenewswire.com)
  • A drug, including a bulk drug substance, which has not been used in India to a significant extent and whose safety, efficacy and therapeutic value has not been established in India. (mondaq.com)
  • The MYASTERIX project will advance a therapeutic vaccine candidate (designated orphan drug) indicated for the autoimmune disease myasthenia gravis (MG) to clinical proof of concept studies. (europa.eu)
  • Can Drug Prices Be Brought Down to Earth in 2019? (ptcommunity.com)
  • The global orphan drug market totaled nearly $123 billion in 2014 and will continue to grow to reach nearly $191 billion by 2019. (smi-online.co.uk)
  • Effective January 3, 2019, manufacturers, importers and marketers of new drugs patented in India are exempt from price control for a period of five years from the date of commencement of their commercial marketing. (mondaq.com)
  • From January 03, 2019 onwards, the Indian Government has exempted manufacturers, importers and marketers (" Manufacturers ") of patented new drugs in India from price control for a period of five years ( "New Drug Exemption" ). (mondaq.com)
  • How will the market shares for biological drug API manufacturing services submarkets develop from 2019 to 2029? (visiongain.com)
  • This led to the acknowledgment of Plasminogen eye drops as the first-ever drug developed to treat the rare condition and allowed Kedrion to continue its clinical trials. (europa.eu)
  • Accounting to the massive potential held by orphan drugs, there are numerous clinical trials associated with the drugs. (webnewswire.com)
  • We are now working with the BSF to scale up drug manufacturing in preparation for the upcoming Phase II clinical trials," he concluded. (globenewswire.com)
  • Since Cerbios' core business is cGMP production for clinical trials and commercial supply, we have intensively worked in order to set up a state-of-the-art cGMP production unit on the basis of the long-term experience Cerbios has in biotechnology and fermentation. (cerbios.ch)
  • The FDA has cleared an Investigational New Drug application for these programs, and Phase 1/2 clinical trials evaluating SB-318 and SB-913 in adults with MPS I and MPS II, respectively, are open and screening subjects for enrollment. (pharmabiz.com)
  • There are currently no drugs approved in the U.S. to treat AML in patients who are unfit for intensive induction chemotherapy, though hypomethylating agents are recommended by the National Comprehensive Cancer Network (NCCN) guidelines. (yahoo.com)
  • Patients, Pricing and Profits: Is the orphan drug business model fair and sustainable? (terrapinn.com)
  • Previous studies have found a decrease in behavioural and psychiatric symptoms of dementia when patients are treated for pain, but this new study now shows, for the first time, a decrease in symptomatic drug use. (europeanpharmaceuticalreview.com)
  • It is one of the most expensive drugs sold, with an annual cost to U.S. patients of $200,000. (wikipedia.org)
  • Part B is a randomized (3:1 drug:placebo), single-blind, placebo-controlled study in patients with PH1. (businesswire.com)
  • Patients with relatively preserved renal function were enrolled to allow for measurement of urinary oxalate excretion as a marker for hepatic oxalate production. (businesswire.com)
  • This drug was approved for use in patients with endogenous Cushing's syndrome who have type 2 diabetes or glucose intolerance and are not candidates for surgery or who have not responded to prior surgery. (healthcanal.com)
  • The company has voluntarily proposed distributing Korlym through a central pharmacy to ensure the timely, convenient and appropriate delivery of the drug to Cushing's patients or to the health care institutions where this therapy may be initiated. (healthcanal.com)
  • Most retail pharmacies are unlikely to keep adequate supplies of the drug for this rare condition and central distribution will give patients with Cushing's syndrome better access to Korlym. (healthcanal.com)
  • HOBOKEN, N.J. , Dec. 1, 2010 /PRNewswire/ -- The U.S. Food and Drug Administration (FDA) has approved Octapharma USA 's Investigational New Drug Application for octaplex® (Human Prothrombin Complex, Freeze Dried) as a Fast Track Product for 'Reversal of Anticoagulation Therapy in Patients under Vitamin K Antagonist Therapy with the Need for Urgent Surgery or Invasive Procedures. (prnewswire.com)
  • Consistent with the prior clinical study of fostamatinib in ITP, this FIT Phase 3 study demonstrated that fostamatinib provided a robust and enduring benefit for those patients who responded to the drug candidate. (drugs.com)
  • This would be useful for so-called "orphan drugs" -- drugs needed by a small number of patients. (brightsurf.com)
  • These drugs are referred to be "orphan" as under normal conditions because these drugs are not cost effective to be developed by the pharmaceuticals industry, intended for a small number of patients suffering from rare conditions. (marketreportsonline.com)
  • In some cases, Canadian patients can access orphan drugs not approved in Canada through Health Canada's Special Access Programme, but may have to pay for them out-of-pocket. (nationalpost.com)
  • Growing demand for customized devices combined with an expansion of technological innovation drives the major progress in personalized medicine expressed e.g. by the production of small series of individually-selected doses and tailor-made prostheses meet the anatomical needs of patients. (drugpatentwatch.com)
  • In the first human trial, an experimental antisense drug (Ionis-HTTRx, Ionis Pharmaceuticals) successfully lowered the level of mutant huntingtin protein (mHTT) in spinal fluid of patients with Huntington's disease, researchers reported today. (medscape.com)
  • A drug which the FDA approved more than half a century ago-which doctors have been prescribing for their patients with high-risk pregnancies through compounding pharmacies with great success-was designated by the FDA an "orphan drug. (anh-usa.org)
  • When the drug was no longer available, compounding pharmacies were able to compound 17P's ingredients and sell the product to patients whose physicians had prescribed it. (anh-usa.org)
  • The frequency of communication is designed to ensure that questions and issues are resolved quickly, potentially leading to earlier drug approval and access by patients. (pharmabiz.com)
  • The Ministry of Chemicals and Fertilizers (Department of Pharmaceuticals) has notified an order amending the Drugs (Prices Control) Order 2013. (mondaq.com)
  • The dose-dependent reductions of mHTT we observed in the study substantially exceeded our expectations and we were equally encouraged by the safety profile of the drug," C. Frank Bennett, PhD, senior vice president of research at Ionis Pharmaceuticals said in the release. (medscape.com)
  • However, there are no clot-busting drugs currently approved for this indication, and the treatments currently used (plasminogen activators) typically require a prolonged infusion averaging 24 to 36 hours and produce increased risk of bleeding complications. (salesandmarketingnetwork.com)
  • A drug which is already approved which is now proposed to be marketed with modified or new claims such as indication, dosage, dosage forms or route of administration. (mondaq.com)
  • In addition to today's recommendation, the FDA recently granted eltrombopag orphan drug designation for this indication. (bio-medicine.org)
  • A project has successfully examined the commercial viability of the use of marine derived peptones in the production of high value products. (scientistlive.com)
  • The following law established for accurately labeling of drug products? (studystack.com)
  • The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. (healthcanal.com)
  • federal food and drug administration, concerned with general safety standards in the production of drugs, foods, cosmetics.responsible for approval or removal of products on the market. (studystack.com)
  • From orphan to biosimilar to novel blockbuster drugs, the production demands can range from grams to metric tons of biopharmaceutical products each year. (bioprocessintl.com)
  • Pharmacists can legally compound FDA-approved products when a drug is not commercially available (as was the case when the manufacturer pulled it from the market), or when a prescriber determines that the compounded preparation is more clinically appropriate for an individual patient (as when someone is allergic to one particular element in the preparation but another can easily be substituted). (anh-usa.org)
  • ResearchMoz.us includes new market research report 'Global Orphan drug industry 2015 Market Research Reports' to its huge collection of research reports. (sbwire.com)
  • The presence of numerous large scale orphan drug manufacturers across the world will emerge in favor of growth of the overall market in the coming years. (webnewswire.com)
  • Post market study of a drug with volunteer basis, after first three phases are review. (studystack.com)
  • Weighted average selling price has been considered to estimate the market size of various drugs mentioned in the scope of the study. (researchmoz.us)
  • Serdaxin is a game-changing mood disorder drug in a huge market filled with major players (Pfizer, Eli-Lilly, Glaxo). (biomedreports.com)
  • In addition, growing awareness of the several side effects associated with the chemical drugs is expected to boost the overall biotechnology based chemicals market. (openpr.com)
  • EXPLORE the future landscape orphan drugs following the market access and reimbursement challenges providers are currently facing. (smi-online.co.uk)
  • Increasing sales of prescription drugs, substantial benefits for new entrants in rare disease drug market, increased spending on medicines, rising healthcare expenditure and improving economic conditions of nations are some of the significant factors driving growth of the Orphan drugs market. (marketreportsonline.com)
  • They are trying to leverage drugs that they can sell into relatively small groups of physician specialists (like gastroenterologists, for example), not big general drugs that they would have to market with TV ads. (stockgumshoe.com)
  • The biological drug API manufacturing market is estimated to grow at a CAGR of 9.5% in the first half of the forecast period. (visiongain.com)
  • SWOT and STEP Analysis of the biological drug API manufacturing market. (visiongain.com)
  • How is the biological drug API manufacturing services market evolving? (visiongain.com)
  • What is driving and restraining the biological drug API manufacturing services market? (visiongain.com)
  • Visiongain's study is intended for anyone requiring commercial analyses for the biological drug API manufacturing market. (visiongain.com)
  • They are essentially claiming there is no difference between compounding and drug manufacturing-an underhanded attempt to remove from the market compounded drugs that are exempt from the FDA drug approval process. (anh-usa.org)
  • HOBOKEN, N.J. (November 4, 2011) - The U.S. Food and Drug Administration (FDA) yesterday cleared the way for the U.S. market return of octagam® [Immune Globulin Intravenous (human)] 5% Liquid Preparation], the Octapharma USA product used to treat disorders of the immune system. (fiercepharma.com)
  • It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. (businesswire.com)
  • Kineret (anakinra) is a recombinant protein drug which was originally discovered and developed by Amgen. (drugdevelopment-technology.com)
  • ISIS-SMN Rx is designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein. (biospace.com)
  • QR-110 is designed to restore wild-type CEP290 mRNA leading to the production of wild-type CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. (globenewswire.com)
  • Inotersen is an antisense drug designed to reduce the production of transthyretin, or TTR protein, to treat ATTR amyloidosis, a systemic, progressive and fatal disease. (pharmiweb.com)
  • SMA is caused by the lack of or inadequate production of survival motor neuron, a protein created by the SMN1 gene. (fool.com)
  • Nusinersen is an antisense oligonucleotide that increases SMN protein production by improving the activity of SMN2, a gene that's nearly identical to SMN1. (fool.com)
  • For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated," Sarah Tabrizi, MD, PhD, director of the University College London Huntington's Disease Centre and global chief investigator of the phase 1/2a study of the drug, said in a statement issued by the university. (medscape.com)
  • Ionis-HTTRx is designed to reduce the production of all forms of the huntingtin (HTT) protein, which in its mutated variant is responsible for Huntington's disease. (medscape.com)
  • Antagonistic antibody performed synergy effects in combination with other immuno-oncology drug. (ntbinfo.no)
  • Our research indicates that the hospital pharmacies distribution channel segment is expected to witness a significant rise in revenue share over the forecast period, owing to the increasing direct procurement of mesothelioma drugs by governments to ensure availability in hospital pharmacies and oncology centers. (researchmoz.us)
  • Indirect-acting thrombolytics, such as Plasminogen activators, target clots indirectly by first stimulating the production of Plasmin. (ibtimes.com)
  • Using this apparatus, the researchers can manufacture about 1,000 doses of a given drug in 24 hours. (brightsurf.com)
  • After the move of the actual R&D laboratories to their new building the space left empty has already been conceived and set up for a second production line (up-to 1,000 Litre fermentor) with either stainless steel or disposable fermenters. (cerbios.ch)
  • What phase of studies of a drug on a small group of healthy volunteers, to determine safe dosages. (studystack.com)
  • During what phase a drug is approved for selling. (studystack.com)
  • Determine drug dosage and pharmacokintinetics are done during this phase. (studystack.com)
  • During this phase drugs are determine for effectineness and side effects on a small group of people. (studystack.com)
  • What name is given to a drug during the investigation phase? (studystack.com)
  • Further, the drug has demonstrated clinical benefit and positive impact on inflammatory and immunological markers in Phase 2 studies in diffuse cutaneous systemic sclerosis, dermatomyositis and cystic fibrosis. (globenewswire.com)
  • The plant has been confirmed to be suitable for commercial production in the future, subject to successful installation and qualification of additional equipment following the next expansion phase. (cerbios.ch)
  • These first results are extremely encouraging and mean we can plan a phase 3 trial to better evaluate the effectiveness of the drug. (hug-ge.ch)
  • The fact that levels of mutant huntingtin were reduced in correlation to the dose of Ionis-HTTRx that was given is significant, and the fact that participants in this first Phase 1/2a study are able to continue on the drug through open label extension gives us optimism regarding its safety," HDSA President and CEO Louise Vetter said in a statement. (medscape.com)
  • The project is divided into 6 main Work Packages, WP1 management, WP2 production, WP3 toxicity study, WP4 phase 1B clinical trial, WP5, phase 2B clinical trial, WP6 exploitation and dissemination. (europa.eu)
  • Emerson Resources provides pre-formulation through phase III and small commercial production. (i2e.org)
  • They have been producing phase I and II clinical supplies since 2005 and will be adding phase III and small volume and orphan drug commercial manufacturing in 2013. (i2e.org)
  • In a 17-page draft guidance released last week, the FDA says it wants to "establish an infrastructure" designed to help sponsors use cohorts to speed drug approvals without compromising safety. (centerwatch.com)
  • While awaiting official approvals, the companies are going to launch expanded access programs in various markets to help get the drug in patient hands sooner. (fool.com)
  • SB-318 and SB-913 have already received Orphan Drug and Rare Pediatric Disease designations from the FDA. (pharmabiz.com)
  • Dr. Thomas F. Miller, Discovery Labs' Chief Operating Officer commented, "To date, Discovery Labs has successfully procured orphan designations for several respiratory disease targets in both the U.S. and Europe. (nbcnews.com)
  • In addition, one of CytRx's drug candidates, arimoclomol, was sold to Orphazyme A/S in exchange for milestone payments and royalties. (pharmiweb.com)
  • Telotristat etiprate is a member of a new class of oral drugs invented by Lexicon, the serotonin synthesis inhibitors, which are being developed in a spectrum of gastrointestinal indications. (bio-medicine.org)
  • next generation bispecific antibody neutralizing both VEGF-KDR and Notch-DLL4 pathways overcomes anti-cancer drug resistant tumor growth. (ntbinfo.no)
  • Basolateral but not apical delivery of the epidermal growth factor receptor ligand transforming growth factor α results in up-regulation of COX-2 and production of PGs that are released exclusively into the basolateral medium of polarized HCA-7 cells. (aacrjournals.org)
  • LINCL, which qualifies as a rare pediatric disease under Section 529 of the Food, Drug, and Cosmetic Act, is an autosomal recessive neurodegenerative disorder with life expectancy ranging from six to the early teenage years. (prnewswire.com)
  • One of the advantages of this small-scale system is that it could be used to make small amounts of drugs that would be prohibitively expensive to make in a large-scale plant. (brightsurf.com)