Orphan drug production. Medical search

Production of drugs or biologicals which are unlikely to be manufactured by private industry unless special incentives are provided by others.

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.

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.

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))

Laws concerned with manufacturing, dispensing, and marketing of drugs.

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.

The branch of medicine concerned with diseases, mainly of parasitic origin, common in tropical and subtropical regions.

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)

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.

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.

Exclusive legal rights or privileges applied to inventions, plants, etc.

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)

An "Orphan Drug" is a pharmaceutical agent that is developed to treat a rare medical condition, disorder, or disease that affects a small number of people in comparison to other conditions. In the United States, this is defined as a condition or disease that affects fewer than 200,000 people nationwide. Due to the limited market for these drugs, pharmaceutical companies are often reluctant to invest in their development and production.

"Orphan Drug Production," therefore, refers to the manufacturing process of these rare disease treatments. To encourage the development and production of orphan drugs, governments and regulatory agencies offer incentives such as tax credits, grants, and exclusive marketing rights for a certain period of time. These measures help offset the higher costs and lower profit margins associated with developing and producing orphan drugs, ultimately benefiting patients with rare diseases who often have few or no treatment options available to them.

A rare disease, also known as an orphan disease, is a health condition that affects fewer than 200,000 people in the United States or fewer than 1 in 2,000 people in Europe. There are over 7,000 rare diseases identified, and many of them are severe, chronic, and often life-threatening. The causes of rare diseases can be genetic, infectious, environmental, or degenerative. Due to their rarity, research on rare diseases is often underfunded, and treatments may not be available or well-studied. Additionally, the diagnosis of rare diseases can be challenging due to a lack of awareness and understanding among healthcare professionals.

"Drug approval" is the process by which a regulatory agency, such as the US Food and Drug Administration (FDA), grants formal authorization for a pharmaceutical company to market and sell a drug for a specific medical condition. The approval process is based on rigorous evaluation of clinical trial data to ensure that the drug is safe and effective for its intended use.

The FDA's approval process typically involves several stages, including preclinical testing in the lab and animal studies, followed by three phases of clinical trials in human subjects. The first phase tests the safety of the drug in a small group of healthy volunteers, while the second and third phases test the drug's efficacy and side effects in larger groups of patients with the medical condition for which the drug is intended.

If the results of these studies demonstrate that the drug is safe and effective, the pharmaceutical company can submit a New Drug Application (NDA) or Biologics License Application (BLA) to the FDA for review. The application includes data from the clinical trials, as well as information about the manufacturing process, labeling, and proposed use of the drug.

The FDA reviews the application and may seek input from independent experts before making a decision on whether to approve the drug. If approved, the drug can be marketed and sold to patients with the medical condition for which it was approved. The FDA continues to monitor the safety and efficacy of approved drugs after they reach the market to ensure that they remain safe and effective for their intended use.

Pharmaceutical economics is a branch of economics that focuses on the production and distribution of pharmaceutical products and services. It involves the analysis of various factors that influence the development, pricing, and accessibility of medications, including issues related to healthcare policy, regulation, reimbursement, and market competition.

Pharmaceutical economists study topics such as:

1. The research and development (R&D) process for new drugs, including the costs, risks, and uncertainties associated with bringing a new drug to market.
2. The pricing of pharmaceuticals, taking into account factors such as production costs, R&D expenses, market competition, and the value that medications provide to patients and society.
3. The impact of government regulations and policies on the pharmaceutical industry, including issues related to intellectual property protection, drug safety, and efficacy testing.
4. The role of health insurance and other third-party payers in shaping the demand for and access to pharmaceuticals.
5. The evaluation of pharmaceutical interventions' cost-effectiveness and their impact on healthcare outcomes and patient well-being.
6. The analysis of market structures, competitive dynamics, and strategic decision-making within the pharmaceutical industry.
7. The assessment of globalization, international trade, and cross-border collaboration in the pharmaceutical sector.

Pharmaceutical economics plays a crucial role in informing healthcare policy decisions, improving patient access to essential medications, and promoting sustainable and innovative practices within the pharmaceutical industry.

'Drug legislation' refers to the laws and regulations that govern the production, distribution, sale, possession, and use of medications and pharmaceutical products within a given jurisdiction. These laws are designed to protect public health and safety by establishing standards for drug quality, ensuring appropriate prescribing and dispensing practices, preventing drug abuse and diversion, and promoting access to necessary medications. Drug legislation may also include provisions related to clinical trials, advertising, packaging, labeling, and reimbursement. Compliance with these regulations is typically enforced through a combination of government agencies, professional organizations, and legal penalties for non-compliance.

The United States Food and Drug Administration (FDA) is a federal government agency responsible for protecting public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, our country's food supply, cosmetics, and products that emit radiation. The FDA also provides guidance on the proper use of these products, and enforces laws and regulations related to them. It is part of the Department of Health and Human Services (HHS).

Tropical medicine is a branch of medicine that deals with health problems that are prevalent in or unique to tropical and subtropical regions. These regions are typically characterized by hot and humid climates, and often have distinct ecological systems that can contribute to the spread of infectious diseases.

The field of tropical medicine encompasses a wide range of health issues, including:

1. Infectious diseases: Many tropical diseases are caused by infectious agents such as bacteria, viruses, parasites, and fungi. Some of the most common infectious diseases in the tropics include malaria, dengue fever, yellow fever, chikungunya, Zika virus, leishmaniasis, schistosomiasis, and Chagas disease.
2. Neglected tropical diseases (NTDs): A group of chronic infectious diseases that primarily affect poor and marginalized populations in the tropics. NTDs include diseases such as human African trypanosomiasis (sleeping sickness), leprosy, Buruli ulcer, and dracunculiasis (guinea worm disease).
3. Zoonotic diseases: Diseases that are transmitted between animals and humans, often through insect vectors or contaminated food and water. Examples of zoonotic diseases in the tropics include rabies, leptospirosis, and Rift Valley fever.
4. Environmental health issues: The tropical environment can pose unique health challenges, such as exposure to toxic chemicals, heat stress, and poor air quality. Tropical medicine also addresses these environmental health issues.
5. Travel medicine: As global travel increases, there is a growing need for medical professionals who are knowledgeable about the health risks associated with traveling to tropical destinations. Tropical medicine physicians often provide pre-travel consultations and post-travel evaluations for international travelers.

Overall, tropical medicine is an essential field that addresses the unique health challenges faced by populations living in or traveling to tropical and subtropical regions.

Pharmacy ethics refers to the principles that guide the behavior and decision-making of pharmacists and other healthcare professionals involved in the provision of medications and related services. These principles are based on values such as respect for autonomy, beneficence (doing good), non-maleficence (doing no harm), justice, and fidelity (faithfulness to commitments).

Pharmacy ethics encompass a wide range of issues, including:

* Informed consent: ensuring that patients understand the risks and benefits of medications and make voluntary decisions about their use.
* Confidentiality: protecting patient information and respecting privacy.
* Professional competence: maintaining knowledge and skills to provide safe and effective care.
* Conflict of interest: avoiding situations where personal or professional interests could compromise patient care.
* Allocation of resources: making fair and equitable decisions about the distribution of medications and related services.
* End-of-life care: respecting patients' wishes and providing appropriate care at the end of life.

Pharmacy ethics are guided by professional codes of conduct, legal regulations, and ethical theories and principles. Pharmacists and other healthcare professionals are expected to uphold these standards and engage in ongoing reflection and education to promote ethical practice.

The "drug industry" is also commonly referred to as the "pharmaceutical industry." It is a segment of the healthcare sector that involves the research, development, production, and marketing of medications or drugs. This includes both prescription and over-the-counter medicines used to treat, cure, or prevent diseases and medical conditions in humans and animals.

The drug industry comprises various types of organizations, such as:

1. Research-based pharmaceutical companies: These are large corporations that focus on the research and development (R&D) of new drugs, clinical trials, obtaining regulatory approvals, manufacturing, and marketing their products globally. Examples include Pfizer, Johnson & Johnson, Roche, and Merck.

2. Generic drug manufacturers: After the patent for a brand-name drug expires, generic drug manufacturers can produce and sell a similar version of the drug at a lower cost. These companies must demonstrate that their product is bioequivalent to the brand-name drug in terms of safety, quality, and efficacy.

3. Biotechnology companies: These firms specialize in developing drugs using biotechnological methods, such as recombinant DNA technology, gene therapy, or monoclonal antibodies. Many biotech companies focus on specific therapeutic areas, like oncology, immunology, or neurology.

4. Contract research organizations (CROs): CROs provide various services to the drug industry, including clinical trial management, data analysis, regulatory affairs support, and pharmacovigilance. They work with both large pharmaceutical companies and smaller biotech firms to help streamline the drug development process.

5. Drug delivery system companies: These organizations focus on developing innovative technologies for delivering drugs more effectively and safely to patients. Examples include transdermal patches, inhalers, or long-acting injectables.

6. Wholesalers and distributors: Companies that purchase drugs from manufacturers and distribute them to pharmacies, hospitals, and other healthcare providers.

The drug industry plays a crucial role in improving public health by discovering, developing, and delivering new treatments for various diseases and medical conditions. However, it is also subject to criticism and regulation due to concerns about high drug prices, marketing practices, and the potential for conflicts of interest between industry and healthcare professionals.

Off-label use refers to the practice of prescribing or using pharmaceutical drugs for purposes, dosages, patient populations, or routes of administration that are not included in the approved labeling of the drug by the regulatory authority, such as the U.S. Food and Drug Administration (FDA). It is not illegal or unethical for physicians to prescribe medications off-label when they judge that it is medically appropriate for their patients. However, manufacturers are prohibited from promoting their drugs for off-label uses.

A patent, in the context of medicine and healthcare, generally refers to a government-granted exclusive right for an inventor to manufacture, use, or sell their invention for a certain period of time, typically 20 years from the filing date. In the medical field, patents may cover a wide range of inventions, including new drugs, medical devices, diagnostic methods, and even genetic sequences.

The purpose of patents is to provide incentives for innovation by allowing inventors to profit from their inventions. However, patents can also have significant implications for access to medical technologies and healthcare costs. For example, a patent on a life-saving drug may give the patent holder the exclusive right to manufacture and sell the drug, potentially limiting access and driving up prices.

It's worth noting that the patent system is complex and varies from country to country. In some cases, there may be ways to challenge or circumvent patents in order to increase access to medical technologies, such as through compulsory licensing or generic substitution.

... 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 ...

https://en.wikipedia.org/wiki/Orphan-Nor

It was awarded orphan drug status. DCVax-Direct is a therapy to treat inoperable solid tumors in Phase 1 trials in the US. ... "Northwest Biotherapeutics Gets Ready to Scale-Up Production". News: Bioprocessing. Gen. Eng. Biotechnol. News (paper). Vol. 34 ... "Study of a Drug [DCVax®-L] to Treat Newly Diagnosed GBM Brain Cancer - Full Text View - ClinicalTrials.gov". clinicaltrials.gov ...

https://en.wikipedia.org/wiki/Northwest_Biotherapeutics

"Orphan drugs in glioblastoma multiforme: a review". Orphan Drugs: Research and Reviews. Retrieved 2020-07-02. "Gliovac (ERC1671 ... It creates a tissue bank for proliferative tumors and a Good Manufacturing Practice-compliant production facility in Schaijk, ... Gliovac obtains Orphan Drug status from the US FDA. Glioblastoma multiforme is the most common and deadly brain tumor in adults ... Orphan Drug status, and initiating Phase II trials. Stathopoulos conducts a proof-of-concept study on rats. The study finds ...

https://en.wikipedia.org/wiki/Epitopoietic_Research_Corporation

... 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: 40 Number of orphan drugs in phase 2 trial: 231 Number of orphan drugs in U.S. ... The 2014 Orphan Drug report stated that the percentage of orphan drug sales as part of all prescription drug sales had been ...

https://en.wikipedia.org/wiki/Orphan_drug

... was granted Orphan Drug status by the Food and Drug Administration. Also in May 2019, the company began recruiting patients for ... "Mustang Bio Announces License Agreement with CSL Behring for the Cytegrity™ Stable Producer Cell Line for the Production of MB- ... "Mustang Bio's brain cancer-targeting gene therapy granted FDA Orphan Drug designation". www.beckershospitalreview.com. ...

https://en.wikipedia.org/wiki/Mustang_Bio

... 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 ... "FDA Office of Orphan Products Development". U.S. Food and Drug Administration. 20 October 2020. 99% Invisible: Orphan Drugs, ( ...

https://en.wikipedia.org/wiki/Orphan_Drug_Act_of_1983

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. Office of the Commissioner. " ... Both the FDA and European Medicines Agency has given ezutromid an orphan drug designation. ...

https://en.wikipedia.org/wiki/Ezutromid

... 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 ... Drug interactions between imiglucerase and miglustat. Drugs.com, accessed 2019-04-11. (CS1 German-language sources (de), Drugs ... Drugs acting on the gastrointestinal system and metabolism, Orphan drugs, Recombinant proteins, Sanofi). ...

https://en.wikipedia.org/wiki/Imiglucerase

"Inotersen Orphan Drug Designation and Approval". U.S. Food and Drug Administration (FDA). 24 July 2012. Archived from the ... which allows it to interfere with and block the production of trasnthyretin. As such, it was the first FDA-approved siRNA ... "FDA approves new orphan drug Kynamro to treat inherited cholesterol disorder". Fierce Biotech. 29 January 2013. Retrieved 7 ... The application for inotersen was granted orphan drug designation. It was developed by Ionis Pharmaceuticals and licensed to ...

https://en.wikipedia.org/wiki/Antisense_therapy

... as well as the drugs amikacin and clarithromycin. However, in the United States, clofazimine is considered an orphan drug, is ... The drug is currently no longer commercially available in the United States as Novartis has discontinued production of ... Antileprotic drugs, Orphan drugs, World Health Organization essential medicines, Phenazines, Chloroarenes, Irish inventions, ... From AMA Drug Evaluations Annual, 1993, p1619). The drug is given as an alternative to people who can not tolerate the effects ...

https://en.wikipedia.org/wiki/Clofazimine

Experimental cancer drugs, Chromones, Protein kinase inhibitors, Orphan drugs, All stub articles, Antineoplastic and ... Treatment of cells with alvocidib leads to inhibition of P-TEFb and the loss of mRNA production. The compound is a synthetic ... "FDA grants orphan drug status to Alvocidib for AML". v t e (Articles without InChI source, Articles with changed CASNo ... The FDA has granted orphan drug designation to alvocidib for the treatment of patients with acute myeloid leukemia. Sekine C, ...

https://en.wikipedia.org/wiki/Alvocidib

The company pursued but did not secure orphan drug status via the United States Food and Drug Administration. Telesta's ... In response, the company started cutting staff, and attempted to find a buyer for its Belleville and Montreal production ... The drug MCNA had a 100% - 250% higher efficacy in the pivotal clinical trial than Valstar (. ... the Biologics License Application of Telestra's main drug, MCNA, was rejected by the FDA. ...

https://en.wikipedia.org/wiki/Telesta_Therapeutics

"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 ...

https://en.wikipedia.org/wiki/Respiratory_tract_antimicrobial_defense_system

... and it is considered an orphan drug; the Argentine government committed itself to manufacture and sponsor Candid #1 vaccine. ... Details of the vaccine were published in 2011, and a protocol for production of the vaccine was published in 2018. Demand for ... Ambrosio AM, Mariani MA, Maiza AS, Gamboa GS, Fossa SE, Bottale AJ (2018). "Protocol for the Production of a Vaccine Against ... On 29 August 2006 the Maiztegui Institute obtained certification for the production of the vaccine in Argentina. The vaccine ...

https://en.wikipedia.org/wiki/Argentine_hemorrhagic_fever

"Production Underway on Final Season of Space Original Series ORPHAN BLACK with Emmy® Winner Tatiana Maslany" (Press release). ... Kevin Hanchard as Art Bell, a detective and Beth's police partner Michael Mando as Vic Schmidt, Sarah's abusive, drug-dealing ... Wikiquote has quotations related to Orphan Black. Orphan Black at CTV Orphan Black at BBC America Orphan Black at IMDb Portals ... "Additional Casting For SPACE Original Series ORPHAN BLACK Announced as Production Begins in Toronto" (Press release). Bell ...

https://en.wikipedia.org/wiki/Orphan_Black

BTG subsequently won approvals for orphan drug status by the Food and Drug Administration for treating alcoholic hepatitis, ... However, in part due to bad publicity from its illicit use by bodybuilders, production of Anavar was discontinued by Searle ... 2935-. ISBN 978-0-8155-1856-3. "Drugs@FDA: FDA Approved Drug Products". United States Food and Drug Administration. Retrieved ... Drugs of Abuse (PDF). United States Drug Enforcement Administration. 2011. p. 22. Levounis P, Zerbo E, Aggarwal R (3 May 2016 ...

https://en.wikipedia.org/wiki/Oxandrolone

90Y-DOTATOC (international nonproprietary name: yttrium (90Y) edotreotide) and 177Lu-DOTATOC are designated as orphan drugs, ... The cost of small volume production of the relevant radionuclides is high. The cost of Lutathera, a commercial 177Lu-DOTATATE ... Office of the Commissioner (26 January 2018). "FDA approves new treatment for certain digestive tract cancers". Food and Drug ... Dash, Ashutosh; Pillai, Maroor Raghavan Ambikalmajan; Knapp, Furn F. (17 February 2015). "Production of 177Lu for Targeted ...

https://en.wikipedia.org/wiki/Peptide_receptor_radionuclide_therapy

... and the first drug approved by the FDA to treat this condition. It is a gene silencing drug that interferes with the production ... Patisiran was granted orphan drug status, fast track designation, priority review and breakthrough therapy designation due to ... New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Archived from the ... "Drug Approval Package: Onpattro (patisiran)". U.S. Food and Drug Administration (FDA). 7 September 2018. Archived from the ...

https://en.wikipedia.org/wiki/Patisiran

... and orphan drug designations by the Food and Drug Administration. The phase 3 HELP study evaluated efficacy and safety of ... The proteolysis of the kininogen forces an upscaled production of bradykinin and kininogen within the patient. Increased ... Drugs that are a monoclonal antibody, Breakthrough therapy, Monoclonal antibodies, Orphan drugs). ... "Drug Trials Snapshots: Takhzyro". U.S. Food and Drug Administration (FDA). 9 February 2019. Archived from the original on 30 ...

https://en.wikipedia.org/wiki/Lanadelumab

"Alicaforsen US Orphan designation". Orphanet. Retrieved 28 April 2017. "EU/3/09/641 Orphan drug designation". European ... Alicaforsen (trade name Camligo) is an antisense oligonucleotide therapeutic that targets the messenger RNA for the production ... Drugs missing an ATC code, Drugs with no legal status, Articles containing unverified chemical infoboxes, Drugs acting on the ... The use of the enema formulation of alicaforsen to treat pouchitis was granted orphan drug status in the US in 2008 and ...

https://en.wikipedia.org/wiki/Alicaforsen

"The top 50 thought-leaders in orphan drugs and rare disease" (PDF), World Orphan Drug Congress USA, 2013, retrieved July 20, ... research and development for other drugs and to allow them to fund programs that distribute a small portion of production for ... The drugs are also protected by orphan drug acts in various countries which provides extensive protection from competition and ... of the top fifty leaders of thought in orphan drugs and rare diseases in a list published by Terrapin for the World Orphan Drug ...

https://en.wikipedia.org/wiki/Henri_Termeer

... 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 - food ...

https://en.wikipedia.org/wiki/List_of_MeSH_codes_(J01)

... "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 ... attention due their action to increase the price of an orphan drug to such extent it became impossible to pay for the drug. In ...

https://en.wikipedia.org/wiki/Leadiant_Biosciences

In 1991 the FDA approved the first version of Genzyme's orphan drug alglucerase, the only treatment for Gaucher's disease.: 123 ... 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% ...

https://en.wikipedia.org/wiki/Specialty_pharmacy

It is co-marketed by Merck Serono and Pfizer in the US under an exception to the Orphan Drug Act.[citation needed] It was ... 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 on the top ten 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 ...

https://en.wikipedia.org/wiki/Interferon_beta-1a

To deliver the drug directly to the liver, siRNA is encased in a lipid nanoparticle. The siRNA molecule halts the production of ... It was also awarded Orphan Drug Designations given to those treatments that are intended to safely treat conditions affecting ... Patisiran, an investigational RNAi therapeutic drug, uses this process to decrease the production of mutant and wild-type ... "New Kind of Drug, Silencing Genes, Gets FDA Approval". The Wall Street Journal. 10 August 2018. Retrieved 26 March 2021. ...

https://en.wikipedia.org/wiki/Small_interfering_RNA

"Orphan designation, Europe". Orphanet, the portal for rare diseases and orphan drugs. 2017-10-05. "Treatment of glioblastoma ... In MDS, CD95L-signaling is a negative regulator of erythrocyte production in the bone marrow and its blockade has been shown to ... Drugs with non-standard legal status, Infobox drug articles without a structure image, Articles without EBI source, Chemical ... Asunercept has been granted orphan drug status for the treatment of GBM and MDS in the EU and the US. It has also received ...

https://en.wikipedia.org/wiki/Asunercept

Many of the roles are played in the classical tradition by men in drag. 1993 Psycho Beach Party, Camille 1998 How Mrs. Grinchly ... It is a group of actors, musicians, dancers, writers and visual artists dedicated to the production of shows that are often ... The Gold Dust Orphans are a fringe theater company based in Boston and Provincetown, Massachusetts. It was founded in 1995 by ... Grinchley's Christmas Carol Won for Outstanding Production (Fringe Theater) - Mrs. Grinchley's Christmas Carol 2012 Elliot ...

https://en.wikipedia.org/wiki/The_Gold_Dust_Orphans

... was given orphan drug status in May 2019 and March 2020. During the 2018 Équateur province Ebola outbreak, ansuvimab ... The heavy and light chain sequences of ansuvimab mAb were cloned into CHO cell lines to enable large-scale production of ... "Ansuvimab Orphan Drug Designations and Approvals". accessdata.fda.gov. 8 May 2019. Retrieved 24 December 2020. "Ansuvimab ... "Ridgeback Biotherapeutics LP Announces Orphan Drug Designation for mAb114" (Press release). Ridgeback Biotherapeutics LP. ...

https://en.wikipedia.org/wiki/Ansuvimab

"Casimersen Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 4 June 2019. Retrieved 25 February ... This change causes the production of an internally truncated dysphotrophin protein. Casimersen was evaluated in a double-blind ... Food and Drug Administration (FDA) granted the application for casimersen fast track, priority review, and orphan drug ... "Drug Approval Package: Amondys 45". U.S. Food and Drug Administration (FDA). 18 March 2021. Retrieved 13 September 2021. " ...

https://en.wikipedia.org/wiki/Casimersen

... J Gen Intern Med. 2019 Mar;34(3): ... Orphan Drug Production / economics* * United States / epidemiology * Young Adult ...

https://pubmed.ncbi.nlm.nih.gov/30324293/

The portal for rare diseases and orphan drugs ... Orphan drugs *Search. *Expert centres and Networks *Expert ... The portal for rare diseases and orphan drugs. COVID-19 & Rare diseases. Rare Diseases Resources for Refugees/Displaced Persons ... Type of production : Biotechnology. *Orphan designation : *. Orphan designation - Europe. *Diseases list. *Myelodysplastic ...

https://www.orpha.net/consor/cgi-bin/Drugs_Search.php?lng=EN&data_id=4359&Substance=Magrolimab&search=Drugs_Search_SubstanceTradename&data_type=Product&diseaseType=Drug&Typ=Sub&title=&diseaseGroup=

Orphan Drug & Rare Disease Development: Understanding the European and US Regulatory Landscape. Life Sciences, Clinical Trials ... Carol has about 20 years of drug development experience gained in small, medium and large pharma companies working in Germany, ... In addition to industry appointments, he was previously an adjunct professor in the Drug Regulatory Program at LIU. ... also known as orphan medicinal products. Our speakers will present an overview of current guidance documents, possible ...

https://xtalks.com/webinars/orphan-drug-and-rare-disease-regulation/

Invested about $60 million to build two new vaccine production facilities 07.23.19. ... La Jolla Pharma Receives Orphan Designation for Malaria Treatment LJPC-0118, artesunate, demonstrated to be superior to quinine ...

https://www.contractpharma.com/contents/view_breaking-news/2019-07-30/mustang-bios-aml-treatment-receives-orphan-drug-designation/

Orphan-Nor 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 ...

https://en.wikipedia.org/wiki/Orphan-Nor

The World Orphan Drug Congress USA is where rare disease stakeholders gather to talk about strategies to expedite orphan drug ... roadblocks in manufacturing cell and gene therapies and how can companies efficiently scale-up for commercial production? ... The orphan drug space is booming with innovation, M&A activity and partnerships. We play match maker for up and coming biotechs ... The World Orphan Drug Congress USA is a place where innovation and expertise is showcased, solutions are found and learning is ...

https://www.terrapinn.com/conference/world-orphan-drug-congress-usa/

Novo Nordisk to Make Multi-Billion Dollar Investment in Expansion of Production Facilities in France ... EU Grants Orphan Drug Designation to ADV7103 for Treatment of Distal Renal Tubulopathy Acidosis. ... Biopharmaceutical company, Advicenne, announced that the European Union has granted orphan drug designation to its lead ... View MoreCOVID-19 UpdateAnalytical Method DevelopmentAPIsAseptic ProcessingBiologic DrugsDrug DeliveryEquipmentExcipients ...

https://www.pharmtech.com/view/eu-grants-orphan-drug-designation-adv7103-treatment-distal-renal-tubulopathy-acidosis

Eliminate domestic production activities deduction. -$95 billion. Eliminate orphan drug tax credit. -$54 billion. ... eliminating the orphan drug tax credit ($54 billion), and eliminating private activity bonds ($39 billion). The business tax ... eliminating the domestic production activities deduction ($95 billion), limiting carryover of net operating losses ($156 ...

https://www.crfb.org/blogs/tax-cuts-and-jobs-act-will-cost-15-trillion

Unveiled ECO Synthesis™ technology for RNAi therapeutics production at TIDES USA. 2022. CDX-6512 Received Orphan Drug ...

https://www.codexis.com/about-us/history

Contrary to current treatment approaches, which seek to reduce amyloid production by targeting plasma cells, NEOD001 targets ... NEOD001 has received orphan drug designation from the FDA and EMA.. At present, Prothena has two studies underway involving ... The pharmacodynamic effects of the drug did not result in meaningful clinical benefit for patients, though, as measured by ...

https://www.drugdiscoverynews.com/change-of-direction-for-prothena-11958

The U.S. Food and Drug Administration has granted Orphan Drug designation to fostamatinib for the treatment of patients with ... Unlike other therapies that modulate the immune system in different ways or stimulate platelet production, fostamatinib may ... the company expects to submit a New Drug Application with the U.S. Food and Drug Administration in the first quarter of 2017. ... Fostamatinib is an oral investigational drug with a unique mechanism of action designed to inhibit SYK kinase, a key player in ...

https://www.prnewswire.com/news-releases/rigels-fostamatinib-meets-primary-endpoint-in-phase-3-study-in-chronic-itp-300319730.html

Patients with rare diseases could lose possible treatments if Congress cuts tax credits that biotech companies rely on for drug ... the production of orphan drugs has increased. If theyre not given this credit, many people believe theyll stop creating drugs ... 963 million per new orphan drug. BioMarin, another orphan drug maker, spent $134 million per drug. ... Read More: ArmaGen Drug To Treat Rare Brain Disease Receives FDA Orphan Drug Status: First Successful Attempt To Get Large ...

https://www.medicaldaily.com/biotech-firms-align-drugmakers-health-advocates-keep-tax-credit-rare-disease-drug-development-250451

Product-related issues (biosimilars, doublets, companion diagnostics, orphan drugs). *Classification issues and "product- ... Regulatory issues (clinical trials, marketing authorizations, certifications, production, distribution). * ...

https://www.dentons.com/en/find-your-dentons-team/industry-sectors/life-sciences-and-health-care/life-sciences/regional-sectors/life-sciences-in-germany?Type=oMSq5Q565N4QsZOIL81UYGPfYft2/qAdAQlh47XuKv3XKsGtFqhgH+49M6L+d6PR

Prilenia holds Orphan Drug designation for pridopidine in ALS and Huntingtons Disease (HD) in the U.S. and EU. In addition, ... mitochondrial energy production and calcium homeostasis, which are essential to neuronal function and survival, and may lead to ... This EAP will evaluate the benefits of the investigational drug pridopidine in individuals with ALS and will be led by Healey ... pridopidine has received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD. ...

https://www.massgeneral.org/neurology/als/news/healeyamg-awarded-ninds-grant-for-eap-pridopidine

... orphan drugs and the making of Europe: An exercise in co-production, Annual Conference Science and Democracy Network, SDN, ... The Politics of Rare Diseases and Orphan Drugs, IX th Annual International Conference on Rare Diseases and Orphan Drugs (ICORD ... Hollak, C. E. M., Biegstraaten, M., Levi, M., & Hagendijk, R. (2015). Post-authorisation assessment of orphan drugs. Lancet, ... The politics of rare diseases and orphan drugs in Europe and beyond. ...

https://www.uva.nl/profiel/h/a/r.p.hagendijk/r.p.hagendijk.html

Rare diseases and disorders are usually treated with orphan drugs. Orphan drugs are medicinal products intended for use in ... As such, it is up to governments and rare disease organisations to advocate for the development and production of rare ... They are called orphan because the pharmaceutical industry has little interest in developing products for a small number of ...

https://www.wincalendar.com/ca/Rare-Disease-Day

Renewable electricity production credit.. *Indian employment credit.. *Employer social security credit.. *Orphan drug credit. ... orphan drugs (i.e. certain drugs intended to treat rare diseases or conditions that are designated as such by the FDA). For ... Research credit and orphan drug credit. The Credit for Increasing Research Activities under Section 41 (R&D credit) is ... The orphan drug credit (ODC), found in Section 45C, provides a credit for qualified clinical trial expenses (QCTEs) relating to ...

https://taxsummaries.pwc.com/united-states/corporate/tax-credits-and-incentives

Form 8820: Orphan Drug Credit. Form 8826: Disabled Access Credit. Form 8828: Recapture of Federal Mortgage Subsidy ... Form 8835: Renewable Electricity, Refined Coal, and Indian Coal Production Credit. Form 8844: Empowerment Zone and Renewal ...

https://www.hrblock.com/tax-center/support/online/prepare-to-file-taxes-online/tax-form-availability/

In addition, focusing on orphan drug production will require more samples for clinical trials. Globally, due to growing demand ... has proven essential to conducting in-depth drug research. Thus, such factors will fuel the growth of the market in this region ... from governments for more evidence-based drugs and with these trials using biological samples collected by biobanks will become ...

https://www.technavio.com/report/biobanking-market-industry-analysis

Repligen Receives Orphan Drug Designation from the FDA for RG1068 for MRI Imaging of the Pancreas ... the detection and delineation of normal and abnormal structures of the pancreas and to quantify pancreatic fluid production ... Food and Drug Administration (FDA) granted orphan drug designation to RG1068, synthetic human secretin, for use with magnetic ... Repligen Receives Orphan Drug Designation from the FDA for RG1068 for MRI Imaging of the Pancreas. WALTHAM, Mass., Nov. 21 ( ...

https://salesandmarketingnetwork.com/news_release_bs.html?pipe=0000ac177000191&ID=2015030&key=Repligen

Rare diseases are defined in the US Orphan Drug Act as those that affect fewer than 200,000 individuals in the US. Of these ... Appistry will help build a production-ready pipeline that uses an NIH UDP method of assembling and comparing genomes to ... Designed by the NIH and brought into production by Appistry, the pipeline analyses family genetics to narrow the search for ...

https://www.scientific-computing.com/news/appistry-and-nih-create-genetic-analysis-pipeline

FDA Grants Orphan Drug Designation to KRP203 Following allo-HSCT in Patients with Hematologic Malignancies ... Grant Aims to Strengthen Vaccine Production in Africa. November 30th 2023. Article ... FDA Grants Orphan Drug Designation to KRP203 Following allo-HSCT in Patients with Hematologic Malignancies ...

https://www.pharmacytimes.com/view/keeping-cancer-on-the-defensive

Inhibition of RORγ activity by inverse agonists has been shown to reduce the production of pro-inflammatory cytokines and ... Retinoic Acid Receptor-Related Orphan Receptor Gamma (RORγ). What is the Retinoic Acid Receptor-Related Orphan Receptor Gamma ( ... The development of RORγ-targeted drugs is an active area of research and holds great promise for the treatment of various ... INDIGOs RAR-related Orphan Receptor Gamma (RORγ) assay kit is an all-inclusive firefly luciferase reporter assay system that ...

https://www.stratech.co.uk/indigo/retinoic-acid-related-orphan-receptors-in-drug-discovery/

Sod House Theater teamed up the University of Minnesotas Center for Orphan Drug Research to develop an original work focued on ... 8 productions in venues from the Twin Cities to Fargo ND, Prescott Wi and Rochester MN. ... undereserved communities by working with local amateurs and organizations to participate with professionals in the production, ...

https://www.guidestar.org/Profile/82-2051698

Swedish Orphan Biovitrum AB (publ). All rights reserved. The product, its production and/or its use may be covered by one or ... Subscribe to Drugs.com newsletters Subscribe to Drugs.com newsletters for the latest medication news, new drug approvals, ... 7. Drug Interactions. No drug-drug interaction studies in human subjects have been conducted. Toxicologic and toxicokinetic ... This drug is known to be substantially excreted by the kidney, and the risk of toxic reactions to this drug may be greater in ...

https://www.drugs.com/pro/kineret.html

Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs). HSPs can rescue ... Arimoclomol has been granted Orphan Drug Designation (EU and USA) for the treatment of ALS. ... Arimoclomol, the companys lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, ...

https://www.worldwide.com/newsroom/worldwide-works-with-orphazyme-to-complete-recruitment-of-phase-3-trial-in-als/

coggle-session-production. 3 years. No description. coggle-session-production.sig. 3 years. No description. ... DAK-gesundheit, GBA, Germany, HTA, IQWiG, market access, orphan drugs, rare diseases, reimbursement ... The latest focus of attention seems to be orphan drug (OD) status, the relative Health Technology Assessment (HTA) and pricing ... We are award winning global experts in access for orphan drugs and Cell and Gene Therapies. ...

https://partners4access.com/blogs/winds-of-change-blowing-over-germany-for-orphan-drugs/

... drug target, MoA, RoA, and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, ... The Liver Failure pipeline drugs market research report provides comprehensive information on the therapeutics under ... Jul 17, 2017: Versantis Announces FDA Grants Orphan Drug Designation to VS-01 for the Treatment of Acute-on-Chronic Liver ... Aug 29, 2018: cGMP Compliance Status for Promethera Biosciences Production Site in Durham, NC Confirmed by Health Canada. ...

https://www.globaldata.com/store/report/liver-failure-hepatic-insufficiency-drugs-in-development-analysis/

Genoscience Pharma Received Orphan Drug Designation For Hepatocellular Carcinoma Drug. Genoscience Pharma has received FDA ODD ... Manufacturing & Production. Johnson & Johnson scale back COVID-19 vaccination production. US-based Johnson & Johnson (J&J) has ... A strongly drug-resistant bacterium has been found in various brands of artificial tears eye drops, infecting 55 people ... A team of scientists have broken records with their new AI which has found a novel drug candidate for liver cancer in only 30 ...

https://www.pharmafocus.com/collections/pharmafocus-march23/johnson-johnson-scale-back-covid19-vaccination-production

4SC receives Orphan Drug Designation (ODD) for resminostat (Kinselby) in CTCL from the US FDA [pta] ... WACKER Expands Specialty Silicone Production in China [pta]. Creactives Group will join the XIII edition of the "Lugano IRTOP ...

https://newsfox.com/

Biopharmaceutical company, Advicenne, announced that the European Union has granted orphan drug designation to its lead candidate ADV7103 for the treatment of distal renal tubulopathy acidosis (dRTA). (pharmtech.com)
WALTHAM, Mass., Nov. 21 (HSMN NewsFeed) -- Repligen Corporation (Nasdaq: RGEN ) announced today that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) granted orphan drug designation to RG1068, synthetic human secretin, for use with magnetic resonance imaging (MRI) of the pancreas. (salesandmarketingnetwork.com)
Arimoclomol has been granted Orphan Drug Designation (EU and USA) for the treatment of ALS. (worldwide.com)
In November 2015, the U.S. F.D.A. granted orphan drug designation for the combination of two active ingredients of AB569: sodium nitrite and ethylenediaminetetraacetic acid. (archbiopartners.com)
Ionis-HTTRx has been granted orphan drug designation by the US Food and Drug Administration and by the European Medicines Agency. (medscape.com)

Understanding these regulatory nuances can be critical for the development of such medicinal products for rare diseases, also known as orphan medicinal products. (xtalks.com)
Furthermore, the presentation aims to provide the audience with an understanding of how certain legal tools and incentives have stimulated the growth of the drug products for rare diseases and what other things can be done in order to achieve a better impact. (xtalks.com)
Advicenne focuses on the development of pediatric-friendly therapeutics for the treatment of orphan renal and neurological diseases. (pharmtech.com)
The politics of rare diseases and orphan drugs in Europe and beyond. (uva.nl)
If one looks at these changes one is immediately confronted, not only with the cognitive and emotional meanings of such diseases, i.e. what experts and patients know, experience and understand about the diseases, their causes and consequences, but also with politico-economic framings of help and care, with drug development and economic liberalism, with solidarity and with issues of politics, accountability and collective and individual responsibility and mobilizations. (uva.nl)
The U.S. Orphan Drug Act provides incentives for companies developing and marketing therapies for rare diseases, defined as those affecting fewer than 200,000 Americans. (salesandmarketingnetwork.com)
Designed by the NIH and brought into production by Appistry, the pipeline analyses family genetics to narrow the search for genetic changes that underlie many rare and undiagnosed diseases. (scientific-computing.com)
Rare diseases are defined in the US Orphan Drug Act as those that affect fewer than 200,000 individuals in the US. (scientific-computing.com)
Inhibition of RORγ activity by inverse agonists has been shown to reduce the production of pro-inflammatory cytokines and alleviate disease symptoms in animal models of autoimmune diseases. (stratech.co.uk)
The development of RORγ-targeted drugs is an active area of research and holds great promise for the treatment of various immune-mediated diseases. (stratech.co.uk)
Sod House Theater teamed up the University of Minnesotas Center for Orphan Drug Research to develop an original work focued on bringing attention to those diagnosed with rare diseases. (guidestar.org)
Arimoclomol, the company's lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic i nclusion b ody m yositis, and a myotrophic l ateral s clerosis. (worldwide.com)
The act serves to encourage pharmaceutical companies to develop drugs for diseases that individually affect less than 200,000 people in the US. (chemistryworld.com)
Before the ODA stepped in, only 38 drugs were approved in the US specifically to treat rare diseases. (chemistryworld.com)
As of 2010, 200 of the roughly 7000 officially designated orphan diseases have become treatable. (chemistryworld.com)
Although an increased number of drugs for rare diseases have since been developed and marketed, the extremely high price of some such drugs is considered a major public health issue internationally. (elsevierpure.com)
The reduced patient pool for rare diseases limits the number of people who can benefit from orphan drugs and therefore also limits profitability and return on investment. (ramarketingpr.com)
There are over 7,000 rare diseases and the production of orphan drugs changes the lives of those with the condition. (ramarketingpr.com)
We argue that disease specific (rather than drug specific) registries, supervised by independent clinicians are urgently needed for the best long-term evaluation of treatments of these rare diseases. (biomedcentral.com)
The European Union enacted the Orphan Drug Regulation in 2000 ( (EC) No 141/2000 and (EC) No 847/2000 ) in order to improve the availability of innovative medicines for diseases affecting less than 5/10 000 people. (biomedcentral.com)
Existing drug registries for evaluation of the effectiveness of treatments for some orphan diseases have certain limitations in this respect. (biomedcentral.com)
The FDA grants orphan drug status to novel therapies that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 U.S residents. (octapharma.com)
Though the word "orphan" might imply something lost or lacking attention, orphan diseases have been anything but in recent years. (lubrizolcdmo.com)
As a result, there are now treatment options available for more than 700 orphan diseases, improving the lives of millions of previously underserved patients 4 . (lubrizolcdmo.com)
There are of course other treatments besides orphan drugs for these diseases. (atheneum.ai)

Octapharma USA today announced the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has awarded seven years of marketing exclusivity for Octagam ® 10% [Immune Globulin Intravenous (Human)], the first and only intravenous immunoglobulin (IVIg) to be indicated for the treatment of adult dermatomyositis (DM). (octapharma.com)

The conference is a place to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies. (terrapinn.com)
What are the current roadblocks in manufacturing cell and gene therapies and how can companies efficiently scale-up for commercial production? (terrapinn.com)
We are award winning global experts in access for orphan drugs and Cell and Gene Therapies. (partners4access.com)
As a result, we hear that an age of personalized medicine is dawning, in which therapies will be tailored to groups of patients, or even individuals.This brave new world would mean the end of the blockbuster drug model and would transform the way that medicine is practiced, and drugs are made and marketed. (pharmamanufacturing.com)
The manufacturing response In response to the promises and threats of personalized therapies, pharmaceutical companies must accelerate retooling and streamlining of production lines, just as heavy and light manufacturers in other industries have done over the last twenty years. (pharmamanufacturing.com)
Pharmaceutical companies simply could not financially justify pursuing orphan disease therapies. (lubrizolcdmo.com)
In recognition of this challenge, the FDA granted therapies with orphan disease designation a seven-year exclusivity period to motivate the industry and benefit underserved patients down the line 4 . (lubrizolcdmo.com)
It is estimated that only around 50% of orphan drug therapies that have lost exclusive rights face generic competition 6 . (lubrizolcdmo.com)
For individuals with mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke (MELAS) syndrome and for those with other oxidative phosphorylation (OXPHOS) disorders, metabolic therapies are administered to increase the production of adenosine triphosphate (ATP) and to slow or arrest the deterioration of this condition and other mitochondrial encephalomyopathies. (medscape.com)

Meanwhile, according to the EU's Orphan Drug Regulation 141/2000, a disease or disorder is defined as rare when it affects fewer than 1 in 2000 European citizens. (chemistryworld.com)
The US FDA Orphan Drug Act, the EU Regulation (EC) No 141/2000, and other similar incentives have been put in place across the globe. (ramarketingpr.com)

Government intervention to create the market for so-called orphan drugs can include tax incentives, enhanced patent protection and financial subsidies for clinical research. (chemistryworld.com)
Incentives have already been put in place to encourage orphan drug development, but further strategic steps must be made to continue innovation and success in the rare disease space. (ramarketingpr.com)
An orphan drug designation in Europe provides incentives to a pharmaceutical company to develop a medicine for a rare disease. (archbiopartners.com)
Such incentives include protocol assistance, which allows the drug company to get guidance from the EMA on the types of clinical studies needed to demonstrate the quality, benefits and risks of the new medicine. (archbiopartners.com)
Other incentives include access to a centralized authorization procedure and lower regulatory fees whereby a company can make a single new drug application to the EMA, resulting in a single drug approval decision from the EC, valid in all European Union Member States. (archbiopartners.com)
The Orphan Drug Act (ODA) was passed and suddenly created highly attractive incentives for pharmaceutical companies to pursue otherwise unprofitable disease areas. (lubrizolcdmo.com)
What Are the Incentives of the Orphan Drug Act? (lubrizolcdmo.com)

Fulfilling the unmet need for rare disease medicines (known as orphan drugs) remains a challenge for the pharmaceutical industry. (ramarketingpr.com)

The Liver Failure pipeline drugs market research report provides comprehensive information on the therapeutics under development for Liver Failure, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. (globaldata.com)
An important class of therapeutics, the biopharmaceutical or 'biologic' (peptide or protein drugs) would be particularly amenable to in space manufacturing. (parabolicarc.com)

Founded in Trondheim in 2007 the company has been chartered by the Norwegian Ministry of Health and Care Services to improve global access to orphan medicines. (wikipedia.org)
Although the European Medicines Agency grants market access to its 27 member states, in practice, medicines only reach the market when each member state decides that its national health system will reimburse for the drug. (chemistryworld.com)
Today s pipeline drugs are the medicines that companies will be launching and marketing for the next 10 to 20 years, he says. (pharmamanufacturing.com)
For Fabry disease, two enzymes were authorized at the same time resulting in two different drug registries being required by the European Medicines Agency (EMA) to monitor effectiveness and safety. (biomedcentral.com)
TORONTO, ONTARIO-(Marketwired - Feb. 3, 2016) - Arch Biopartners Inc. (Arch, or the Company) (TSX VENTURE:ACH)(OTCBB:FOIFF), a portfolio based biotechnology company, today announced they have submitted an application for orphan drug designation to the European Medicines Authority (EMA) for AB569 in the treatment of Pseudomonas aeruginosa (P. aeruginosa) pulmonary infections in patients with cystic fibrosis. (archbiopartners.com)
Orphan medicines approved by the EC further benefit from ten years of protection from market competition from similar drugs targeting the same indication. (archbiopartners.com)
Looking ahead, preparations for the Phase III study of EscharEx ® are progressing, with an updated protocol shaped by U.S. Food & Drug Administration (FDA) and European Medicines Agency (EMA) guidance. (whnt.com)

DelveInsight's, "Huntington's Disease - Pipeline Insight, 2023," report provides comprehensive insights about 50+ companies and 50+ pipeline drugs in Huntington's Disease pipeline landscape. (delveinsight.com)

Pricing orphan products, especially one-time treatments, is complex and a key element to ensuring timely access for rare disease patients. (terrapinn.com)
How can developers overcome barriers to successfully produce orphan drugs for rare disease treatments? (ramarketingpr.com)
In this blog, we explore two of the biggest barriers orphan drug developers face as they try to get transformative treatments to the market. (ramarketingpr.com)
The existing registries for orphan drug treatments for lysosomal storage disorders (LSD's) illustrate these limitations. (biomedcentral.com)
The FDA approval of orphan drug status for Octagam ® 10% to treat adults with DM should encourage patients to ask their physicians about the therapy because they no longer have to rely on treatments that have not been specifically evaluated by the FDA for DM," said Octapharma USA President Flemming Nielsen. (octapharma.com)
KM-819, developed by Kainos Medicine, is an innovative new drug that improves the degenerative cranial nervous system through a novel mechanism with a dual-action function, unlike existing Parkinson's disease treatments. (fox2now.com)

A condition that affects more than 200,000 people nationwide, but for whom "there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from the sale in the United States of such drug 2 . (lubrizolcdmo.com)

Carol has about 20 years of drug development experience gained in small, medium and large pharma companies working in Germany, the UK and France. (xtalks.com)
What are best practices right now for data collection, ownership and sharing to benefit rare disease diagnosis, drug development and access. (terrapinn.com)
The rare disease space is a breeding ground for innovative approaches in drug development and platform technologies. (terrapinn.com)
Understanding the regulatory and clinical landscape for rare disease drug development. (terrapinn.com)
As the rare cancer field evolves and diagnosis becomes more precise, how can all stakeholders work together to advance drug development for rare cancer patients? (terrapinn.com)
Approaches in AI and digital health span the full development life cycle of drug development and commercialization. (terrapinn.com)
From early phase and bioanalytical science s through late phase, post-approval and real-world evidence , we provide world-class, full-service drug development services. (worldwide.com)
Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, but some statistical burdens are lessened in an effort to maintain development momentum. (chemistryworld.com)
Rare disease drug development unfortunately has a lot of treatment inequalities compared to more common conditions - but why is this the case? (ramarketingpr.com)
The reality is that drug development is never barrier-free, and orphan drug development is no exception. (ramarketingpr.com)
orphan drug development is challenging but vital work. (ramarketingpr.com)
In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Huntington's Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details. (delveinsight.com)
This segment of the Huntington's Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. (delveinsight.com)
To some extent, developed nations subsidise the development of orphan drugs if they are believed to be necessary. (dandc.eu)
Senior researcher Hui-Chen Lu hopes this research will lead to the development of new drugs that can create a chemical blockade against the effects of neurodegenerative illnesses. (bipolarnews.org)
SN BioScience (CEO Park Young-hwan), a drug developer, received orphan drug designation from the U.S. Food and Drug Administration and the FDA in recognition of the small cell lung cancer indication for SNB-101 (main ingredient: SN-38), a new polymer nanoparticle drug under development at the time. (fox2now.com)
Cellengene (CEO Ahn Jae-hyeon), a cell gene therapy developer, was selected as a candidate material stage project in the "New Drug R&D Ecosystem Construction Project" category in the "1st National New Drug Development Project" hosted by the Korea Drug Development Fund (KDDF) in July this year. (fox2now.com)
There is a need for incentive structures to stimulate research and development when there is no market or there is market failure in the production and diffusion of knowledge. (who.int)
The drug was safe and well tolerated, as judged by an independent safety committee, which "supports continued development," Ionis said in a news release . (medscape.com)

From the passage of the ODA in 1983 until May 2010, the FDA approved 353 orphan drugs and granted orphan designations to 2116 compounds. (chemistryworld.com)
This paper examines the history of orphan drug policy, from the emergence of 'orphans' in the American pharmaceutical market in the 1960s, through the debates and agitations that resulted in the passage of the US Orphan Drug Act of 1983, to attempts in the 1990s to prevent abuse of that Act and restore its original intentions. (elsevierpure.com)
The present paper traces the origins of this issue to the market-based approach to resolving the problem of orphan drugs embodied in the 1983 Act. (elsevierpure.com)

Contrary to current treatment approaches, which seek to reduce amyloid production by targeting plasma cells, NEOD001 targets the accumulated amyloid, and its proposed mechanism of action is "to neutralize soluble aggregates and clear insoluble aggregates from organs," per the website. (drugdiscoverynews.com)
Treatment with a new class of drugs, called senolytics, in donors improved the physical fitness of the recipients, a new study has shown. (medicaldaily.com)
Prilenia has an orphan drug designation for pridopidine for the treatment of Huntington's disease in both the US and Europe. (delveinsight.com)
To date, there is no pharmaceutical treatment specifically for coronavirus, so drug repurposing offers rays of hope. (dandc.eu)
About two dozen previously existing drugs are currently being tested for Covid-19 treatment. (dandc.eu)
It is nonetheless in huge demand as a supportive treatment or sometimes as a survival drug, not least in India in view of the current coronavirus surge. (dandc.eu)
The average number of patients per orphan treatment is only 5,730 individuals 3 , 4 . (lubrizolcdmo.com)
In 1997, Duke University applied for Orphan Drug Application for treatment using recombinant GAA modified with M6P, which application was associated with a press release asserted as prior art in the IPR reviewed by the Federal Circuit. (patentdocs.org)

But that began to change in the 1980s, when the US Food and Drug Administration's (FDA's) Orphan Drug Act (ODA) and, subsequently, the EU's Committee on Orphan Medicinal Products and mirror organisations around the world, stepped in. (chemistryworld.com)
It is expected that the Committee for Orphan Medicinal Products (COMP) will begin review of the application in approximately 30 days and will provide an opinion on the application that is forwarded to the European Commission (EC) by the end of May 2016. (archbiopartners.com)

This EAP will evaluate the benefits of the investigational drug pridopidine in individuals with ALS and will be led by Healey & AMG Center faculty, Drs. Suma Babu, James Berry, and Sabrina Paganoni. (massgeneral.org)
The EAP made possible by this award will allow ALS individuals who are otherwise not eligible for participation in clinical trials of pridopidine to access this investigational drug. (massgeneral.org)

In addition to industry appointments, he was previously an adjunct professor in the Drug Regulatory Program at LIU. (xtalks.com)
The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. (terrapinn.com)
In November, Kainos Medicine's AIDS drug KM-023 was included in the list of priority review drugs selected by the Center of Drug Evaluation of NMPA, China's national drug regulatory authority. (fox2now.com)

Oncology drugs are driving the marketplace closer to the vision of personalized medicine, but major infrastructure change is still needed. (pharmamanufacturing.com)
He has knowledge on a range of orphan oncology indications including Merkel Cell Carcinoma and is able to discuss the challenges around orphan drug patient journeys. (atheneum.ai)

US-based insurers and single payer agencies around the world have a lot of impact of access and reimbursement for orphan products. (terrapinn.com)
In fact, it called for the end of the lighter HTA reserved for OD drugs, and for an overall tougher stance on reimbursement policy, with the government already planning a halving of the free-pricing period from 12 to 6 months. (partners4access.com)

Expanded Access, also referred to as Compassionate Use, is an FDA-regulated pathway that allows individuals with a serious and life-threatening disease to access an investigational drug that is not yet approved by the FDA. (massgeneral.org)
Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs). (worldwide.com)
Tominersen (formerly known as IONIS-HTTRx and RG6042) is an investigational antisense medicine designed to reduce the production of the huntingtin (HTT) protein, which is the genetic cause of Huntington's disease (HD). (delveinsight.com)

This was largely due to the fact that, with such small patient populations per indication, the potential profit margin for a drug was slim. (lubrizolcdmo.com)
So, within just the span of a couple of years, going from zero approved indication specifically for this rare skin condition to multiple agents, really shows the spectrum of what can happen in the orphan disease space. (atheneum.ai)

The pharmacodynamic effects of the drug did not result in meaningful clinical benefit for patients, though, as measured by responses in the Psoriasis Area and Severity Index 75. (drugdiscoverynews.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. (prnewswire.com)
For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1000 patients in a Phase III clinical trial, as there may not even be 1000 patients with a particular disease. (chemistryworld.com)
Others see the blockbuster model as outdated, since even the most successful drugs don t work in all the patients all the time. (pharmamanufacturing.com)
However, once patients were singled out and treated who expressed the HER2/neu gene, linked to growth of more aggressive tumors, the drug s efficacy shot up, justifying the adverse events. (pharmamanufacturing.com)
CF patients are predisposed to lung infections due to abnormal mucus production in the lungs and airways. (archbiopartners.com)
This drug does not cure the disease, but it has been proven that it reduces the time patients are hospitalised, need to stay in intensive-care units (ICUs) and depend on ventilators. (dandc.eu)
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)

Finally, we provided some insights into the correlation between the sugar side chain of MIC and its role in enhancing of RIP cytotoxicity and altering of drug cell tropism. (bvsalud.org)

Several natural compounds, such as melatonin and honokiol, have also been shown to modulate RORγ activity and are being investigated as potential drug candidates for various indications, including inflammation and cancer. (stratech.co.uk)
The idea is to use an established drug for new therapeutic indications. (dandc.eu)

The lysosomal storage disorder (LSD) alpha-Mannosidosis is a rare genetic disease and according to the EU regulations, designated as an "orphan" disease. (europa.eu)

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. (prnewswire.com)

Hear from top US and international payers on their strategies for orphan drugs. (terrapinn.com)
What are the current best practices for pricing orphan products, negotiating innovative contracts and working with payers around the world? (terrapinn.com)
Therefore, a restriction of the orphan "privilege" is on the horizon in Germany, with policymakers, payers and HTA bodies calling for a rehaul of the OD status. (partners4access.com)
The high costs of orphan drugs have generated a lot of attention from payers and policy groups. (ramarketingpr.com)

Some of the targets of the Liver Failure pipeline drugs market are Caspase, Dual Specificity Mitogen Activated Protein Kinase Kinase 4, Toll Like Receptor 4, Tumor Necrosis Factor, and Androgen Receptor among others. (globaldata.com)
Caspase leads this segment of the Liver Failure pipeline drugs market. (globaldata.com)
Caspase Inhibitor leads the Liver Failure pipeline drugs market in terms of MoA. (globaldata.com)
The key routes of administration in the Liver Failure pipeline drugs market are intravenous, oral, intraperitoneal, intramuscular, and parenteral among others. (globaldata.com)
Intravenous leads the Liver Failure pipeline drugs market in terms of RoA. (globaldata.com)
The molecule types in the Liver Failure pipeline drugs market are small molecule, cell therapy, biologic, oligonucleotide, and blood derivative among others. (globaldata.com)
Small Molecule leads the Liver Failure pipeline drugs market in terms of molecule types. (globaldata.com)
Some of the key companies in the Liver Failure pipeline drugs market are Akaza Bioscience Ltd, Cellaion SA, Genfit SA, HepaRegeniX GmbH, Immune Pharmaceuticals Inc, and Jiangsu Hengrui Medicine Co Ltd among others. (globaldata.com)
the act also received some criticism for allowing some pharmaceutical companies to make a large profit from drugs that have a small market but still sell for a high price. (chemistryworld.com)
For example, 35 orphan drugs reached the market in Belgium, 44 in the Netherlands, and 28 in Sweden in 2008. (chemistryworld.com)
The paper also makes visible an alternative trajectory that existed for a while in the United Kingdom but was eventually abandoned in order to help the biotechnology industry grow in the context of an increasingly integrated European drug market. (elsevierpure.com)
Mikami, K 2019, ' Orphans in the Market: The History of Orphan Drug Policy ', Social History of Medicine , vol. 32, no. 3, pp. 609-630. (elsevierpure.com)
Proper management of these areas will enhance the probability of drug success in the market. (ramarketingpr.com)
After market approval for the orphan designation, the FDA/OOPD will award seven years of market exclusivity. (octapharma.com)
In addition, the duration of protected status is often even longer than seven years, as patent protection frequently applies beyond the date of expiration for orphan market exclusivity 6 . (lubrizolcdmo.com)
Furthermore, even when a patent does expire, there is a substantial lack of generic competition in the orphan drug market as compared to other industry segments. (lubrizolcdmo.com)
It is no secret to anyone in the industry that the time and effort expensed in bringing a new drug to market is significant. (lubrizolcdmo.com)
A standard drug product takes an average of 12 years to develop and market, with a large portion of that time spent in clinical trials. (lubrizolcdmo.com)

Appistry will help build a production-ready pipeline that uses an NIH UDP method of assembling and comparing genomes to identify changes that may be causing disease. (scientific-computing.com)
It covers the pipeline drug profiles, including clinical and nonclinical stage products. (delveinsight.com)

Ali and colleagues screened 1280 compounds to identify those that might increase NMNAT2 production. (bipolarnews.org)
Thirteen of the compounds tested decreased NMNAT2 production. (bipolarnews.org)

Twenty-four of these looked promising, including caffeine and rolipram, an "orphaned drug" once studied as an antidepressant but discontinued in the 1990s. (bipolarnews.org)

What are the best commercial strategies for orphan products? (terrapinn.com)
On the other hand, pharma companies that specialise in innovative patent-protected drugs will benefit if repurposing means they can sell branded products for more than the originally intended application. (dandc.eu)
This advantage should be strongly considered when looking into developing an orphan drug, as should choosing an outsourcing partner that knows the in-and-outs of these niche products. (lubrizolcdmo.com)

We might experience the onset of a race between ODs to be considered "soloist", should direct therapy comparison become the norm for all the "non-soloist" drugs. (partners4access.com)

The single-dose shot from Pfizer, known as Abrysvo, has already been approved by the Food and Drug Administration. (medicaldaily.com)
Enter the Food and Drug Administration. (lubrizolcdmo.com)

Awareness of these barriers and the ability of drug developers to address them can make or break an orphan drug project and drastically change patient therapeutic access. (ramarketingpr.com)
Due to system maintenance, the drug interactions feature you are attempting to access is temporarily unavailable. (medscape.com)

The use of a non- invasive procedure such as MRI to improve the detection and delineation of normal and abnormal structures of the pancreas and to quantify pancreatic fluid production following secretin administration harnesses the natural biologic properties of the hormone and may improve the diagnostic quality of the MRI image. (salesandmarketingnetwork.com)
If successful in developing the Astropharmacy technology, the quality of astronaut healthcare will be improved by eliminating concerns of biologic drug degradation. (parabolicarc.com)

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)
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)

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)
Some of these mutations change single protein building blocks (amino acids) in the ROR2 protein, while others lead to the production of an abnormally short, nonfunctional version of the protein. (medlineplus.gov)

The designation also provides for benefit from certain tax credits and waives the company's obligation to pay the FDA application user fees for this product as required by the Prescription Drug User Fee Act. (salesandmarketingnetwork.com)
A full HTA and a regular benefit assessment is triggered if the €50million turnover threshold is hit by the drug. (partners4access.com)
Health-care systems can benefit from the repurposing of generic drugs in particular. (dandc.eu)

Orphan drug designation qualifies Repligen for seven years of exclusive marketing rights in the United States if the company is first to receive marketing approval for RG1068 for MRI imaging of the pancreas. (salesandmarketingnetwork.com)
Under the law, companies that develop these drugs may sell them without competition for seven years. (chemistryworld.com)

This could help to help drive successful HTA outcomes with a view to sustain current price tags and revenues in Germany for orphan drugs. (partners4access.com)
How would you describe the current orphan drug patient journey? (atheneum.ai)

A small but growing number of companies are applying a technique called theranostics, identifying potential responders a priori, and bundling appropriate drugs with diagnostics.Consider Genentech s Herceptin, for example. (pharmamanufacturing.com)

By Angelo De Palma, Ph.D., Contributing Editor Recent breakthroughs in genomics have made it possible to predict, more accurately than ever before, how each patient will respond to any given drug. (pharmamanufacturing.com)
Armed with new testing methodologies, manufacturers would nimbly change product and production schedules in response to changing patient needs.Are we there yet? (pharmamanufacturing.com)
Improperly expressed proteins can cause a drug to be completely ineffective but innocuous in one patient, ineffective but toxic in another, or safe and effective in yet another. (pharmamanufacturing.com)

The orphan drug space is booming with innovation, M&A activity and partnerships. (terrapinn.com)
We propose a concept to tailor-make drugs, initially non-glycosylated biologics, by pre-programming cells that are space hardy due to being in the spore form, to produce them with the addition of ~1 mL of sterile medium using a lightweight, small volume system adapted from standard laboratory protocols and enabled by judicious genetic engineering prior to launch. (parabolicarc.com)
Finally, such systems could have spin-offs for on-demand, on-site production of small quantities of other useful peptides/proteins in space, as well as on Earth, an economical path forward for orphan drugs, and improved stability for prototyping genetic circuits in cell-free systems. (parabolicarc.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)

Diseases1

Analytical, Diagnostic and Therapeutic Techniques and Equipment2

Disciplines and Occupations1

Anthropology, Education, Sociology and Social Phenomena4

Technology, Industry, Agriculture2

Humanities1

Health Care5

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

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

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

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

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

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

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

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

Orphan Drug Production

Rare Diseases

Drug Approval

Economics, Pharmaceutical

Legislation, Drug

United States Food and Drug Administration

Tropical Medicine

Ethics, Pharmacy

Drug Industry

Off-Label Use

Patents as Topic

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

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

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

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

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

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

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

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

Orphan-Nor

Northwest Biotherapeutics

Epitopoietic Research Corporation

Orphan drug

Mustang Bio

Orphan Drug Act of 1983

Ezutromid

Imiglucerase

Antisense therapy

Clofazimine

Alvocidib

Telesta Therapeutics

Respiratory tract antimicrobial defense system

Argentine hemorrhagic fever

Orphan Black

Oxandrolone

Peptide receptor radionuclide therapy

Patisiran

Lanadelumab

Alicaforsen

Henri Termeer

List of MeSH codes (J01)

Leadiant Biosciences

Specialty pharmacy

Interferon beta-1a

Small interfering RNA

Asunercept

The Gold Dust Orphans

Ansuvimab

Casimersen

Out-of-pocket Spending on Orphan Drug Prescriptions Among Commercially Insured Adults in 2014

Orphanet: Search for an orphan drug

Orphan Drug & Rare Disease Development: Understanding the European and US Regulatory Landscape - Xtalks

Mustang Bio's AML Treatment Receives Orphan Drug Designation | Contract Pharma

Orphan-Nor - Wikipedia

World Orphan Drug Congress USA 2024 | 23 - 25 April 2024

EU Grants Orphan Drug Designation to ADV7103 for Treatment of Distal Renal Tubulopathy Acidosis

Tax Cuts and Jobs Act Will Cost $1.5 Trillion | Committee for a Responsible Federal Budget

Our History :: Codexis, Inc. (CDXS)

Change of direction for Prothena | Drug Discovery News

Rigel's Fostamatinib Meets Primary Endpoint in Phase 3 Study in Chronic ITP

Biotech Firms Align With Drugmakers, Health Advocates, To Keep Tax Credit For Rare-Disease Drug Development

Dentons - Life Sciences in Germany

Sean M. Healey & AMG Center for ALS awarded NIH U01 Grant to support Expanded Access to Pridopidine in Collaboration with...

Dr. R.P. (Rob) Hagendijk - Universiteit van Amsterdam

Rare Disease Day in Canada - Thursday, February 29, 2024

Tax credits and incentives

Tax Form Availability Online | H&R Block

Biobanking Market Size, Share & Trends [2023 Report]

News for Medical Independent Sales Representatives and Medical Distributors: Repligen Receives Orphan Drug Designation from the...

Appistry and NIH create genetic analysis pipeline | Scientific Computing World

Keeping Cancer on the Defensive

Retinoic Acid-related Orphan Receptors in Drug Discovery - Stratech

Sod House Theater Company - GuideStar Profile

Kineret: Package Insert - Drugs.com

Worldwide Helps Orphazyme Complete Recruitment of Phase 3 ALS Trial

Winds of change blowing over Germany for Orphan Drugs » Partners4Access

Liver Failure (Hepatic Insufficiency) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022...

Johnson & Johnson Scale Back COVID-19 Vaccination Production

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Granted orphan drug designation5