CRAIGAVON, Northern Ireland, November 15, 2012 /PRNewswire/ --. Galen announced today that the European Commission has designated liposomal daunorubicin as an orphan medicinal product for the treatment of acute myeloid leukaemia (AML).[1] The designation follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA).[2] Liposomal daunorubicin is currently approved in a number of European countries, the U.S. and Brazil for the treatment of advanced HIV-related Kaposis sarcoma, as the medicinal product DaunoXome®.. Acute myeloid leukaemia is estimated to affect not more than 1.2 in 10,000 people in the European Union. It is chronically debilitating and life threatening due to bone marrow dysfunction. If left untreated, the condition progresses rapidly and is often fatal.[2]. Galen is dedicated to supporting the development and provision of innovative medicines in an effort to improve health worldwide. Speaking about the European ...
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Looking for Orphan drugs? Find out information about Orphan drugs. drug developed under the U.S. Orphan Drug Act to treat a disease that affects fewer than 200,000 people in the United States. The orphan drug law offers tax... Explanation of Orphan drugs
The 8th annual World Orphan Drug Congress is the marketplace for orphan drug professionals looking at the complete value chain of orphan drug development, from clinical development and R&D to corporate development and market access.. Being Europes largest Orphan Drug Congress, this event will provide a platform for you to showcase your thought leadership and expertise pharma, biotechs, payers, regulators and patient advocates.. Join us in November and make sure you are at the forefront of the orphan drug industry. Regardless of whether your interest lies in research, clinical development, patient access, global pricing and reimbursement or just to engage with patient advocacy groups more intimately, we have content, networking and potential partners for you!. Take just three days out of the office to meet with 400+ potential customers who need to find solutions to challenges around the commercial, regulatory and scientific issues in orphan drugs. Through strategic keynote plenaries, themed ...
There is some good news for mesotheliomasufferers in a recent report from Thomson Reuters on the orphan drug market. According to Reuters, rare diseases like mesothelioma are "winning unprecedented attention" from drug manufacturers. More than a quarter of the 39 new drugs approved by the FDA in 2012 were granted orphan drug status because they were designed to treat diseases (such as mesothelioma) that affect fewer than 200,000 people a year.. Mesothelioma is an aggressive asbestos-linked cancer of internal membranes. It is considered an orphan disease because it claims the lives of about 2,500 Americans annually. The Reuters report on orphan drugs is good news for mesothelioma patients because few treatment options currently exist for them and their prognosis is often poor. By forgoing certain regulations and fees, orphan drugs can be brought to market faster and potentially benefit more people with diseases like mesothelioma.. Two of the orphan drugs approved in 2012 were mesothelioma drugs. ...
GARD has information from the Food and Drug Administration (FDA) on treatments approved for rare diseases, known as orphan products/drugs. The Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product. The following information comes from the FDA database of orphan drug designations and approvals. You can output the data into an Excel spreadsheet from the FDA website. More information on orphan drug development is available from Global Genes and Orphanet ...
The objective of the Orphan Drug Act of 1983 was simple: To inspire drug companies to develop new drugs for small populations - under 200,000 - theyd get a seven-year exclusivity deal and protection from any competition, so long as no one else came up with a clinically superior product. To sweeten the deal, the government added a 50-percent tax credit on all research expenses.. The government got what it wanted. As of 2001, there were some 1,000 designated orphan products and 242 had been given official marketing sanction, all approved by a special orphan drug office, which the OIG gives high marks for responsiveness and ease of access. In 1983, by contrast, there were only a handful of orphan products, and the new drugs created since included big advances for some terrible conditions like cystic fibrosis, hemophilia, and multiple sclerosis.. For some, orphan drugs can make the difference between life and death. But that does not change the realities of the drug development business. Orphan ...
FDA Orphan Drug Designation received earlier this month. , EMA and FDA Orphan designations validate odiparcil potential to improve treatment options for MPS VI patients. , iMProveS phase IIa study in MPS VI patients on track to begin recruitment by year-end 2017. Daix (France), August 29, 2017 at 07:30am CEST - Inventiva, a biopharmaceutical company developing innovative therapies, particularly in fibrosis, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to odiparcil (formerly IVA336) for the treatment of MPS VI.. "This decision could accelerate the availability of a much needed new treatment for MPS VI patients,;" said Christine Lavery, President of the UK MPS Society added Professor Chris Hendriksz, of FYMCA Medical Ltd. and University of Pretoria, South Africa.. "We recently received U.S. orphan drug status and with this new EU designation we continue delivering on our regulatory strategy for odiparcil. Clearly the recent preclinical data we ...
The decision has been communicated by the Agency on June 21 and follows the positive opinion issued by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) on November 12, 2015, recommending the designation of the medicinal product containing Sirolimus as an orphan medicinal product for the indication: treatment of beta thalassaemia intermedia and major.. Marco Prosdocimi, Managing Director of Rare Partners, said that "This important result further confirm that the collaboration we established since 2011 with Roberto Gambari (University of Ferrara) and his collaborators is extremely fruitful. Their research, supported for many years by AVLT (Associazione Veneta per la Lotta alla Talassemia) has obtained outstanding results, with potential application in patients within a short time frame. A collaborative grant, obtained by Rare Partners from Wellcome Trust (UK) within the Pathfinder Award scheme, allowed completion of fundamental preclinical work and ...
CPX-351, a drug therapy for acute myeloid leukemia, has been raised by the U.S. Food and Drug Administration, FDA, to orphan drug status. Now with seven years market exclusivity in the U.S, Celator Pharmaceuticals will continue through to phase 2 clinical trials.. According to medcitynews.com, its goals involve a fund of $5 million, and $2.57m of this target has been raised through equity, options and securities so far.. Understand how governments are collaborating to improve the orphan drugs challenges, learn the strategic solutions, and discover the trends of technology investments at the World Orphan Drugs Congress USA 2012.. The congress speakers consist of business and scientific industry leaders from North America and beyond. Register now, and meet decision-makers from big pharma, early stage and mid stage biotechs, government and regulatory bodies.. ...
An experimental eye treatment under development by Advanced Cell Technology Inc. has received orphan drug status from European authorities.
GARD has information from the Food and Drug Administration (FDA) on treatments approved for rare diseases, known as orphan products/drugs. The Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product. The following information comes from the FDA database of orphan drug designations and approvals. You can output the data into an Excel spreadsheet from the FDA website. More information on orphan drug development is available from Global Genes and Orphanet ...
H3 Biomedicine, a member of Eisais global Oncology Business Group, has received orphan drug designation from the US Food and Drug Administration (FDA) for its H3B-8800, a clinical compound used to treat patients with acute myelogenous leukaemia (AML) and chronic myelomonocytic leukaemia (CMML).. H3 Biomedicine is a clinical stage biopharmaceutical company that focuses on the discovery and development of precision medicines for oncology.. H3B-8800 is a potent, selective and orally bioavailable small molecule modulator of wild-type and mutant SF3b complexes.. H3 Biomedicine president and CEO Dr Markus Warmuth said: "Receiving the orphan drug designation for H3B-8800 is a critical milestone for H3s ongoing cancer genomics driven drug discovery programme.. ...
as its vice-chair. Both have been elected for a three-year mandate.. Prof. Sepodes brings his academic experience as a research scientist in pharmacology, immunopharmacology and pharmacotoxicology to his role as chair of the COMP, a committee that has a long tradition in dealing with state-of-the-art pharmaceutical innovation. He first became a member of the COMP in 2008 following nomination by the European Commission on the recommendation of the Agency.. Ms Greenes election continues another tradition of the COMP: since the establishment of the Committee in 2001, the vice-chair has always been a representative of a patient organisation. This highlights the importance the contribution of civil society to the work of the COMP.. COMP opinions. During its September 2012 meeting, the COMP adopted a total of 12 recommendations for orphan designation. One of these medicines is recombinant human lecithin cholesterol acyltransferase, intended for the treatment of lecithin cholesterol acyltransferase ...
Why a regulation for Orphan Medicines is needed? Some conditions occur so infrequently that the cost of developing a medicinal product would not be recovered by the expected revenues. Therefore the pharmaceutical industry is unwilling to develop these medicines under normal market conditions. Patients suffering from rare conditions should be entitled to the same quality of treatment as other patients (EC Regulation No 141/2000)
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORDs founding at a dinner tonight in Washington, D.C.
Ensuring the development and availability of drugs that are medically important but commercially unprofitable poses a significant problem in our free market economy. Recent attempts by the federal government to develop a policy regarding these "orphan" drugs provides an important first step. The federal Interagency Task Force on Drugs of Limited Commercial Value recommendations propose initiation of a wide spectrum of administrative and legislative changes; these are designed to create incentives for industry to cooperate with government in developing and making orphan drugs available to those who need them. But before the proposed recommendations are accepted or rejected, a clearer understanding is needed of the nature of interrelated problems of orphan drugs. Thereafter, the wide spectrum of "stakeholders" involved in and affected by orphan drug policies or programs should seek means for interactive planning to determine desired ends and to devise means for achieving them. ...
Tuesday - April 18, 2006, WEST HAVEN, CT and EMERYVILLE, CA Bayer Pharmaceuticals Corporation (NYSE: BAY) and Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) announced today that Nexavar (sorafenib) tabl...
Albireo AB, a biopharmaceutical company specializing in gastroenterology, today announced that the European Medicines Agency Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on an application for orphan medicinal product status for the companys lead hepatology candidate, A4250.
The U.S. Food and Drug Administration has granted a second orphan drug designation to Waltham ophthalmologic device and pharmaceutical developer Avedro Inc. for a treatment of corneal ectasia. The designation relates to VibeX, its riboflavin solution intended for use with UVA irradiation, the KXL system for Corneal Cross-linking, which the company said on its website helps strengthen the cornea following refractive surgery.. SOURCE ...
The Food and Drug Administration on Nov. 21 granted orphan drug designation to rofecoxib (TRM-201), a cyclooxygenase 2-selective nonsteroidal anti-inflammatory
The FDA has granted orphan drug designation to Alvocidib (flavopiridol) to treat patients with acute myeloid leukemia. Alvocidib, a potent c...
Hatfield, England (ots/PRNewswire) - The investigational monoclonal antibody amatuximab (development code: MORAb-009), has today been granted orphan drug designation (ODD) for...
The Food and Drug Admnistration (FDA) has granted Orphan Drug designation for PRM-151 (Promedior) for the treatment of myelofibrosis.
Acceleron Pharma announced that its pulmonary arterial hypertension (PAH) therapy candidate sotatercept was granted orphan drug designation by the FDA.
The Orphan Drug Tax Credit (ODTC) is a key provision of the Orphan Drug Act, and promotes research spending on rare diseases by lowering development costs for manufacturers - but this is in jeopardy: the tax reform bill passed in November by the House would eliminate the credit altogether, and the Senate bill, which passed earlier this month, cut it nearly in half.. The EveryLife Foundation for Rare Diseases, NORD and Global Genes feel that without the Orphan Drug Tax Credit, investment in therapies for rare disease patients is at risk! Before the Orphan Drug Act, manufacturers were often hesitant to invest in developing new treatments for rare diseases because the small patient population made it difficult to recover development costs.. TAKE ACTION NOW to preserve the ODTC by telling Congress why YOU are grateful for it! Use #RareGratitude and put it to good use!. Here are 4 things you can do to show your #RareGratitude. It only takes a few minutes to help 30 million Americans waiting for ...
Guildford, UK, 29 August 2013: ReNeuron is pleased to announce that its ReN003 retinal stem cell therapy candidate for retinitis pigmentosa has achieved a significant regulatory milestone. The therapy has been granted Orphan Drug Designation in both Europe and the US by the European Commission and the Food and Drug Administration, respectively.. Orphan Drug Designation is typically granted to treatments that provide significant benefit to patients with rare diseases that are life-threatening or chronically debilitating. Treatments with this designation benefit from significant commercial and regulatory advantages such as market exclusivity for 10 years from approval in the disease concerned, against other treatments offering no greater therapeutic advantage.. ReNeuron is using its proprietary human retinal progenitor cells as the basis of its ReN003 therapeutic candidate targeting retinitis pigmentosa, a group of hereditary diseases of the eye that lead to progressive loss of sight due to cells ...
Growing number of people suffering orphan diseases boosts the orphan drugs market. Know the Orphan drugs market trend by disease type, product type & others.
Orphan drugs are a relatively niche segment because they are used for the treatment of rare or orphan diseases. Numerous trends are shaping up the orphan drugs market and it becomes imperative for stakeholders to learn about these trends and be prepared for change.
The Orphan Drug Act permits the Secretary of Health and Human Services (the "Secretary") to designate a drug as an orphan drug. According to statute, "[t]he manufacturer or the sponsor of a drug may request the Secretary to designate the drug as a drug for a rare disease or condition." 21 U.S.C. § 360bb(a)(1). The statute further instructs that, if the Secretary finds that the drug "is being or will be investigated for a rare disease or condition" and the approval, certification or licensure of that drug "would be for use for such a disease or condition," the Secretary "shall designate the drug as a drug for such disease or condition." Id. The Food and Drug Administration (FDA), an agency under the control of the Secretary, oversees the designation and approval of orphan drugs.. Distinction between orphan drug designation and approval to market the drug. The designation of a drug as an orphan drug is separate from approval to market the drug in the United States. Even though a drug has been ...
Meet Orphan Drug scientists and Experts in Rare Diseases from USA (America) UK, Europe, Middle East and Asia Pacific countries- Baltimore, Dubai, Spain, Brisbane, Bangalore, Philadelphia, Chicago, Boston, New York, Switzerland, San Antonio at Orphan Drugs conferences and Pharmaceutical Events held from May 15-17, 2017 Munich, Germany
...THE WOODLANDS Texas March 20 2012 /- a href ht... The Orphan Drug designation of telotristat etiprate by the FDA provid...In the U.S. Orphan Drug designation is generally granted for drugs in...,FDA,Grants,Lexicon,Orphan,Drug,Designation,for,Telotristat,Etiprate,for,the,Treatment,of,Carcinoid,Syndrome,biological,advanced biology technology,biology laboratory technology,biology device technology,latest biology technology
GW Pharmaceuticals plc (AIM:GWP) (Nasdaq:GWPH) ("GW") announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Epidiolex®, GWs product candidate that contains plant-derived Cannabidiol (CBD) as its active ingredient, for use in treating children with Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood-onset epilepsy. Epidiolex is an oral liquid formulation of a highly purified extract of CBD, a non-psychoactive molecule from the cannabis plant.. In November 2013, GW announced that the FDA had granted orphan drug designation for Epidiolex in the treatment of Dravet syndrome. Following the successful follow-on offering completed in January 2014, GW intends to advance a full clinical development program for Epidiolex for the treatment of both LGS and Dravet syndromes. Working with leading pediatric epilepsy specialists in the United States, GW expects to commence an initial Phase 2 clinical trial for Epidiolex in the second half of ...
The debate over cost and access to drugs has long raged between patients, health advocates, and pharmaceutical companies. For patients with "orphan diseases," or rare diseases which affect fewer than 200,000 people in the United States, this debate becomes particularly acute, as the Orphan Drug Act passed by Congress in 1983 threatens to drive up prices for highly specialized treatments. While the Orphan Drug Act has helped to bring drugs for rare diseases to millions of patients and continues to stimulate research and development of orphan drugs, the law is certainly not without its problems and caveats.. Continue Reading. ...
Orphan Drug Designation (ODD) in the U.S. means that a drug has been designated by the U.S. FDA as an orphan drug pursuant to the Orphan Drug Act of 1983. This law was passed to facilitate the development of drugs for rare diseases that affect small numbers of people living in the U.S. (which is defined as affecting less than 200,000 people in the U.S.). Orphan drug designation does not mean that the drug is safe or effective, and does not mean that it is legal to market or sell the drug in the U.S. Rather, the designation means only that the drugs sponsor qualifies for certain benefits from the U.S. government, including tax incentives and market exclusivity for 7 years. Orphan drugs normally follow the same regulatory approval process as other drugs (including dosing, safety and efficacy). However, some statistical requirements are lessened (such as not requiring the same number of patients in a Phase III clinical trial as would be required for a non-orphan drug), in order to try to provide ...
The US FDA now gives special "orphan drug" status to potential treatments for rare diseases, changing the approach of regulators and pharma companies
The FDAs Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Orphan designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.. About Pracinostat. Pracinostat is an orally available histone deacetylase (HDAC) inhibitor that has been tested in a number of Phase I and Phase II clinical trials in advanced hematologic disorders and solid tumor indications in both adult and pediatric patients. Pracinostat has been generally well tolerated in more than 200 patients to date, with readily manageable side effects that are often associated with drugs of this class, such as fatigue. In a Phase I dose-escalation trial, Pracinostat demonstrated evidence of single-agent ...
WALTHAM, Mass. - July 21, 2014 - Proteon Therapeutics Inc., a privately held biopharmaceutical company developing novel, first-in-class pharmaceuticals to address the critical medical needs of patients with kidney and vascular diseases, announced today that the first patient has been treated in a pivotal Phase 3 clinical study of its lead product, PRT-201, in chronic kidney disease (CKD) patients undergoing surgical placement of an arteriovenous fistula (AVF). PRT-201, a locally-acting recombinant human elastase, is an investigational drug that may prolong the patency and reduce the failure of hemodialysis vascular access in patients with CKD. PRT-201 has received fast track and orphan drug designations from the U.S. Food and Drug Administration (FDA) and orphan medicinal product designation from the European Commission for hemodialysis vascular access indications.. "Hemodialysis is a life-saving treatment for patients with CKD, but many of these patients suffer from vascular access failure, ...
Due to the promising early results seen in patients with primary brain cancer, Reata has begun clinical trials of RTA 744 in patients who have other types of tumors (for example, lung or breast cancer) that have spread to the central nervous system (CNS). Reata Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing novel treatments for cancer, inflammation, and neurodegenerative diseases. Reata is matching its clinical and preclinical drug development programs with a best-of-class drug discovery platform to identify small molecule chaperones that can induce proper folding of p53, SOD, and Tau, misfolded proteins that are involved in cancer and neurodegenerative disease.
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BONITA, CA--(Marketwired - October 09, 2015) - The combination of PharmaCyte Biotechs (OTCQB: PMCB) signature live-cell encapsulation technology, Cell-in-a-Box® with low doses of ifosfamide has already won the FDAs Orphan Drug status here in the U.S
The disease may manifest in patients with symptoms of chronic pain, difficulty swallowing, poor growth, malnutrition, and weight loss.
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<p>HOBOKEN, N.J., <span class=xn-chron>Aug. 2</span> /CNW/ -- FDA Confirms Orphan Drug Exclusivity Approval for wilate®, Octapharma USAs
New Therapy for Common Inherited Bleeding Disorder.
The Multi-Stakeholder Symposium on Improving Patient Access to Rare Disease Therapies aimed at addressing a crucial bottleneck in making orphan medicinal products (OMP) accessible across Europe.. A unique combination of nearly 300 patient advocates, academics, policymakers, industry representatives, payers and HTA bodies came together to discuss the current state of play and how to shape a more effective way to address value determination, appraisal, pricing and reimbursement of orphan medicines, all with the aim of improving patients access to rare disease therapies throughout Europe.. By bringing together such a varied range of stakeholders, the Symposium created an opportunity for exchange to reach an understanding of the varied perspectives on issues and challenges surrounding patient access. Participants discussed various methods of appraisal, value determination and reimbursement and also took part in pricing simulation exercises.. Speakers included European Commissioner for Health ...
LACHEN, Switzerland, Jan. 13 /PRNewswire/ -- Octapharma Receives Orphan Drug Exclusivity Approval for wilate(R) - A Replacement Therapy Developed...
Copyright ©2020 NORD - National Organization for Rare Disorders, Inc. All rights reserved. NORD is a registered 501(c)(3) charity organization. Please note that NORD provides this information for the benefit of the rare disease community. NORD is not a medical provider or health care facility and thus can neither diagnose any disease or disorder nor endorse or recommend any specific medical treatments. Patients must rely on the personal and individualized medical advice of their qualified health care professionals before seeking any information related to their particular diagnosis, cure or treatment of a condition or disorder. ...
TORONTO, ON--(Marketwired - May 18, 2017) - On Thursday, June 1, 2017, Xtalks will host a complimentary webinar featuring Dr. Carlos Camozzi, Chief Medical Officer and Dr. Fabrice Chartier, Chief Operating Officer, both from Simbec-Orion Group, as the speakers. Discussion topics include:Key challenges to improve cost-efficiency in clinical development of...
Martin A. Makary, MD, MPH, FACS. Professor of Surgery, Johns Hopkins School of Medicine Professor of Health Policy & Management, Johns Hopkins University School of Public Health Chief, Islet Transplant Surgery Johns Hopkins Hospital. "Surgery and the Future of Health Care" - Makary is an advocate for innovation in medicine and physician-led initiatives such as The Surgical Checklist, which he developed at Johns Hopkins, and was later popularized in Atul Gawandes best-selling book Checklist Manifesto.[7] In 2016, Makary and his colleagues exposed loopholes in the Orphan Drug Act accounting for higher drug pricing. His article "The Orphan Drug Act: Restoring the Mission to Rare Diseases",[8] covered by Kaiser Heath News,[9] led Senator Grassleys office to announce an investigation[10] into the problem. Makary has advocated for the need for more transparency in healthcare and argued that professional physician associations are in the best position to define and endorse the validity of quality ...
Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases. They are "orphans" because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.. ...
The U.S. Food and Drug Administration has granted orphan drug designation for San Diego-based Organovos 3D bioprinted tissue treatment of a protein deficiency disease. The designation paves the way for more frequent FDA interactions, tax credits for clinical research costs and the potential for seven years of marketing exclusivity after the drug is approved.. Organovo designs and creates functional, three-dimensional human tissues for use in drug discovery, clinical development and therapeutic applications. The companys NovoTissues is intended to treat alpha-1 antitrypsin deficiency, a condition in which the body does not make enough of a protein that protects the lungs and liver from damage. The condition can lead to, among other things, liver disease. "The FDAs rapid action recognizes the importance of developing regenerative medicine therapeutic applications, and mirrors our own urgency in addressing this devastating disease. With tens of thousands of patients being treated for inborn ...