Topic Orphan Drug Designation

News Orphan Drug Designation

• AiCuris Receives Orphan Drug Designation for Letermovir (AIC246) from FDA

  AiCuris announced that AIC246 (Letermovir), the Company’s inhibitor against the human cytomegalovirus HCMV, has been granted Orphan Drug Designation in the US for the prevention of HCMV viremia and disease in at risk populations. This decision was made on 12 December 2011 by the FDA Office of Orphan more

• Amsterdam Molecular Therapeutics Receives US Orphan Designation for Hemophilia B Gene Therapy

  Amsterdam Molecular Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its gene therapy program for the treatment of hemophilia B. Orphan designation in the U.S. could provide up to seven years market exclusivity on regulatory approval. Orp more

• Australian anti-cancer drug picked as one of the world’s top 10 most promising new drug products in 2011

  Immune System Therapeutics (IST) announced its lead anti-cancer product, MDX-1097, had been recognized as one of Windhover's Top 10 most promising new drug products for 2011. Windhover is a provider of business information to the pharmaceutical, biotechnology, and medical device industries. MDX-1097 more

Publications Orphan Drug Designation

• European regulation on orphan medicinal products: 10 years of experience and future perspectives

  European regulation on orphan medicinal products: 10 years of experience and future perspectives Nature Reviews Drug Discovery 10, 341 (2011). doi:10.1038/nrd3445 Authors: The Committee for Orphan Medicinal Products and the European Medicines Agency Scientific Secretariat In more

• Access to orphan drugs despite poor quality of clinical evidence

  WHAT IS ALREADY KNOWN ABOUT THIS SUBJECT • The clinical evidence level of orphan medicines in European market authorization submissions is low: few randomized controlled trials, short treatment follow‐up, rarely hard clinical endpoints. • The success rate at centralized market authorization more

• Despite surge in orphan drug designations, approvals still lag

  Despite surge in orphan drug designations, approvals still lag Nature Medicine 17, 236 (2011). doi:10.1038/nm0311-236b Author: Monica Heger more

Market studies Orphan Drug Designation

• Neurometabolic Disorders Market to 2017 - Novel Therapies for Rare Diseases such as Gaucher’s and Niemann-Pick Poised to Supersede Existing Therapies

  GBI Research has released its latest research, “Neurometabolic Disorders Market to 2017 - Novel Therapies for Rare Diseases such as Gaucher’s and Niemann-Pick Poised to Supersede Existing Therapies” which provides insights into global neurometabolic disorders market and market forecast until 2017. R more

• Vanda Pharmaceuticals Inc. (VNDA) - Financial and Strategic SWOT Analysis Review

  Vanda Pharmaceuticals Inc. (Vanda), is a biopharmaceutical company, engaged in developing and commercializing clinical-stage drug candidates targeting central nervous system disorders. Recently, the company and its development partner Novartis Pharmaceuticals Corporation have launched Fanapt (iloper more

• Provectus Pharmaceuticals, Inc. (PVCT) - Financial and Strategic SWOT Analysis Review

  Provectus Pharmaceuticals, Inc. (Provectus) is a biotechnology company, that is engaged in the development of oncology and dermatology therapies. The company's product pipeline includes PV-10, an injectable formulation of rose bengal designed to selectively target and destroy cancer cells without ha more

Websites Orphan Drug Designation