Orphan Drug Designation

News Orphan Drug Designation

• Aspireo Receives Positive Opinion from EMA for Orphan Disease Designation for Somatoprim

  Aspireo Pharmaceuticals Limited announced that the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) has issued a positive opinion on an application for orphan medicinal product status for Aspireo’s Somatoprim (DG3173) for the treatment of acromegaly.The positive more

• Cytheris announces US Orphan Drug Designation for CYT107 for Progressive Multifocal Leucoencephalopathy

  Cytheris SA announced that the US Food and Drug Administration (FDA) has granted an Orphan Drug Designation for Cytheris’ CYT017, glycosylated recombinant human interleukin-7 (glycosylated r-h-IL7), for the treatment of Progressive Multifocal Leukoencephalopathy (PML). This designation from the FDA more

• Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy

  Prosensa has selected clinical candidates for two more compounds for the treatment of Duchenne muscular dystrophy (DMD) and has been granted orphan drug designation for two additional DMD compounds in its pipeline.Prosensa identified suitable oligonucleotide candidates for PRO052 and PRO055, designe more

Publications Orphan Drug Designation

• Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

  Purpose To assess the methodological quality of Orphan Medicinal Product (OMP) dossiers and discuss possible reasons for the small number of products licensed. Methods Information about orphan drug designation, approval, refusal or withdrawal was obtained from the website of the European Medicines A more

• 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

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