Europe’s November 2013 Products Receiving Orphan Drug Designation
The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting November 5 – 6, 2013. The EMA COMP November 2013 Meeting on the review of applications for orphan designation is published November 12.
Some observations about the positive opinions on the recommendations for orphan designation :
• Lumena receives 4 EMA orphan drug designations for the same 4 rare cholestatic liver disease indications as the company receives in September 2013 by the FDA
• Novoteris receives an orphan designation for Nitric Oxide for the treatment of Cystic Fibrosis
• Chugai Pharma Europe LTD receives an orphan designation for the treatment of Hemophilia A.
At this meeting, there are 13 positive opinions recommending orphan designation for the treatment of (public summaries of the opinions will be available on the EMA website following adoption of the respective decisions on orphan designation by the European Commission) :
• Glioma (ERC Belgium)
• Follicular Lymphoma (Janssen-Cilag International)
• Necrotising Enterocolitis (Laboratorio Farmaceutico S.I.T. s.r.l.)
• Hypoparathyroidism (NPS Pharma UK)
• Primary Biliary Cirrhosis (Lumena Pharma UK)
• Alagille Syndrome (Lumena Pharma UK)
• Primary Sclerosing Cholangitis (Lumena Pharma UK)
• Progressive Familial Intrahepatic Cholestatis (Lumena Pharma UK)
• Dravet Syndrome (Brabant Pharma)
• Hemophilia A (Chugai Pharma Europe)
• Cystic Fibrosis (Novoteris)
• Dengue Fever (Cote Orphan Consulting UK)
• Arteriovenous Access Dysfunction (Proteon Therapeutics).
Please Note: “Interferon” courtesy of Linda Bartlett (Photographer) from the National Cancer Institute (NCI). [Public domain in the US].