Europe’s May 2014 Products Recommended For Orphan Drug Designation

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Orphan Druganaut Blog

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting May 13 – 14, 2014. The EMA COMP May 2014 Meeting Report on the review of applications for orphan designation is published May 16.

At this meeting, there are 10 positive opinions recommending the following medicines for designation as orphan medicinal products. COMP’s opinions are then forwarded to the European Commission (EC). The EC will then decide whether to grant an orphan designation for the medicines in question. Public summaries of the opinions will be available on the EMA website following adoption of the respective decisions on orphan designation by the EC :

 Product Name Sponsor Company Indication
1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropanecarboxamide Vertex Pharmaceuticals Cystic Fibrosis
Isavuconazonium sulfate Fondazione Telethon Stargardt’s Disease
Mixture of two adeno-associated viral vectors serotype 8 containing the 5’-half sequence of human MYO7A gene and the 3’-half sequence of human MYO7A gene Fondazione Telethon Usher Syndrome
Adeno-associated viral vector…

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