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Europe’s May 2014 Products Recommended For Orphan Drug Designation

May 17, 2014

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 serotype 2 containing the human REP1 gene NightstaRx Ltd Choroideraemia
Afamelanotide Clinuvel UK Limited Familial benign chronic pemphigus (Hailey-Hailey disease)
Beloranib Dr Ulrich Granzer Prader-Willi Syndrome
Humanised Fc engineered monoclonal antibody against CDl9 MorphoSys AG Chronic lymphocytic leukaemia /small lymphocytic lymphoma
Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene Cochamo Systems Ltd Mucopolysaccharidosis IIIA (Sanfilippo A syndrome)
Norursodeoxycholic acid Dr Falk Pharma GmbH Primary sclerosing cholangitis
Recombinant human alpha-1-microglobulin A1M Pharma AB Pre-eclampsia


Please Note: “Erlenmeyer Flasks” From Argonne US National Lab  [Public domain in the US] | Wikimedia Commons.

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