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Europe’s September 2013 Products Receiving Orphan Drug Designation

September 10, 2013

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting September 3 – 4, 2013. The EMA COMP September 2013 Meeting Report on the review of applications for orphan designation is published September 9, 2013.

Some observations about the positive opinions on the recommendations for orphan designation :

•   ProQR Therapeutics, located in the Netherlands, receives orphan designation for an antisense oligonucleotide targeting the F508delta mutation of CFTR for the treatment of Cystic Fibrosis

•   NicOx, a French company, receives orphan designation for drug Naproxcinod for Duchenne Muscular Dystrophy (DMD); NicOx presented pre-clinical results for Naproxcinod in models of DMD at the MDA Scientific Conference in April 2013

•   Genethon, a French company that designs gene therapy products for rare diseases, receives orphan designation for a gene therapy for the rare disease Wiskott-Aldrich Syndrome (WAS).

At this meeting, there are 9 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) :

•   Congenital Factor VII Deficiency (CSL Behring GmbH)
•   Plasma Cell Myeloma (Patrys GmbH)
•   Sarcoidosis (Araim Pharma Europe Ltd)
•   Myotonic Disorders (Agenzia Industrie Difesa-Stabilimento Chimico Farmaceutico Militare)
•   Allan-Herndon-Dudley Syndrome (CATS Consultants GmbH)
•   Cystic Fibrosis (ProQR Therapeutics)
•   Duchenne Muscular Dystrophy (NicOx)
•   Wiskott-Aldrich Syndrome (Genethon)
•   Complex Regional Pain Syndrome (Axsome Therapeutics).

2013 EMA COMP Calendar

  1. October 8 – 9, 2013
  2. November 5 – 6, 2013
  3. December 10 – 11, 2013.

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

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