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Europe’s July 2013 Products Receiving Orphan Drug Designation

July 24, 2013

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting July 9 – 11, 2013. The EMA COMP July 2013 Meeting Report on the review of applications for orphan designation is published July 19, 2013.

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

•   Gilead Sciences receives 4 designations for oncology for Idelalisib
•   Celgene receives EMA Orphan Drug Designation (ODD) for Apremilast for Behçet’s Disease, 6 months after receiving FDA ODD
•   Alexion Pharmaceuticals receives EMA ODD for Eculizumab for NMO, 1 month after receiving FDA ODD
•   Approximately 37% of the orphan indications are for oncology, which follows the pattern of orphan drug indications.

At this meeting, there are 19 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) :

  1. Prevention of graft rejection following solid organ transplantation (iReg Medical AB)
  2. Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (Gilead Sciences)
  3. Mucosa-Associated Lymphoid Tissue (MALT Lymphoma) (Gilead Sciences)
  4. Nodal Marginal Zone Lymphoma (Gilead Sciences)
  5. Splenic Marginal Zone Lymphoma (Gilead Sciences)
  6. Osteosarcoma (Richardson Associates Regulatory Affairs Ltd)
  7. Acromegaly (Larode Ltd)
  8. Autosomal Dominant Polycystic Kidney Disease (Otsuka Pharmaceutical Europe)
  9. Acute Myeloid Leukemia (Oryzon Genomics)
  10. Sickle Cell Disease (Pfizer Limited)
  11. Behçet’s Disease (Celgene Europe Limited)
  12. Eosinophilic Oesophagitis (Dr. Falk Pharma GmbH)
  13. Pancreatic Cancer (GANYMED Pharmaceuticals)
  14. Mastocytosis (Lipomed GmbH)
  15. Neuromyelitis Optica (NMO) (Alexion Pharmaceuticals)
  16. Non-Traumatic Ostenonecrosis (Bone Therapeutics)
  17. Pseudomonas Aeruginosa lung infection in Cystic Fibrosis (KaloBios Ltd)
  18. Growth Hormone Deficiency (Larode Ltd)
  19. Congenital Sucrase-Isomaltase Deficiency (QOL Therapeutics EU).

2013 EMA COMP Calendar

  1. September 3 – 4, 2013
  2. October 8 – 9, 2013
  3. November 5 – 6, 2013
  4. December 10 – 11, 2013.

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

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