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Europe’s June 2013 Products Receiving Orphan Drug Designation

June 26, 2013

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting June 11 – 12, 2013. The EMA COMP June 2013 Meeting Report on the review of applications for orphan designation is published June 20, 2013.

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

•   Two Stem Cell companies developing treatments for ALS
•   Promethera Biosciences receives 6 designations
•   Gilead Sciences receives 2 designations for oncology.

At this meeting, there are 22 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. Amyotrophic Lateral Sclerosis (ALS) (Brainstorm Cell Therapeutics UK)
  2. Pulmonary Alveolar Proteinosis (Serendex ApS)
  3. B-Lymphoblastic Leukemia/Lymphoma (MedImmune)
  4. Pancreatic Cancer (Merck KGaA)
  5. Follicular Lymphoma (Voisin Consulting S.A.R.L.)
  6. Amyotrophic Lateral Sclerosis (ALS) (California Stem Cell (UK))
  7. Malignant Thymomas (TopoTarget A/S)
  8. Plasma Cell Myeloma (Janssen-Cilag International)
  9. Ataxia Telangiectasia (Erydel S.p.A.)
  10. Limbal Stem Cell Deficiency (University Newcastle upon Tyne)
  11. Ovarian Cancer (Diamond BioPharm Limited )
  12. Hyperargininaemia (Promethera Biosciences)
  13. Carbamoyl-Phosphate Synthase-1 Deficiency (Promethera Biosciences)
  14. Ornithine Translocase Deficiency (Promethera Biosciences)
  15. Citrullinaemia Type 2 (Promethera Biosciences)
  16. Argininosuccinic Aciduria (Promethera Biosciences)
  17. Citrullinaemia Type 1 (Promethera Biosciences)
  18. N-Acetylglutamate Synthetase (NAGS) Deficiency (Promethera Biosciences)
  19. Prevention of Ischaemia/Reperfusion injury associated with solid organ transplantation (Borders Technology Management Ltd)
  20. Follicular Lymphoma (Gilead Sciences)
  21. Lymphoplasmacytic Lymphoma (Gilead Sciences)
  22. Prevention of Hepatitis B Re-Infection following liver transplantation (CRO-PharmaNet Services GmbH).

2013 EMA COMP Calendar

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

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

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