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Europe’s February 2014 Products Recommended For Orphan Drug Designation

February 20, 2014

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting February 4 – 6, 2014. The EMA COMP February 2014 Meeting Report on the review of applications for orphan designation is published February 20.

At this meeting, there are 13 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.

  Indication Sponsor Company Product Name
bronchopulmonary dysplasia Viridian Pharma Caffeine citrate
Pancreatic Cancer Raptor Pharmaceuticals Europe Cysteamine Bitartrate
Prevention of Graft Rejection following Solid Organ Transplantation Alexion Europe Eculizumab
Prevention of Graft Rejection following Solid Organ Transplantation iCell Science AB Ex-vivo-cultured human mesenchymal stromal cells
ATTR-Amyloidosis Isis USA Phosphorothioate oligonucleotide targeted to transthyretin
Glycogen Storage Disease Type II (Pompe’s Disease) Genzyme Europe Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic   bismannose-6-phosphate-tetra-mannose glycan
Prevention of Bronchopulmonary Dysplasia Dr Ulrich Granzer Recombinant human surfactant protein D
Leber’s Congenital Amaurosis Fondazione Telethon Adeno-associated viral vector serotype 8 containing the human GUCY2D gene
Nontuberculous Mycobacterial Lung Disease Insmed Limited Amikacin sulfate
Recombination-Activating Gene 1 Deficient Severe Combined   Immunodeficiency Prof. F.J.T. Staal Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene
Soft Tissue Sarcoma Eudax Srl Doxorubicin(6-maleimidocaproyl)hydrazone
Charcot-Marie-Tooth Disease Type 1A Pharnext SAS Fixed-dose combination of (R-S) baclofen, naltrexone hydrochloride and D-sorbitol
Acute Myeloid Leukemia Boehringer Ingelheim International Volasertib


Please Note: “Cambio de color a diferente pHs” by Rubashkyn (Own work) [CC-BY-SA-3.0] | Wikimedia Commons.

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