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Europe’s March 2014 Products Recommended For Orphan Drug Designation

March 17, 2014

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting March 11 – 12, 2014. The EMA COMP March 2014 Meeting Report on the review of applications for orphan designation is published March 14.

At this meeting, there are 6 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 Name
Autologous CD34+   haematopoietic stem cells transduced with lentiviral vector encoding the   human beta A-T87Q-globin gene Bluebird bio France Sickle Cell Disease
Autologous T cells transduced   with lentiviral vector containing a chimeric antigen receptor directed   against CD19 Novartis Europharm Limited B-Lymphoblastic Leukemia/Lymphoma
Genetically modified serotype   5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor Oncos Therapeutics Oy Ovarian Cancer
Synthetic double-stranded   siRNA oligonucleotide directed against transthyretin mRNA and covalently   linked to a ligand containing three N-acetylgalactosamine residues Voisin Consulting S.A.R.L. ATTR Amyloidosis
Humanised monoclonal antibody   against CD38 Sanofi-Aventis Groupe Plasma Cell Myeloma
Ibrutinib Janssen-Cilag International N.V. Lymphoplasmacytic Lymphoma

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

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