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Europe’s June 2014 Products Recommended For Orphan Drug Designation

June 17, 2014

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting June 10 – 11, 2014. The EMA COMP June 2014 Meeting Report on the review of applications for orphan designation is published June 16th.

At this meeting, there are 16 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
Carboxy pyrrolidine hexanoyl pyrrolidine carboxylate GSK Trading Services Limited AL Amyloidosis
Marizomib Richardson Associates Regulatory Affairs Ltd Plasma Cell Myeloma
Recombinant monoclonal antibody to human serum amyloid P component GSK Trading Services Limited AL amyloidosis
Sodium acetate salt of the synthetic peptide H-D-Ala-Ser-Pro-Met-Leu-Val-Ala-Tyr-Asp-D-Ala-OH Dr Ulrich Granzer Necrotising Soft Tissue Infections
Adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene Fondazione Salvatore Maugeri Clinica del Lavoro e della Riabilitazione Catecholaminergic Polymorphic Ventricular Tachycardia
Cediranib AstraZeneca AB Ovarian Cancer
Cysteamine bitartrate Raptor Pharmaceuticals Europe BV Huntington’s Disease
Eculizumab Alexion Europe SAS Myasthenia Gravis
Humanised anti-alpha ν beta 6 monoclonal antibody Biogen Idec Limited Idiopathic Pulmonary Fibrosis
Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F AbbVie Ltd Glioma
Oxytocin Maïthé Tauber Prader-Willi Syndrome
Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain IDEA Innovative Drug European Associates Limited Beta Thalassaemia Intermedia and Major
Rilotumumab Amgen Europe BV Gastric Cancer
Riociguat Bayer Pharma AG Systemic Sclerosis
Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues Alnylam UK Limited Hemophilia B
Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues Alnylam UK Limited Hemophilia A

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

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