Europe’s April 2015 Products Recommended For Orphan Drug Designation

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting April 14 – 16, 2015.

At this meeting, there are 13 positive opinions recommending the following medicines for designation as orphan medicinal products. COMP’s opinions are 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.

EMA COMP April 2015 ODDs Recommended

Product Name	Sponsor Company	Indication
{2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide	Right Track Regulatory Limited	Ovarian Cancer
AASSGVSTPGSAGHDIITEQPRS (P42)	Centre National de la Recherche Scientifique (CNRS)	Huntington’s Disease
Adeno-associated viral vector serotype 9 containing the human HGSNAT gene	Cochamo Systems Ltd	Mucopolysaccharidosis IIIC (Sanfilippo C Syndrome)
Fusion proteins composed by a genetically modified cholera toxin subunit A1, peptides from the acetylcholine receptor alpha chain & a dimer of the D fragment from Staphylococcus aureus protein A	Toleranzia AB	Myasthenia Gravis
Reduced oxydised N-acetyl heparin	Sigma-Tau Pharma Ltd	Plasma Cell Myeloma
Triamcinolone acetonide	S-cubed Limited	Non-Infectious uveitis
2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl)acetamide	Clinical Network Services (UK) Ltd	Cystic Fibrosis
5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride	Prana Biotechnology UK Limited	Huntington’s Disease
Adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells	Regulatory Resources Group Ltd	Thromboangiitis obliterans (Buerger’s Disease)
Allopurinol sodium	ACE Pharmaceuticals BV	Perinatal asphyxia
Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1	ImmunoGen Europe Limited	Diffuse large B-cell lymphoma
Trehalose	Dr. Ulrich Granzer	Oculopharyngeal Muscular Dystrophy
Triheptanoin	Pharma Gateway AB	Glucose transporter type-1 deficiency syndrome

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

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