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Europe’s October 2014 Products Recommended For Orphan Drug Designation

October 16, 2014

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting October 7 – 9, 2014.

At this meeting, there are 29 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 October 2014 ODDs Recommended

Product Name Sponsor Company Indication
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid Inventiva Systemic Sclerosis
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid Inventiva Idiopathic Pulmonary Fibrosis
4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid Cote Orphan Consulting UK Limited Cystic Fibrosis
combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH Apitope International NV Hemophilia A
Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment Kiadis Pharma Netherlands B.V. Acute Myeloid Leukemia
Imatinib Numedicus Limited Acute Respiratory Distress Syndrome
Mexiletine Hydrochloride Temmler Pharma GmbH Myotonic Disorders
Recombinant human pentraxin-2 FGK Representative Service GmbH post-essential thrombocythaemia myelofibrosis
Recombinant human pentraxin-2 FGK Representative Service GmbH post-polycythaemia vera myelofibrosis
Recombinant human pentraxin-2 FGK Representative Service GmbH primary myelofibrosis
Selinexor Clinipace GmbH Plasma Cell Myeloma
Selinexor Clinipace GmbH chronic lymphocytic leukaemia / small lymphocytic lymphoma
(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4- methyl-3-trifluoromethylphenyl)amide ChemoCentryx Limited microscopic polyangiitis
(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4- methyl-3-trifluoromethylphenyl)amide ChemoCentryx Limited granulomatosis with polyangiitis
(3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate Genzyme Europe BV Gaucher Disease
Adeno-associated viral vector serotype 8 containing the human MD1 gene Généthon Duchenne Muscular Dystrophy
Arimoclomol citrate Orphazyme ApS Niemann-Pick disease
Ataluren PTC Therapeutics Mucopolysaccharidosis type I
Bazedoxifene acetate Consejo Superior de Investigaciones Cientificas (CSIC) hereditary haemorrhagic telangiectasia
Chloroquine DualTpharma B.V. Glioma
Dantrolene sodium Eagle Laboratories Ltd malignant hyperthermia
Diaspirin cross-linked haemoglobin New B Innovation (UK) Limited hepatocellular carcinoma
Humanised IgG1 monoclonal antibody against human eotaxin-2 CBR Biotech Strategies GmbH Systemic Sclerosis
Olaptesed Pegol Noxxon Pharma AG Glioma
Palovarotene Medpace Germany GmbH  fibrodysplasia ossificans progressive
Pentosan polysulfate sodium Plexcera Therapeutics EU Limited mucopolysaccharidosis type I
Pro-Pro-Thr-Val-Pro-Thr-Arg Alain TAIEB xeroderma pigmentosum
Siponimod Novartis Europharm Limited Dermatomyositis
Siponimod Novartis Europharm Limited Polymyositis


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

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