EMA Orphan Drug Designations – October 2014

In Regulation & Government by Cameron4 Comments

Find the EMA’s orphan designation and approvals for September here.

EMA – 09/10/2014
Drug: 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid
Orphan Designation: Systemic Sclerosis
Sponsor: Inventiva
EMA – 09/10/2014
Drug: 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid
Orphan Designation: Idiopathic Pulmonary Fibrosis
Sponsor: Inventiva
EMA – 09/10/2014
Drug: 4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid
Orphan Designation: Cystic Fibrosis
Sponsor: Cote Orphan Consulting UK Limited
EMA – 09/10/2014
Drug: 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
Orphan Designation: Hemophilia A
Sponsor: Apitope International NV
EMA – 09/10/2014
Drug: Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment
Orphan Designation: Acute Myeloid Leukemia
Sponsor: Kiadis Pharma Netherlands B.V.
EMA – 09/10/2014
Drug: Imatinib
Orphan Designation: Acute Respiratory Distress Syndrome
Sponsor: Numedicus Limited
EMA – 09/10/2014
Drug: Mexiletine Hydrochloride
Orphan Designation: Myotonic Disorders
Sponsor: Temmler Pharma GmbH
EMA – 09/10/2014
Drug: Recombinant human pentraxin-2
Orphan Designation: post-essential thrombocythaemia myelofibrosis
Sponsor: FGK Representative Service GmbH
EMA – 09/10/2014
Drug: Recombinant human pentraxin-2
Orphan Designation: FGK Representative Service GmbH
Sponsor: post-polycythaemia vera myelofibrosis
EMA – 09/10/2014
Drug: Recombinant human pentraxin-2
Orphan Designation: primary myelofibrosis
Sponsor: FGK Representative Service GmbH
EMA – 09/10/2014
Drug: Selinexor
Orphan Designation: Plasma Cell Myeloma
Sponsor: Clinipace GmbH
EMA – 09/10/2014
Drug: Selinexor
Orphan Designation: chronic lymphocytic leukaemia / small lymphocytic lymphoma
Sponsor: Clinipace GmbH
EMA – 09/10/2014
Drug: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4- methyl-3-trifluoromethylphenyl)amide
Orphan Designation: microscopic polyangiitis
Sponsor: ChemoCentryx Limited
EMA – 09/10/2014
Drug: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4- methyl-3-trifluoromethylphenyl)amide
Orphan Designation: granulomatosis with polyangiitis
Sponsor: ChemoCentryx Limited
EMA – 09/10/2014
Drug: (3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate
Orphan Designation: Gaucher Disease
Sponsor: Genzyme Europe BV
EMA – 09/10/2014
Drug: Adeno-associated viral vector serotype 8 containing the humanMD1 gene
Orphan Designation: Duchenne Muscular Dystrophy
Sponsor: Généthon
EMA – 09/10/2014
Drug: Arimoclomol citrate
Orphan Designation: Niemann-Pick disease
Sponsor: Orphazyme ApS
EMA – 09/10/2014
Drug: Ataluren
Orphan Designation: Mucopolysaccharidosis type I
Sponsor: PTC Therapeutics
EMA – 09/10/2014
Drug: Bazedoxifene acetate
Orphan Designation: hereditary haemorrhagic telangiectasia
Sponsor: Consejo Superior de Investigaciones Cientificas (CSIC)
EMA – 09/10/2014
Drug: Chloroquine
Orphan Designation: Glioma
Sponsor: DualTpharma B.V.
EMA – 09/10/2014
Drug: Dantrolene sodium
Orphan Designation: malignant hyperthermia
Sponsor: Eagle Laboratories Ltd
EMA – 09/10/2014
Drug: Diaspirin cross-linked haemoglobin
Orphan Designation: hepatocellular carcinoma
Sponsor: New B Innovation (UK) Limited
EMA – 09/10/2014
Drug: Humanised IgG1 monoclonal antibody against human eotaxin-2
Orphan Designation: Systemic Sclerosis
Sponsor: CBR Biotech Strategies GmbH
EMA – 09/10/2014
Drug: Olaptesed Pegol
Orphan Designation: Glioma
Sponsor: Noxxon Pharma AG
EMA – 09/10/2014
Drug: Palovarotene
Orphan Designation: fibrodysplasia ossificans progressive
Sponsor: Medpace Germany GmbH
EMA – 09/10/2014
Drug: Pentosan polysulfate sodium
Orphan Designation: mucopolysaccharidosis type I
Sponsor: Plexcera Therapeutics EU Limited
EMA – 09/10/2014
Drug: Pro-Pro-Thr-Val-Pro-Thr-Arg
Orphan Designation: xeroderma pigmentosum
Sponsor: Alain TAIEB
EMA – 09/10/2014
Drug: Siponimod
Orphan Designation: Dermatomyositis
Sponsor: Novartis Europharm Limited
EMA – 09/10/2014
Drug: Siponimod
Orphan Designation: Polymyositis
Sponsor: Novartis Europharm Limited

Comments

  1. David Bennett

    Hello
    Much appreciate your summary of OD designations. One in particular interests me: Pentosan polysulfate sodium for MPS1. I can’t find this designation on the EMA site. Could you point me in the right direction?
    Best regards
    David

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