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Europe’s July 2014 Products Recommended For Orphan Drug Designation

July 22, 2014

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting July 8 – 10, 2014. The EMA COMP July 2014 Meeting Report on the review of applications for orphan designation is published July 22nd.

At this meeting, there are 27 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.

Observations

•   27 ODDs are recommended

•   Vertex Pharmaceuticals’ Lumacaftor and Kalydeco (Ivacaftor) combination for Cystic Fibrosis

•   Genzyme Europe for Lysosomal Storage Disorder (LSD), Fabry Disease

•   Duchenne Muscular Dystrophy indication

•   3 Bleeding Disorder indications:

1.   Hemophilia A

2.   Hemophilia B

3.   Congenital Factor VII Deficiency.

EMA COMP July 2014 ODDs Recommended

Product Name Sponsor Company Indication
(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 Fabry Disease
17α,21-dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione NDA Group AB Duchenne Muscular Dystrophy
Gevokizumab Les Laboratoires Schnitzler Syndrome
Lumacaftor and ivacaftor fixed-dose combination Vertex Pharmaceuticals (UK) Cystic Fibrosis
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin Richardson Associates Regulatory Affairs Ltd Congenital Factor VII Deficiency
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin Richardson Associates Regulatory Affairs Ltd Hemophilia A
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin Richardson Associates Regulatory Affairs Ltd Hemophilia B
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 Myelodysplastic Syndromes
Recombinant human apolipoprotein A-I in a complex with phospholipids Cerenis Therapeutics Holding SA apolipoprotein A-I deficiency
Recombinant human apolipoprotein A-I in a complex with phospholipids Cerenis Therapeutics Holding SA ATP-binding cassette transporter A1 deficiency
Retinol Dr. Philipp Heinrich Novak bronchopulmonary dysplasia
Sodium ascorbate and menadione sodium bisulfite JJG Consultancy Ltd autosomal dominant polycystic liver disease
Ulinastatin BSV BioScience GmbH acute pancreatitis
2-(2-methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide DualT pharma B.V. Small cell lung cancer
4-{[(2R,3S,4R,5S)-4-(4-chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluoro-phenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid Roche Registration Limited Acute Myeloid Leukemia
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene Fondazione Telethon Crigler-Najjar syndrome
Humanised IgG1 monoclonal antibody against human KIR3DL2 Innate Pharma S.A. cutaneous T-cell lymphoma
Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene Centro de Investigación Biomédica en Red (CIBER) pyruvate kinase deficiency
Macromolecular conjugate of heparin sodium on a polymer backbone Corline Systems AB prevention of ischaemia reperfusion injury associated with solid organ transplantation
Obinutuzumab Roche Registration Limited diffuse large B-cell lymphoma
Recombinant human diamine oxidase Medical University of Vienna Mastocytosis
S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide) Amyndas Pharmaceuticals paroxysmal nocturnal haemoglobinuria
Variant of recombinant human fibroblast growth factor 19 Diamond BioPharm Limited Primary biliary cirrhosis
Vector based on an adeno-associated virus serotype 2 backbone, pseudo-serotyped with a type 8 capsid, which carries the coding sequence of the human TYMP gene under the control of the humanthyroxine binding globulin promoter Vall d’Hebron Institute of Research mitochondrial neurogastrointestinal encephalomyopathy
(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N’-(pyrazin-2-yl)acrylohydrazide Clinipace GmbH diffuse large B-cell lymphoma
(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N’-(pyrazin-2-yl)acrylohydrazide Clinipace GmbH acute myeloid leukemia
[5-amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxy-propoxy)-phenyl]-methanone Synovo GmbH Pancreatic Cancer

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

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