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Europe’s February 2015 Products Recommended For Orphan Drug Designation

March 5, 2015

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting February 10 – 12, 2015.

At this meeting, there are 22 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 February 2015 ODDs Recommended

Product Name Sponsor Company Indication
5′-SASTSCSASG

STSCSTSGSASUSASASGSCSTSA-3′

CTI Clinical Trial & Consulting ServicesEurope GmbH Alport Syndrome
6-ethoxy-7-methoxy-2-(2-methylsulfanylphenyl)-3,1-benzoxazin-4-one Sixera Pharma AB Netherton Syndrome
Human plasma-derived alpha-1 proteinase inhibitor Richardson Associates Regulatory Affairs Ltd Graft-Versus-Host Disease
Recombinant human club cell 10 KDa protein RLM Consulting Prevention of broncho-pulmonary dysplasia
Tideglusib QRC Consultants Ltd Fragile X Syndrome
[5-(5-chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine hydrochloride Daiichi Sankyo Development Ltd Tenosynovial giant cell tumor, localized and diffuse type
5,10,15,20-tetrakis(2,6-difluoro-3-N-methylsulfamoylphenyl)bacteriochlorin Luzitin S.A. Biliary Tract Cancer
Adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene Gauchers Association Gaucher Disease
Autologous adipose tissue-derived stromal vascular fraction cells Assistance Publique Hôpitaux de Marseille Systemic sclerosis
Chimeric 2′-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA Isis USA Ltd Huntington’s Disease
Enoxacin Impasara Ltd Amyotrophic lateral sclerosis
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a COL17A1-encoding retroviral vector Chiesi Farmaceutici S.p.A. Epidermolysis Bullosa
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a COL7A1-encoding retroviral vector Chiesi Farmaceutici S.p.A. Epidermolysis bullosa
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a LAMB3-encoding retroviral vector Chiesi Farmaceutici S.p.A. Epidermolysis bullosa
Gallium (68Ga)-edotreotide for Advanced Accelerator Applications SA Diagnosis ofgastro-entero-pancreatic neuro-endocrine tumors
Human reovirus type 3 Dearing strain Oncolytics Biotech (UK) Limited Ovarian Cancer
Humanized anti-folate receptor 1 monoclonal antibody conjugated to maytansinoid DM4 ImmunoGen Europe Limited Ovarian Cancer
Lenvatinib Eisai Europe Limited Hepatocellular Carcinoma
Melphalan Flufenamide Oncopeptides AB Plasma Cell Myeloma
Recombinant human monoclonal antibody binding to vascular adhesion protein-1 Biotie Therapies Corp Primary Sclerosing Cholangitis
Sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2]dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a]purin-9-one Universitätsklinikum Tübingen (UKT) Retinitis Pigmentosa
Trientine tetrahydrochloride GMP-Orphan SAS Wilson’s Disease

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

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