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Europe’s November 2014 Products Recommended For Orphan Drug Designation

November 22, 2014

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting November 11– 13, 2014.

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.

EMA COMP November 2014 ODDs Recommended

Product Name Sponsor Company Indication
Adeno-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA Lysogene Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
Exisulind Cell2B Advanced Therapeutics Prevention of graft-versus-host disease
4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid Firc Institute of Molecular Oncology (IFOM) Familial cerebral cavernous malformations
Heat-killed Mycobacterium obuense (whole cell) Immodulon Therapeutics Ltd Pancreatic Cancer
Single-chain urokinase plasminogen activator Coté Orphan Consulting UK Limited Pleural empyema
((E)-1-(4′-chlorophenyl)-3-(4-hydroxy-3-metoxyphenyl)prop-2-en-1-one) Centre National de la Recherche Scientifique (CNRS) WHIM syndrome
(1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7-carboxylic acid-L-lysine MIMETECH S.r.l. Neurotrophic keratitis
1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one AstraZeneca AB Multiple system atrophy
2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one Aprea AB Ovarian Cancer
5,5’-(4-(trifluromethyl)benzylazanediyl)bis(methylene)diquinolin-8-ol Prof. Olivier Blin Glioma
5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine TMC Pharma Services Ltd Malignant mesothelioma
5-bromo-N-(prop-2-yn-1-yl)-2-(1H-1,2,4-triazol-1-yl) pyrimidine-4,6-diamine Palobiofarma S.L. Huntington’s disease
Adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain Alan Boyd Consultants Ltd Glioma
Allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist Novartis Europharm Limited acute lymphoblastic leukaemia
Allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells IPD-Therapeutics BV acute myeloid leukaemia
Allogeneic adipose-derived adult mesenchymal stem cells contained in a fibrin-based bioengineered dermis Biodan Yelah S.L. Epidermolysis bullosa (EB)
Amikacin sulfate PlumeStars s.r.l. Pseudomonas aeuriginosa lung infections in cystic fibrosis
Autologous collagen type II-specific regulatory T cells TxCell Non-infectious uveitis
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor Kite Pharma UK, Ltd Diffuse large B cell lymphoma
Benserazide hydrochloride Isabelle Ramirez Beta-thalassaemia intermedia and major
Bevacizumab Dr Sophie Dupuis-Girod Hereditary haemorrhagic telangiectasia
Chenodeoxycholic acid Sigma-Tau Pharma Ltd Inborn errors in primary bile acid synthesis
Edaravone Treeway B.V. Amyotrophic lateral sclerosis
Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor Oncos Therapeutics Oy Malignant mesothelioma
Pegylated recombinant human hyaluronidase PH20 Pharm. Research Associates (UK) Limited Pancreatic cancer
Plerixafor Groupe d’étude des neutropénies WHIM syndrome
Riluzole Dr Laurent Vinay Traumatic spinal cord injury


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

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