EMA Orphan Drug Designations – November 2014

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See the FDA Orphan Drug designation for October here.

EMA
Drug: Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome
Orphan Designation: Adeno-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA
Sponsor: Lysogene
EMA
Drug: Allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media
Orphan Designation: graft-versus-host disease
Sponsor: Cell2B Advanced Therapeutics
EMA
Drug: Exisulind
Orphan Designation: familial cerebral cavernous malformations;
Sponsor: Molecular Oncology
EMA
Drug: Heat-killed Mycobacterium obuense (whole cell)
Orphan Designation: pancreatic cancer
Sponsor: Immodulon therapeutics
EMA
Drug: Single-chain urokinase plasminogen activator
Orphan Designation: pleural empyema
Sponsor: Cote Orphan Consulting
EMA
Drug: ((E)-1-(4′-chlorophenyl)-3-(4-hydroxy-3-metoxyphenyl)prop-2-en-1-one)
Orphan Designation: WHIM Syndrome
Sponsor:  Centre National de la Recherche Scientifique (CNRS)
EMA
Drug: (1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7-carboxylic acid-L-lysine
Orphan Designation: neurotrophic keratitis
Sponsor: MIMETECH
EMA
Drug: 1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one
Orphan Designation: multiple system atrophy
Sponsor: AstraZeneca
EMA
Drug: 2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one
Orphan Designation: ovarian cancer
Sponsor: Aprea
EMA
Drug: 5,5’-(4-(trifluromethyl)benzylazanediyl)bis(methylene)diquinolin-8-ol
Orphan Designation: glioma
Sponsor: Olivier Blin
EMA
Drug: 5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine
Orphan Designation: malignant mesothelioma
Sponsor: TMC Pharma Services
EMA
Drug: 5-bromo-N-(prop-2-yn-1-yl)-2-(1H-1,2,4-triazol-1-yl) pyrimidine-4,6-diamine
Orphan Designation: Huntington’s disease
Sponsor: Palobiofarma
EMA
Drug: Adenovirus serotype 5 containing partial E1A deletion and an integrin-binding
Orphan Designation: glioma
Sponsor: Alan Boyd Consultants
EMA
Drug: Allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist
Orphan Designation: acute lymphoblastic leukaemia
Sponsor: Novartis Europharm
EMA
Drug: Allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells
Orphan Designation: acute myeloid leukaemia
Sponsor: IPD-Therapeutics BV
EMA
Drug: Allogeneic adipose-derived adult mesenchymal stem cells contained in a fibrin-based bioengineered dermis
Orphan Designation: epidermolysis bullosa
Sponsor: Biodan Yelah
EMA
Drug: Amikacin sulfate for treatment of Pseudomonas aeuriginosa
Orphan Designation: cystic fibrosis
Sponsor: PlumeStars
EMA
Drug: Autologous collagen type II-specific regulatory T cells
Orphan Designation: non-infectious uveitis
Sponsor: TxCell
EMA
Drug: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor
Orphan Designation: diffuse large B cell lymphoma
Sponsor: Kite Pharma
EMA
Drug: Benserazide hydrochloride
Orphan Designation: beta-thalassaemia intermedia and major
Sponsor: Isabelle Ramirez
EMA
Drug: Bevacizumab
Orphan Designation: hereditary haemorrhagic telangiectasia
Sponsor: Dr Sophie Dupuis-Girod
EMA
Drug: Chenodeoxycholic acid
Orphan Designation: inborn errors in primary bile acid synthesi
Sponsor: Sigma-Tau Pharma
EMA
Drug: Edaravone
Orphan Designation: amyotrophic lateral sclerosis
Sponsor: Treeway
EMA
Drug: Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor
Orphan Designation: malignant mesothelioma
Sponsor: Oncos Therapeutics
EMA
Drug: Pegylated recombinant human hyaluronidase PH20
Orphan Designation: pancreatic cancer
Sponsor: Pharm. Research Associates
EMA
Drug: Plerixafor
Orphan Designation: WHIM syndrome
Sponsor: Groupe d’étude des neutropénies
EMA
Drug: Riluzole
Orphan Designation: Traumatic spinal cord injury
Sponsor: Dr Laurent Vinay

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