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Europe’s December 2014 Products Recommended For Orphan Drug Designation

December 20, 2014

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting December 9 – 11, 2014.

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 December 2014 ODDs Recommended

Product Name Sponsor Company Indication
Lentiviral vector pseudotyped by the Indiana serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins Theravectys Adult T-cell leukaemia/lymphoma
Lentiviral vector pseudotyped by the New-Jersey serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins Theravectys Adult T-cell leukaemia/lymphoma
Adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter  Magnus Invention Management Ltd Placental insufficiency
Allogeneic, umbilical cord blood-derived, ex vivo-expanded, haematopoietic CD133+ cells / allogeneic, umbilical cord blood-derived, non-expanded, haematopoietic CD133- cells Regulatory Resources Group Ltd Acute myeloid leukaemia
Chimeric monoclonal antibody to O-acetyl-GD2 antigen Atlab Pharma SAS Neuroblastoma
Emtricitabine Dr. Yanick Crow Aicardi-Goutières syndrome
Herpes simplex type 1 virus containing cellular B-myb gene as tumour-specific promoter Karcinolys S.A.S Pancreatic Cancer
Pentosan polysulfate sodium Dr Ulrich Granzer Interstitial cystitis
Recombinant human aspartylglucosaminidase ACE Biosciences A/S Aspartylglucosaminuria
Sodium thiosulfate Hope Pharmaceuticals Calciphylaxis
Tenofovir disoproxil fumarate Dr. Yanick Crow Aicardi-Goutières syndrome
Adeno-associated viral vector serotype 8 containing the human factor-VII gene Professor Edward G. Tuddenham Congenital factor VII deficiency
Allogeneic peripheral blood mononuclear cells induced to an early apoptotic state Richardson Associates Regulatory Affairs Ltd Prevention of graft-versus-host disease
Ceftriaxone Ospedale San Raffaele s.r.l. Spinocerebellar ataxia
Chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 BioMarin Europe Ltd. Mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Humanised Fc engineered monoclonal antibody against CD19 MorphoSys AG Diffuse large B-cell lymphoma
N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride TMC Pharma Services Ltd Ovarian Cancer
Pegylated recombinant arginine deiminase Designerx Europe Limited Malignant mesothelioma
Ponatinib hydrochloride Ariad Pharma Ltd Gastrointestinal stromal tumours
Recombinant human alkaline phosphatase AM-Pharma BV Hypophosphatasia
Sodium valproate Alan Boyd Consultants Ltd Wolfram syndrome
Synthetic signal peptide of human Mucin-1 (amino acids 1-21) Richardson Associates Regulatory Affairs Ltd Plasma cell myeloma

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

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