Orphan Drug Designations – May 2014

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A list of the Orphan drug designations and approvals by the US Food and Drug Administration in May 2014.

FDA - 06/05/2014
Generic Name:  filanesib
Trade Name:
Orphan Designation:  Treatment of multiple myeloma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Array BioPharma, Inc.
FDA - 06/05/2014
Generic Name:  autologous dendritic cells pulsed with allogeneic tumor cell lysate
Trade Name:
Orphan Designation:  Treatment of malignant mesothelioma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Amphera BV
FDA - 08/05/2014
Generic Name:  ex vivo cultured human mesenchymal stromal cells
Trade Name:
Orphan Designation:
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  iCell Science AB
FDA - 13/05/2014
Generic Name:  adalimumab
Trade Name:  Humira
Orphan Designation:  Treatment of non-infectious intermediate, posterior, or pan-uveitis, or chronic non-infectious anterior uveitis
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  AbbVie, Inc.
FDA - 13/05/2014
Generic Name:  vasoactive intestinal peptide (VIP)-elastin-like peptide (ELP) fusion protein
Trade Name:
Orphan Designation:  Treatment of pulmonary arterial hypertension, WHO Group 1
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  PhaseBio Pharmaceuticals, Inc.
FDA - 14/05/2014
Generic Name:  (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N-(pyrazin-2-yl)acrylohydrazide
Trade Name:
Orphan Designation:  Treatment of diffuse large B-cell lymphoma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Karyopharm Therapeutics, Inc.
FDA - 14/05/2014
Generic Name:  177Lu-tetraxetan-tetulomab
Trade Name:
Orphan Designation:  Treatment of Follicular Lymphoma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Nordic Nanovector AS
FDA - 10/04/2014
Generic Name:
Trade Name:
Orphan Designation:
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:
FDA - 14/05/2014
Generic Name: recombinant humanized monoclonal antibody of the immunoglobulin G1 subclass directed against colony stimulating factor-1 receptor expressed on macrophages
Trade Name:
Orphan Designation:  Treatment of pigmented villonodular synovitis and tenosynovial giant cell tumor
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Genentech, Inc.
FDA - 14/05/2014
Generic Name:  afamelanotide
Trade Name:
Orphan Designation:  Treatment of familial benign chronic pemphigus (Hailey-Hailey disease)
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Clinuvel, Inc.
FDA - 14/05/2014
Generic Name:  [a-N-(2'succinyl-paclitaxel)Thr]-Phe-Phe-Tyr-Gly-Gly-Ser-Arg-Gly-[epsilon-N-(2'succinyl-paclitaxel)Lys]-Arg-Asn-Asn-Phe-[epsilon-N-(2'succinyl-paclitaxel)Lys]-Thr-Glu-Glu-Tyr
Trade Name:
Orphan Designation:  Treatment of glioblastoma multiforme
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Angiochem, Inc.
FDA - 14/05/2014
Generic Name:  menadione sodium bisulfite
Trade Name:
Orphan Designation:  Treatment of autosomal dominant polycystic liver disease
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  IC-MedTech Corporation
FDA - 14/05/2014
Generic Name:  Inecalcitol
Trade Name:
Orphan Designation:  Treatment of chronic lymphocytic leukemia
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Hybrigenics, S.A.
FDA - 14/05/2014
Generic Name: Selinexor; (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N-(pyrazin-2-yl)acrylohydrazide
Trade Name:
Orphan Designation:  Treatment of acute myeloid leukemia
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Karyopharm Therapeutics, Inc.
FDA - 14/05/2014
Generic Name:  Menadione Sodium Bisulfite
Trade Name:
Orphan Designation:  For the treatment of autosomal dominant polycystic kidney disease
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  IC-MedTech Corporation
FDA - 14/05/2014
Generic Name:  Targeted Gastrin 17 Complexed Peptide
Trade Name:
Orphan Designation:  Treatment of pancreatic cancer
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Tyg Oncology Ltd
FDA - 19/05/2014
Generic Name:  recombinant lens epithelium derived growth factor 1-326
Trade Name:
Orphan Designation:  Treatment of retinitis pigmentosa
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Ocugen, Inc
FDA - 19/05/2014
Generic Name:  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
Trade Name:
Orphan Designation:  Treatment of Acute Myeloid Leukemia
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Roche Genentech
FDA - 10/05/2014
Generic Name:  all-cis-docosa-4,7,10,13,16,19-hexaenoic acid
Trade Name:  Retriacyl
Orphan Designation:  Treatment of retinitis pigmentosa
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Natac Pharma, S.L.
FDA - 21/05/2014
Generic Name:  live attenuated bioengineered Listeria monocytogenes immunotherapy
Trade Name:
Orphan Designation:  Treatment of osteosarcoma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Advaxis, Inc.
FDA - 22/05/2014
Generic Name:  humanized immunoglobulin monoclonal antibody against CD38
Trade Name:
Orphan Designation:  Treatment of multiple myeloma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Sanofi-Aventis U.S., LLC

See April 2014′s designation.

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