FDA Orphan Drug Designations US October 2014

FDA Orphan Drug Designations – October 2014

In Regulation & Government by CameronLeave a Comment

Find the EMA’s orphan designation and approvals for October here.

FDA – 10/01/2014
Generic Name:  PEGylated recombinant human hyaluronidase PH20
Trade Name:  N/A
Orphan Designation:  Treatment of pancreatic cancer
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Halozyme Therapeutics, Inc.
FDA – 10/01/2014
Generic Name:  Theranost 68 Ga RGD
Trade Name:  N/A
Orphan Designation:  A Diagnostic for the management of Moyamoya disease (MMD)
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Advanced Imaging Projects, LLC (AIP)
FDA – 10/01/2014
Generic Name:  adenovirus delta 24-RGD, an oncolytic adenovirus genetically modified for treatment of glioma
Trade Name:  N/A
Orphan Designation:  Treatment of glioma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  DNAtrix, Inc.
FDA – 10/01/2014
Generic Name:  interferon gamma-1b
Trade Name:  Actimmune
Orphan Designation:  Treatment of Friedriech’s Ataxia
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Horizon Pharma Ireland Limited
FDA – 10/02/2014
Generic Name:  Inhibitor of primary and secondary KIT and PDGFR alpha kinase mutants found in GIST
Trade Name:  N/A
Orphan Designation:  Treatment of gastrointestinal stromal tumors (GIST)
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Deciphera Pharmaceuticals, LLC
FDA – 10/09/2014
Generic Name:  A-dmDT390-bisFv(UCHT1)
Trade Name:  Resimmune
Orphan Designation:  Treatment of cutaneous T-cell lymphoma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Angimmune, LLC
FDA – 10/09/2014
Generic Name:  Combination of nivolumab and ipilimumab
Trade Name:  N/A
Orphan Designation:  Treatment of Stage IIb to Stage IV melanoma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Bristol-Myers Squibb Company
FDA – 10/09/2014
Generic Name:  S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide)
Trade Name:  N/A
Orphan Designation:  Treatment of paroxysmal nocturnal hemoglobinuria (PNH)
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Amyndas Pharmaceuticals
FDA – 10/09/2014
Generic Name:  olaratumab
Trade Name:  N/A
Orphan Designation:  Treatment of soft tissue sarcoma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Eli Lilly and Company
FDA – 10/15/2014
Generic Name:  N-(3,4-dihydroxyphenyl)-3,4-dihydroxybenzamide
Trade Name:  N/A
Orphan Designation:  Treatment of AL Amyloidosis
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  ProteoTech, Inc.
FDA – 10/15/2014
Generic Name:  N-Methanocarbathymidine
Trade Name:  N/A
Orphan Designation:  Treatment of neonatal herpes
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  N & N Pharmaceuticals, Inc.
FDA – 10/15/2014
Generic Name:  diacerein
Trade Name:  N/A
Orphan Designation:  Treatment of epidermolysis bullosa
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  TWI Biotechnology, Inc.
FDA – 10/16/2014
Generic Name:  cardiotrophin-1
Trade Name:  N/A
Orphan Designation:  Prevention of ischemia-reperfusion injury in kidney transplant recipients
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Digna Biotech, S.L.
FDA – 
Generic Name:  [5-Amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxy-propoxy)-phenyl]-methanone
Trade Name:  N/A
Orphan Designation:  Treatment of pancreatic cancer
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Synovo GmbH
FDA – 10/20/2014
Generic Name:  dabrafenib
Trade Name:  Tafinler
Orphan Designation:  Treatment of patients with BRAF mutation positive non-small cell lung cancer
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  GlaxoSmithKline
FDA – 10/20/2014
Generic Name:  ibalizumab
Trade Name:  N/A
Orphan Designation:  Treatment of HIV-1 infection in treatment experienced adult patients with documented multi-antiretroviral class resistance and evidence of HIV-1 replication despite ongoing antiretroviral therapy
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  TaiMed Biologics, Inc.
FDA – 10/20/2014
Generic Name:  isavuconazonium sulfate
Trade Name:  N/A
Orphan Designation:  Treatment of invasive candidiasis/candidemia
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Astellas Pharma Global Development, Inc.
FDA – 10/20/2014
Generic Name:  non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channelrhodopsin-2 protein
Trade Name:  N/A
Orphan Designation:  Treatment of retinitis pigmentosa
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  RetroSense Therapeutics, LLC
FDA – 10/20/2014
Generic Name:  rt-PA
Trade Name:  Activase
Orphan Designation:  Treatment of plastic bronchitis
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Kathleen A Stringer, PharmD, FCCP – Professor
FDA – 10/21/2014
Generic Name:  N-(4-{3-(2-aminopyrimidin-4-yl)pyridin-2-yl]oxy}phenyl)-4-(4-methylthiophen-2-yl)phthalazin-1-amine bis-mesylate dihydrate
Trade Name:  N/A
Orphan Designation:  Treatment of ovarian cancer
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Amgen, Inc.
FDA – 10/21/2014
Generic Name:  autologous bone marrow CD34+ cells transduced ex vivo with a self activating HIV-1 -based lentiviral vector, EFS-ADA
Trade Name:  N/A
Orphan Designation:  Treatment of adenosine deaminase deficient severe combined immunodeficiency
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Donald B. Kohn, MD – Professor, UC-UCLA
FDA – 10/21/2014
Generic Name:  triheptanoin
Trade Name:  N/A
Orphan Designation:  Treatment of glucose transporter type-1 deficiency syndrome
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Ultragenyx Pharmaceutical, Inc.
FDA – 10/22/2014
Generic Name:  autologous adipose derived mesenchymal stromal cells
Trade Name:  N/A
Orphan Designation:  Treatment of amyotrophic lateral sclerosis
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Mayo Clinic
FDA – 10/22/2014
Generic Name:  bi-shRNA furin and GMCSF Autologous Tumor Cell Vaccine
Trade Name:  Fang
Orphan Designation:  Treatment of Ewing’s sarcoma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Gradalis, Inc.
FDA – 10/22/2014
Generic Name:  human trtravalent, bi-specific monoclonal antibody that binds and co-inhibits two growth factor receptors; insulin-like growth factor receptor 1 (IGF-1R) and v-erb-b2 avian erythroblastic leukemia viral oncogene homolog 3 (ErbB3)
Trade Name:  N/A
Orphan Designation:  Treatment of pancreatic cancer
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Merrimack Pharmaceuticals, Inc.
FDA – 10/23/2014
Generic Name:  alpha-1 proteinase inhibitor (human)
Trade Name:  Glassia
Orphan Designation:  Treatment of graft versus host disease
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Kamada Ltd.
FDA – 10/27/2014
Generic Name:  clenbuterol
Trade Name:  Spiropent, Ventipulmin
Orphan Designation:  Adjunctive therapy with enzyme replacement therapy in the treatment of Pompe disease
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Duke University Medical Center
FDA – 10/28/2014
Generic Name:  oprozomib
Trade Name:  N/A
Orphan Designation:  Treatment of multiple myeloma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Onyx Therapeutics, Inc.
FDA – 10/28/2014
Generic Name:  pritumumab
Trade Name:  N/A
Orphan Designation:  Treatment of glioma
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Nascent Biotech, Inc.
FDA – 10/28/2014
Generic Name:  sodium thiosulfate
Trade Name:  N/A
Orphan Designation:  Treatment of dermatomyositis
Orphan Designation Status:  Designated
FDA Orphan Approved:  Not FDA Approved for Orphan Indication
Sponsor:  Hope Pharmaceuticals

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