FDA Orphan Drug Designations – August 2014

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Find the FDA’s orphan designation and approvals for July here.

FDA – /08/2014
Generic Name: Roseburia hominis
Trade Name: Rosburix
Orphan Designation: Treatment of ulcerative colitis in pediatric patients age 0 through 16 years
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: GT Biologics Ltd
FDA – 07/08/2014
Generic Name: Anti-Beta1 integrin monoclonal antibody
Trade Name: N/A
Orphan Designation: Treatment of glioblastoma
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: OncoSynergy, Inc.
FDA – 07/08/2014
Generic Name: N1,N14,-diethyl-3,12,-dihydroxyhomospermine
Trade Name: N/A
Orphan Designation: Treatment of pancreatic cancer
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Sun BioPharma, Inc.
FDA – 07/08/2014
Generic Name: dry extract from Betulae Cortex (birch bark)
Trade Name: N/A
Orphan Designation: Treatment of epidermolysis bullosa
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Birken AG
FDA – 07/08/2014
Generic Name: mocetinostat
Trade Name: N/A
Orphan Designation: Treatment of diffuse large B-cell lymphoma.
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: MethylGene, Inc.
FDA – 07/08/2014
Generic Name: nivolumab
Trade Name: N/A
Orphan Designation: Treatment of Hodgkin lymphoma
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Bristol-Myers Squibb Co.
FDA – 18/08/2014
Generic Name: Immune Globulin Subcutaneous (Human), 20% Liquid
Trade Name: Hizentra(R)
Orphan Designation: Treatment of chronic inflammatory demyelinating polyneuropathy (CIDP)
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: CSL Behring
FDA – 18/08/2014
Generic Name: recombinant human deoxyribonuclease I (DNase I)
Trade Name: N/A
Orphan Designation: Prevention of graft-vs-host disease
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Guardum Pharmaeuticals, LLC
FDA – /08/2014
Generic Name: altiratinib
Trade Name: N/A
Orphan Designation: Treatment of glioblastoma multiforme.
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Deciphera Pharmaceuticals, LLC
FDA – 19/08/2014
Generic Name: dexrazoxane hydrochloride
Trade Name: N/A
Orphan Designation: Prevention of cardiomyopathy for chilren and adolescents 0 through 16 years of age treated with anthracyclines
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Satiscor, LLC
FDA – /08/2014
Generic Name: olaptesed pegol
Trade Name: N/A
Orphan Designation: Treatment of glioblastoma in conjunction with radiotherapy.
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Noxxon Pharma AG
FDA – 19/08/2014
Generic Name: recombinant human deoxyribonuclease I
Trade Name: N/A
Orphan Designation: Treatment of graft-vs-host disease
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Guardum Pharmaceuticals, LLC
FDA – 19/08/2014
Generic Name: recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor
Trade Name: N/A
Orphan Designation: Treatment of pigmented villonodular synovitis, including giant cell tumor of the tendon sheath
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Novartis Pharmaceuticals Corp
FDA – 19/08/2014
Generic Name: selective inhibitor of fungal lanosterol demethylase
Trade Name: N/A
Orphan Designation: Treatment of cryptococcal meningitis
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Viamet Pharmaceuticals, Inc.
FDA – 19/08/2014
Generic Name: sodium phenylbutyrate
Trade Name: N/A
Orphan Designation: Treatment of maple syrup urine disease
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Acer Therapeutics, Inc.
FDA – 20/08/2014
Generic Name: cannabidiol
Trade Name: N/A
Orphan Designation: Treatment of glioblastoma multiforme
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Insys Therapeutics, Inc.
FDA – 25/08/2014
Generic Name: monoclonal antibody consisting of three mouse/human chimeric IgG1 monoclonal antibodies (c2G4, c4G7, and c13C6) that target Ebola virus
Trade Name: N/A
Orphan Designation: Treatment of Ebola virus infection
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: LeafBio, Inc.
FDA – 25/08/2014
Generic Name: oprozomib
Trade Name: N/A
Orphan Designation: Treatment of Waldenstrom’s macroglobulinemia
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Onyx Therapeutics, Inc.
FDA – 26/08/2014
Generic Name: (3S)-1-azabicylo[2.2.2]oct-3yl {2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate
Trade Name: N/A
Orphan Designation: Treatment of Fabry’s disease
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Genzyme Corporation
FDA – 26/08/2014
Generic Name: gefitinib
Trade Name: N/A
Orphan Designation: Treatment of epidermal growth factor receptor mutation-positive non-small cell lung cancer.
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: AstraZeneca Pharmaceuticals LP
FDA – 26/08/2014
Generic Name: recombinant human Pentraxin-2
Trade Name: N/A
Orphan Designation: Treatment of myelofibrosis
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Promedior, Inc.
FDA – 26/08/2014
Generic Name: vemurafenib
Trade Name: N/A
Orphan Designation: Treatment of hairy cell leukemia (HCL)
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Genentech, Inc.
FDA – 27/08/2014
Generic Name: hematopoietic stem and progenitor cells expanded ex-vivo with a low molecular weight aryl hydrocarbon receptor (AHR) antagonist
Trade Name: N/A
Orphan Designation: Hematopoietic support in patients with acute lymphoblastic leukemia (ALL)
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Novartis Pharmaceuticals Corporation
FDA – 28/08/2014
Generic Name: 2-{[(1r,4r)-4-(((4-chlorophenyl)(phenyl)carbamoyloxy)methyl)cyclohexy]methoxy} acetic acid
Trade Name: N/A
Orphan Designation: Treatment of pulmonary arterial hypertension (PAH)
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Arena Pharmaceuticals, Inc.
FDA – 28/08/2014
Generic Name: Methotrexate
Trade Name: Amethopterin
Orphan Designation: Treatment of Myasthenia Gravis
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: The Universtiy of Kanasa Medical Center

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