FDA August 2014 Products Receiving Orphan Designation

 

 

 

 

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The chart below identifies FDA August 2014 Products Receiving Orphan Designation as of 08/31/14 in ascending “Orphan Drug Designation Date” order.

FDA August 2014 Products Receiving Orphan Designation

Name/ ODD Date	Sponsor Company	Indication
Roseburia Hominis/08.04	GT Biologics (UK)	Ulcerative Colitis, pediatric
Anti-Beta1 integrin monoclonal antibody/ 08.07	OncoSynergy	Glioblastoma
N1,N14,-diethyl-3,12,-dihydroxyhomospermine/ 08.07	Sun BioPharma	Pancreatic Cancer
Nivolumab/ 08.07	Bristol-Myers Squibb	Hodgkin  Lymphoma
Dry extract from Betulae Cortex (birch bark)/08.07	Birken AG (Germany)	Epidermolysis Bullosa
Mocetinostat/ 08.07	MethylGene	Diffuse large B-cell lymphoma
Recombinant human deoxyribonuclease I (DNase I)/ 08.18	Guardum Pharmaeuticals	Prevention of  graft -vs-host disease
Immune Globulin Subcutaneous (Human), 20% Liquid/08.18	CSL Behring	Chronic inflammatory demyelinating polyneuropathy
Recombinant human deoxyribonuclease I/ 08.19	Guardum Pharmaceuticals	Treatment of graft vs-host disease
Recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor/ 08.19	Novartis	Pigmented villonodular synovitis, includinggiant celltumor of tendon sheath
Selective inhibitor of fungal lanosterol demethylase/ 08.19	Viamet Pharmaceuticals	Cryptococcal meningitis
Sodium phenylbutyrate/ 08.19	Acer Therapeutics	Maple syrup urine disease
Altiratinib / 08.19	Deciphera Pharmaceuticals	Glioblastoma multiforme
Olaptesed pegol/ 08.19	Noxxon Pharma AG (Germany)	Glioblastoma
Dexrazoxane hydrochloride/ 08.19	Satiscor	Prevention of cardiomyopathy
Cannabidiol/ 08.20	Insys Therapeutics	Glioblastoma
Monoclonal antibody consisting of three mouse/human chimeric IgG1 monoclonal antibodies (c2G4, c4G7, and c13C6) that target Ebola virus/ 08.25	LeafBio, Inc	Ebola virus infection
Oprozomib/ 08.25	Onyx Therapeutics	Waldenstrom’s macro-globulinemia
Recombinant human Pentraxin-2/ 08.26	Promedior	Myelofibrosis
Vemurafenib/ 08.26	Genentech	Hairy cell leukemia
(3S)-1-azabicylo[2.2.2]oct-3yl {2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate/ 08.26	Genzyme	Fabry’s disease
Gefitinib/ 08.26	AstraZeneca	Epidermal growth factor receptor mutation-positive NSCLC
Hematopoietic stem and progenitor cells expanded ex-vivo with a low molecular weight aryl hydrocarbon receptor (AHR) antagonist/ 08.27	Novartis	Hematopoietic support in patients with acute lymphoblastic leukemia
2-{[(1r,4r)-4-(((4-chlorophenyl)(phenyl)carbamoyloxy)methyl)cyclohexy]methoxy} acetic acid/ 08.28	Arena Pharmaceuticals	Pulmonary arterial hypertension
Methotrexate/ 08.28	The Universtiy of Kanasa Medical Center	Myasthenia Gravis

** Generic Name/ODD Date” Column Link = Is the FDA Orphan Drug Product Designation Database Record.

OBSERVATIONS

•   ODD for LeafBio’s Ebola Virus treatment

•   Novartis receives 2 ODDs

•   Approximately 50% are for an oncology indication

•   ODD for several rare diseases:

1.   Epidermolysis Bullosa (EB)

2.   Maple Syrup Urine Disease

3.   Fabry’s Disease

4.   Myasthenia Gravis

5.   Pulmonary Arterial Hypertension (PAH).

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