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FDA August 2014 Products Receiving Orphan Designation

September 1, 2014






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



Anti-Beta1 integrin monoclonal antibody/ 08.07 OncoSynergy Glioblastoma
N1,N14,-diethyl-3,12,-dihydroxyhomospermine/ 08.07 Sun BioPharma Pancreatic


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



Recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor/ 08.19 Novartis Pigmented villonodular synovitis, includinggiant celltumor of



Selective inhibitor of fungal lanosterol demethylase/ 08.19 Viamet Pharmaceuticals Cryptococcal meningitis
Sodium phenylbutyrate/ 08.19 Acer Therapeutics Maple




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


Gefitinib/ 08.26 AstraZeneca Epidermal growth


receptor mutation-positive


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.


•   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).

Please Note: “Two small test tubes held in spring clamps” courtesy of  Amitchell125 at English Wikipedia [CC-BY-SA-3.0] | Wikimedia Commons.

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