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FDA March 2015 Products Receiving Orphan Designation

April 2, 2015





The chart below identifies FDA March 2015 Products Receiving Orphan Designation as of 04/02/15 in ascending “Orphan Drug Designation Date” order.

FDA March 2015 Products Receiving Orphan Designation

# Generic Name/ODD Date Sponsor Company Indication
1  Alpha1-proteinase inhibitor (Human)/ 03.03 Grifols Therapeutics Type 1 diabetes mellitus with residual beta-cell function
2 Amifampridine phosphate/ 03.03 Catalyst Pharmaceuticals Congenital myasthenic syndromes
3 Dacomitinib/ 03.03 Pfizer NSCLC with EGFR, HER2, HER4, or DDR2 mutations
4 Recombinant activated Factor X variant/ 03.03 Pfizer Intracerebral hemorrhage
5 Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg](CH3COO)/ 03.09 Alsonex Pty Ltd (Australia) ALS
6 Autologous Epstein-Barr virus specific T-cells/ 03.09 Cell Medica Epstein-Barr virus positive non-Hodgkin lymphomas
7 Lebrikizumab/ 03.09 Genentech IPF
8 Edaravone/ 03.12 Treeway B.V. (Netherlands) ALS
9 Ponatinib/ 03.12 ARIAD Pharmaceuticals Gastrointestinal stromal tumors (GIST)
10 Hydroxyurea/ 03.16 Ebelle D’Ebelle Pharmaceuticals Symptomatic sickle cell disease, pediatric (< 17)
11 Naproxcinod/ 03.16 Nicox SA (France) DMD
12 Pegylated Human recombinant arginase I/ 03.16 AERase Inc. Arginase I deficiency
13 Phenol, 4-[ 2-(aminomethyl)-4-thiazolyl]-2,6-bis  (1,1-dimethyethyl) monohydrochloride/ 03.16 Ipsen Bio-pharmaceuticals Huntington’s Disease
14 Melphalan flufenamide hydrochloride/ 03.16 Oncopeptides AB (Sweden) Multiple Myeloma
15 Tacrolimus/ 03.16 Selten Pharma PAH
16 Tetra-substituted porphyrin derivative containing manganese (III)/ 03.16 Aeolus Pharmaceuticals IPF
17 Sevuparin/ 03.17 Dilaforette AB Sickle cell disease
18 Polyethylene glycol modified recombinant C-terminal truncate of human cystathionine beta-synthase / 03.17 Orphan Technologies Limited (British Virgin Islands) Homocystinuria
19 2-[4-[[(2R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propy]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate/ 03.18 CymaBay Therapeutics HoFH
20 Adeno-associated viral vector type 2 expressing human recombinant retinal pigment epithelial 65KDa protein gene / 03.18 Spark Therapeutics Retinitis Pigmentosa due to autosomal recessive RPE65 gene mutation
21 Hydrocortisone modified release capsules/ 03.18 Diurnal Limited (UK) Congenital adrenal hyperpasia
22 Masitinib mesylate/ 03.18 AB Science (France) ALS
23 Methotrexate oral liquid formulation/ 03.18 Chesapeake Therapeutics ALL (pediatric, 0 – 16)
24 (Monomethoxypolyethylene glycol) recombinant adenosine deaminase/ 03.19 Sigma-Tau Pharmaceuticals Adenosine deaminase deficiency in patients with SCID
25 Cladribine/ 03.19 Chord Therapeutics Neuromyelitis optica & neuromyelitis optica spectrum disorder
26 Reduced Oxidized N-acetyl heparin/ 03.19 Sigma-Tau Research (Switzerland) Multiple Myeloma
27 Sonidegib/ 03.23 Novartis Medulloblastoma
28 4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid/ 03.24 Clanotech AB (Sweden) Scarring post ab externo glaucoma surgery
29 Lactobacillus acidophilus & Bifidobacterium animalis subsp. lactis/ 03.24 Sigma-Tau Pharmaceuticals Prevention of necrotizing enterocolitis in premature infants
30 Live-attenuated Listeria monocytogenes actA/inIB/human mesothelin (hMeso) Strain hMeso38/ 03.24 Aduro Biotech Mesothelioma
31 Allosteric activator of red blood cell-specific form of pyruvate kinase/ 03.24 Agios Pharmaceuticals Pyruvate kinase deficiency
32 Ganaxolone / 03.24 Marinus Pharmaceuticals Protocadherin 19 (PCDH19) female epilepsy
33 Amylopectin / 03.24 Michael K. Davis, Nemours Children’s Clinic Glycogen storage diseases types Ia & Ib
34 Avicin d/ 03.24 Avicin Therapeutics Multiple Myeloma
35 Mouse-human chimeric antibody of the IgG1 isotype, directed against human CD37/ 03.24 Boehringer Ingelheim Pharmaceuticals Chronic Lymphocytic Leukemia
36 Bardoxolone/ 03.30 Reata Pharmaceuticals PAH
37 Dalbavancin/ 03.30 Durata Therapeutics (Netherlands) Acute osteomyelitis (pediatrics, 0 – 16)
38 5’-CTATCTGUC*G1TTCTCTGU-3’.17 Na+/ 03.31 Idera Pharmaceuticals Diffuse Large B-cell lymphoma
39 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid/ 03.31 Inventiva Pharma (France) Systemic sclerosis
40 Synthetic 47 amino acid-long N-terminally nyristoylated, HBV-L-protein derived lipopeptide/ 03.31 MYR GmbH (Germany) Hepatitis D
41 Bryostatin 1/ 03.31 Neurotrope BioScience Fragile X Syndrome


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

Please Note : “Two small test tubes held in spring clamps” courtesy of  Amitchell125 at English Wikipedia [CC-BY-SA-30] | Wikimedia Commons.

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