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FDA April 2015 Products Receiving Orphan Designation

May 1, 2015





The chart below identifies FDA April 2015 Products Receiving Orphan Designation as of 05/01/15 in ascending “Orphan Drug Designation Date” order.


•   Approximately 57% of the April 2015 FDA ODDs are for an oncology or blood cancer

•   Celgene’s Revlimid (Lenalidomide) gets a 7th oncology FDA ODD for extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue

•   Genentech’s Gazyva (Obinutuzumab) gets a 3rd oncology FDA ODD for follicular lymphoma

•   Two FDA ODDs for uveal melanoma

•   Several FDA ODDs for rare diseases:

1.   Duchenne Muscular Dystrophy (DMD)
2.   Short Bowel Syndrome (SBS)
3.   Sickle Cell Disease (SCD)
4.   Facioscapulohumeral Muscular Dystrophy.

FDA April 2015 Products Receiving Orphan Designation

# Generic Name/ODD Date Sponsor Company Indication
1 Golimumab/ 04.02 Janssen Polyarticular juvenile idiopathic arthritis (0 – 16 yrs of age)
2 C24H28N807S2/ 04.02 Curis Inc. Diffuse large B-cell Lymphoma (DLBCL)
3 PEG-b-PLA micelles containing (1) paclitaxel (2) sirolimus (rapamycin) & (3) tanespimycin (17-AAG)(also known as triolimus) / 04.02 Co-D Therapeutics Angiosarcoma
4 Human insulin (rDNA)/ 04.06 Nutrinia Ltd (Israel) Short bowel syndrome (SBS)
5 Fluciclovine (18F)(anit-1-amino-3-fluorocyclobutane-1-carboxylic acid, labelled with Fluorine-18)/ 04.07 Blue Earth Dianostics (UK) Diagnosis of Glioma
6 Eflornithine HCL/ 04.07 Cancer Prevention Pharmaceuticals Gastric Cancer
7 Modified human papillomavirus capsid protein conjugated to a near infrared dye/ 04.08 Aura Biosciences Uveal melanoma
8 Treosulfan/ 04.08 Medac GmbH (Germany) Conditioning treatment prior to hematopoietic stem cell transplantation (HSCT) in malignant/non-malignant diseases
9 Amantadine hydrochloride/ 04.09 Adamas Pharmaceuticals Levodopa-induced dyskinesia
10 2-Pyrazinecarbonitrile, 5-[[5-2-(3-aminopropoxy)-6-methoxyphenyl]-1H-pyrazol-3-yl]amino] monomesylate monohydrate/ 04.09 Eli Lilly Anal cancer
11 Sodium ascorbate & menadione sodium bisulfite/ 04.09 IC-MedTech Corporation Noninfected painful total joint without mechanical complication
12 [α−N-(2’succinyl-paclitaxel)Thr]-Phe-Phe-Tyr-Gly-Gly-Ser-Arg-Gly-[ε−N-(2’succinyl-paclitaxel)Lys]-Arg-Asn-Asn-Phe-[ε−N-(2’succinyl-paclitaxel)Lys]-Thr-Glu-Glu-Tyr / 04.15 Angiochem (Canada) Breast cancer with brain metastases
13 Pelareorep/ 04.15 Oncolytics Biotech Malignant Glioma
14 Selumetinib/ 04.15 AstraZeneca Uveal melanoma
15 Obinutuzumab/ 04.15 Genentech Follicular Lymphoma
16 Diphenylcyclopropenone gel/ 04.15 RXi Pharmaceuticals Malignant melanoma stage IIB – IV
17 2-[4-[[(2-R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate/ 04.15 Cymabay Therapeutics Frederickson Type I/V hyperlipoprotein- emia
18 Zoledronate D,L-lysine monohydrate (ZLM) / 04.15 Thar Pharmaceuticals Complex regional pain syndrome (CRPS)
19 Triheptanoin/ 04.15 Ultragenyx Pharmaceuticals Fatty acid oxidation disorders
20 177-LU-DOTA-GlyGlyNle-CycMSHhex/ 04.16 SolaranRx Stage IIB – IV malignant melanoma
21 Cantrixil/ 04.20 CanTx Ovarian cancer
22 Allogeneic cardiosphere-derived cells/ 04.21 Capricor Duchenne Muscular Dystrophy
23 Cannabidiol/ 04.22 GW Pharma Neonatal hypoxic ischemic encephalopathy
24 Recombinant human histidyl tRNA synthetase/ 04.22 aTyr Pharma Facioscapulo- humeral muscular dystrophy
25 Synthetic double-stranded (hybridized duplex) ribonucleic acid oligonucleotide specific to hydroxyacid oxidase 1 gene/ 04.22 Dicerna Pharmaceuticals Primary hyperoxaluria type 1
26 Docosahexaenoic acid/ 04.27 Sancilio & Co Sickle cell disease
27 Liposomal P-ethoxy growth factor receptor-bound protein 2 antisense oligonucleotide/ 04.27 Bio-Path Holdings Acute myeloid leukemia
28 Lenalidomide/ 04.29 Celgene Extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue
29 (S)-7-(1-(9H-purin-6-ylamino)ethyl)-6-(3-fluorophenyl)-3-methyl-5H-thiazolo[3,2-a]pyrimidin-5-one/ 04.29 Incyte Hodgkin lymphoma
30 Antroquinonol/ 04.30 Golden Biotechnology Corp (Taiwan) Acute myeloid leukemia


** 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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