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FDA May 2015 Products Receiving Orphan Designation

June 2, 2015







The chart below identifies FDA May 2015 Products Receiving Orphan Designation as of 06/02/15 in ascending “Orphan Drug Designation Date” order.

FDA May 2015 Products Receiving Orphan Designation

# Generic Name/ODD Date Sponsor Company Indication
1 Tacrolimus/ 05.04 Stanford University School of Medicine Pulmonary Arterial Hypertension
2 Tadalafil/ 05.04 Eli Lilly & Co Duchenne Muscular Dystrophy
3 Pantothenate Phosphate / 05.04 Retrophin Pantothenate Kinase Associated Neurodegeneration
4 Pelareorep/ 05.04 Oncolytics Biotech Gastric Cancer
5 4-(pyrimidin-2-ylmethyl)-7-[4-(trifluoromethoxy)phenyl]-3,4-dihydro-1,4-benzoxazepin-5(2H)-one/ 05.04 Gilead Sciences Congenital Long QT Syndrome
6 LOAd703, a modified adenovirus 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand (TMZ-CD40L) and full length 4-1BBL/ 05.04 Lokon Pharma AB (Sweden) Pancreatic Cancer
7 Humanized monoclonal antibody that targets the a-subunit of the human IL-3 receptor also known as the CD123 antigen; (JNJ-56022473)/ 05.05 Janssen Research & Development Acute Myeloid Leukemia
8 Autologous tumor cells fused with polyethylene glycol to human allogeneic FO-1, DNA plasmid transfected tumor cell line/ 05.06 Sebastiano Gattoni-Celli, MD Glioblastoma
9 Edaravone/ 05.12 Mitsubishi Tanabe Pharma Corporation (Japan) Amyotrophic Lateral Sclerosis
10 N-t-butylhydroxylamine/ 05.12 Andrew Lim, California Institute of Technology Infantile neuronal ceroid lipofuscinosis
11 Recombinant human alkaline phosphatase/ 05.13 AM-Pharma BV Hypophosphatasia
12 Anti-eTau Humanized IgG4 Monoclonal Antibody / 05.13 Bristol-Myers Squibb Progressive Supranuclear Palsy
13 E-selectin antagonist/ 05.13 GlycoMimetrics Acute Myeloid Leukemia
14 Bruton’s Tyrosine Kinase (Btk) Inhibitor/ 05.13 Acerta Pharma BV (Netherlands) Chronic Lymphocytic Leukemia
15 Hydrocortisone oral granules/ 05.13 Diurnal Limited (UK) Adrenal insufficiency ( 0 – 16 Years of Age)
16 Adalimumab/ 05.13 AbbVie Moderate to severe hidradenitis suppurativa
17 Revusiran/ 05.18 Alnylam Pharmaceuticals Transthyretin amyloidosis
18 Xenon gas / 05.18 Neuroprotexeon (UK) To improve neurological outcome in hospitalized cardiac arrest patients
19 Chloroquine/ 05.20 DualTpharma (Netherlands) Glioblastoma multiforme
20 Lutetium (177Lu)-edotreotide/ 05.21 ITG Isotope Technologies Garching GmbH Gastro-entero-pancreatic neuroendocrine tumors
21 Poly-CD-PEG-camptothecin/ 05.21 Cerulean Pharma Ovarian cancer
22 Echinomycin/ 05.21 OncoImmune Acute Myeloid Leukemia
23 Prasugrel hydrochloride/ 05.26 Eli Lilly Sickle Cell Disease
24 Adult hemogenic endothelial cells/ 05.27 HemoGenyx Aplastic Anemia


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

Please Note: “Nessler Cylinders” by Panek (Own work) [GFDL or CC-BY-3.0 | via Wikimedia Commons.

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