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FDA October 2014 Products Receiving Orphan Designation

November 1, 2014





The chart below identifies FDA October 2014 Products Receiving Orphan Designation as of 10/31/14 in ascending “Orphan Drug Designation Date” order.

FDA October 2014 Products Receiving Orphan Designation

# Generic Name/ODD Date Sponsor Company Indication
1 Theranost 68 Ga RGD/ 10.01 Advanced Imaging Projects, LLC Moyamoya disease (MMD)
2 PEGylated recombinant human hyaluronidase PH20/ 10.01 Halozyme Therapeutics Pancreatic Cancer
3 Adenovirus delta 24-RGD, an oncolytic adenovirus genetically modified for treatment of glioma/ 10.01 DNAtrux Inc. Glioma
4 Interferon gamma-1b/ 10.01 Horizon Pharma Ireland Limited (Ireland) Friedriech’s Ataxia
5 Inhibitor of primary and secondary KIT and PDGFR alpha kinase mutants found in GIST/ 10.02 Deciphera Pharmaceuticals Gastrointestinal stromal tumors (GIST)
6 Olaratumab/ 10.09 Eli Lilly Soft Tissue Sarcoma
7 S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide)/ 10.09 Amyndas Pharmaceuticals Paroxysmal nocturnal hemoglobinuria (PNH)
8 Nivolumab + Ipilimumab/ 10.02 Bristol-Myers Squibb Melanoma
9 A-dmDT390-bisFv(UCHT1)/ 10.09 Angimmune LLC Cutaneous T-cell lymphoma
10 N-Methanocarbathymidine/ 10.15 N & N Pharmaceuticals Neonatal Herpes
11 N-(3,4-dihydroxyphenyl)-3,4-dihydroxybenzamide/ 10.15 ProteoTech Inc. AL Amlyoidosis
12 Diacerein/ 10.15 TWI Biotechnology Inc. (Taiwan) Epidermolysis bullosa
13 Cardiotrophin-1/ 10.16 Digna Biotech S.L. (Spain) Ischemia-reperfusion injury in kidney transplant recipients
14 Tipelukast/ 10.20 MediciNova Inc. Idiopathic Pulmonary Fibrosis (IPF)
15 Non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channelrhodopsin-2 protein/ 10.20 RetroSense Therapeutics Retinitis Pigmentosa
16 Rt-PA/ 10.20 Kathleen A Stringer, PharmD, FCCP – Professor University of MI Plastic bronchitis
17 Dabrafenib/ 10.20 GlaxoSmithKline BRAF mutation positive non-small cell lung cancer
18 Ibalizumab/ 10.20 TaiMed Biologics Inc. HIV-1 infection in treatment experienced adult patients with documented multi-antiretroviral class resistance and evidence of HIV-1 replication despite ongoing antiretroviral therapy
19 Isavuconazonium sulfate/ 10.20 Astellas Pharma Invasive candidiasis/candidemia
20 [5-Amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxy-propoxy)-phenyl]-methanone/ 10.20 Synovo GmbH (Germany) Pancreatic Cancer
21 Triheptanoin/ 10.21 Ultragenyx Pharmaceutical Glucose transporter type-1 deficiency syndrome
22 N-(4-{3-(2-aminopyrimidin-4-yl)pyridin-2-yl]oxy}phenyl)-4-(4-methylthiophen-2-yl)phthalazin-1-amine bis-mesylate dehydrate/ 10.21 Amgen Ovarian Cancer
23 Autologous bone marrow CD34+ cells transduced ex vivo with a self activating HIV-1 -based lentiviral vector, EFS-ADA/ 10.21 Donald B. Kohn, MD – Professor, UC-UCLA Adenosine deaminase deficient severe combined immunodeficiency
24 Human trtravalent, bi-specific monoclonal antibody that binds and co-inhibits two growth factor receptors; insulin-like growth factor receptor 1 (IGF-1R) and v-erb-b2 avian erythroblastic leukemia viral oncogene homolog 3 (ErbB3)/ 10.22 Merrimack Pharmaceuticals Pancreatic Cancer
25 Autologous adipose derived mesenchymal stromal cells/ 10.22 Mayo Clinic Amyotrophic lateral sclerosis (ALS)
26 Bi-shRNA furin and GMCSF Autologous Tumor Cell Vaccine/ 10.22 Gradalis Inc. Ewing’s sarcoma
27 Alpha-1 proteinase inhibitor (human)/ 10.23 Kamada Ltd. (Israel) Graft versus host disease
28 Clenbuterol/ 10.27 Duke University Medical Center Adjunctive therapy with ERT for Pompe Disease
29 Sodium thiosulfate/ 10.28 Hope Pharmaceuticals Dermatomyositis
30 Pritumumab/ 10.28 Nascent Biotech Glioma
31 Oprozomib/ 10.28 Onyx Therapeutics Multiple Myeloma


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


•   2 ODDs for Glioma

•   3 ODDs for Pancreatic Cancer

•   Approximately 42 % of ODDs are for an oncology indication

•   ODDs for rare diseases:
1.   Friedriech’s Ataxia
2.   Epidermolysis Bullosa
3.   Glucose Transporter Type-1 Deficiency Syndrome.

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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