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FDA June 2014 Products Receiving Orphan Designation And Approvals

June 30, 2014






The chart below identifies FDA June 2014 Products Receiving Orphan Designation as of 06/30/14 in ascending “Orphan Drug Designation Date” order.

FDA June 2014 Products Receiving Orphan Designation

# Generic Name/ODD Sponsor Company Indication
1 Echothiophate Iodide/ 06.02 Makindus, Inc. Stargardt’s Disease
2 Everolimus/ 06.02 Novartis Diffuse Large B-Cell Lymphoma
3 Antagonist of the Complement 5a Receptor / 06.02 ChemoCentryx Anti-neutrophil cytoplasmic autoantibodies associated vasculitides (granulomatosis with polyangiitis or Wegener’s granulomatosis), microscopic polyangiitis, and Churg-Strauss syndrome
4 2-[4-(1-Methyl-4-pyridin-4-yl-lH-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid / 06.02 Pfizer Huntington’s Disease
5 Afatinib/ 06.04 Boehringer Ingelheim Malignant brain and central nervous system tumors
6 Vatiquinone/ 06.04 Edison Pharmaceuticals Leigh Syndrome
7 Trifarotene / 06.06 Galderma R&D LLC Congenital Ichthyosis
8 Autologous genetically modified human dermal fibroblasts / 06.10 Fibrocell Technologies Dystrophic Epidermolysis Bullosa
9 Isocitrate dehydrogenase 2-mutant inhibitor / 06.12 Agios Pharmaceuticals Acute Myelogenous Leukemia
10 Mocetinostat / 06.12 Mirati Therapeutics Myelodysplastic Syndrome
11 Epstein-Barr virus-specific autologous cytotoxic T lymphocytes/ 06.12 FFCanVac Pte Ltd (Singapore) Epstein-Barr virus positive head and neck cancers (which includes nasopharyngeal carcinoma)
12 6-[4-Deoxy-4-[(2E,4E)-tetradecadienoylglycyl]amino-L-glycero-B-L-manno-heptopyranosyl]amino-9H-purine/ 06.12 DARA BioSciences Multiple Myeloma
13 Small molecule normalizing the p53 function/ 06.12 Critical Outcome Technologies (Canada) Ovarian Cancer
14 Eculizumab / 06.13 Alexion Pharmaceuticals Myasthenia Gravis
15 Carglumic acid/ 06.17 Orphan Europe SARL (France) Organic Acidemias
16 Re188 P2045 somatostatin peptide analogue/ 06.19 Andarix Pharmaceuticals Pancreatic Cancer
17 Cannabidiol/ 06.23 Insys Therapeutics Lennox-Gastaut syndrome
18 (2’R,3’S)-2′-hydroxy-N-carboxy-3′-amino-5′-methyl-Hexanoic,N-tert-butyl ester, 13 ester 5B-20-epoxy-1B,2a,4a,7B,9a,10a,13a-heptahydroxy-4,10-diacetate-2-benzoate-(1″S)-7,9-acrolein acetal-11(15-1)-abeotaxane/ 06.23 Cortice Biosciences Progressive Supranuclear Palsy
19 Recombinant human nerve growth factor / 06.23 Dompe s.p.a. (Italy) Neurotrophic Keratitis
20 Recombinant human surfactant protein D/ 06.23 Airway Therapeutics Bronchopulmonary Dysplasia
21 Clofazimine/ 06.25 Novartis Active Tuberculosis
22 Sirolimus / 06.25 LAM Therapeutics Lymphangioleiomyomatosis
23 Lumacaftor and Ivacaftor/ 06.30 Vertex Pharmaceuticals Cystic Fibrosis
24 Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a nonviral cationic lipid based gene transfer agent/ 06.30 Imperial Innovations Limited (UK) Cystic Fibrosis
25 Humanized anti-IL-6R receptor neutralizing IgG2 monoclonal antibody/ 06.30 Chugai Pharma USA Neuromyelitis optica and neuromyelitis optica spectrum disorder


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

FDA June 2014 Orphan Products Receiving Approval

# Generic Name/Approval Date Sponsor Company Indication
1 Antihemophilic factor (recombinant), Fc fusion protein / 06.06 Biogen Idec Hemophilia A


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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