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FDA May 2014 Products Receiving Orphan Designation

May 31, 2014






The chart below identifies FDA May 2014 Products Receiving Orphan Designation as of 06/02/14 in ascending “Orphan Drug Designation Date” order.

FDA May 2014 Products Receiving Orphan Designation

Updated 06/02/14 and 06/05/14 with FDA updates.

# Generic Name/ODD Date Sponsor Company Indication
1 Filanesib/ 05.06 Array BioPharma Multiple Myeloma
2 Autologous dendritic cells pulsed with allogeneic tumor cell lysate/ 05.06 Amphera BV (Netherlands) Malignant Mesothelioma
3 Imatinib Mesylate/ 05.06 Inhibikase Therapeutics Progressive multifocal leukencephalopathy
4 Ex vivo cultured human mesenchymal stromal cells / 05.08 iCell Science AB (Sweden) Prevention of graft rejection following solid organ transplantation
5 Adalimumab/ 05.13 AbbVie Uveitis
6 Diazoxide choline / 05.13 Essentialis Prader-Willi Syndrome
7 Vasoactive intestinal peptide (VIP)-elastin-like peptide (ELP) fusion protein / 05.13 PhaseBio Pharmaceuticals Pulmonary arterial hypertension
8 (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N-(pyrazin-2-yl)acrylohydrazide / 05.14 Karyopharm Therapeutics Diffuse large B-cell lymphoma
9 177Lu-tetraxetan-tetulomab / 05.14 Nordic Nanovector AS (Norway) Follicular Lymphoma
10 Selinexor; (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N-(pyrazin-2-yl)acrylohydrazide / 05.14 Karyopharm Therapeutics Acute myeloid leukemia
11 Menadione Sodium Bisulfite/ 05.14 IC-MedTech Corporation Autosomal dominant polycystic kidney disease
12 Inecalcitol/ 05.14 Hybrigenics, S.A. (France) Chronic lymphocytic leukemia
13 Targeted Gastrin 17 Complexed Peptide/ 05.14 Tyg Oncology Ltd (UK) Pancreatic cancer
14 Menadione sodium bisulfite/ 05.14 IC-MedTech Corporation Autosomal dominant polycystic liver disease
15 [a-N-(2’succinyl-paclitaxel)Thr]-Phe-Phe-Tyr-Gly-Gly-Ser-Arg-Gly-[epsilon-N-(2’succinyl-paclitaxel)Lys]-Arg-Asn-Asn-Phe-[epsilon-N-(2’succinyl-paclitaxel)Lys]-Thr-Glu-Glu-Tyr/ 05.14 Angiochem (Canada) Glioblastoma multiforme
16 Afamelanotide/ 05.14 Clinuvel Familial benign chronic pemphigus (Hailey-Hailey disease)
17 Recombinant humanized monoclonal antibody of the immunoglobulin G1 subclass directed against colony stimulating factor-1 receptor expressed on macrophages/ 05.14 Genentech Pigmented villonodular synovitis and tenosynovial giant cell tumor
18 4-{[(2R,3S,4R,5S)-4-(4-Chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluoro-phenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid/ 05.19 Roche Genentech Acute Myeloid Leukemia
19 Recombinant lens epithelium derived growth factor 1-326/ 05.19 Ocugen Retinitis pigmentosa
20 Live attenuated bioengineered Listeria monocytogenes immunotherapy/ 05.21 Advaxis Osteosarcoma
21 All-cis-docosa-4,7,10,13,16,19-hexaenoic acid / 05.21 Natac Pharma (Spain) Retinitis pigmentosa
22 Humanized immunoglobulin monoclonal antibody against CD38/ 05.22 Sanofi-Aventis Multiple myeloma
23 Miconazole/ 05.22 Hill Dermaceuticals Fungal otitis externa (otomycosis)
24 Spironolactone/ 05.22 CMP Pharma Pediatric patients with primary hyperaldosteronism
25 p38 mitogen-activated kinase inhibitor/ 05.27 Array BioPharma Dilated cardiomyopathy
26 Human Monoclonal Antibody against Epidermal Growth Factor Receptor Linked to Monomethylauristatin F AbbVie Glioblastoma multiforme
27 Humanized IgG monoclonal antibody hRS7 conjugated to topoisomerase I inhibitor/ 05.29 Immunomedics Pancreatic cancer
28 Metformin/ 05.29 EffRx Pharmaceuticals SA (Switzerland) Pediatric polycystic ovary syndrome
29 Heparan sulfate mimetic/ 05.29 Momenta Pharmaceuticals Pancreatic cancer
30 von Willebrand Factor Human Concentrate/ 05.29 rEVO Biologics Von Willebrand Disease


** “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-3.0] | Wikimedia Commons.

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