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FDA April 2014 Products Receiving Orphan Designation

May 1, 2014





The chart below identifies FDA April 2014 Products Receiving Orphan Designation as of 04/30/14 in ascending “Orphan Drug Designation Date” order.


FDA April 2014 Products Receiving Orphan Designation 

Updated 05/04/14 with #s 25-28.

 # Generic Name/ODD Date Sponsor Company Indication
1 pegylated proline interferon alpha-2b/ 04.10 PharmaEssentia Corp (Taiwan) Myelofibrosis
2 isotretinoin/ 04.10 Patagonia Pharmaceuticals Congenital Ichthyosis
3 Carbetocin/ 04.10 Ferring Pharmaceuticals Prader Willi Syndrome
4 {2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl](3H-benzo[f]azepin-4-yl)}-N,N-dipropycarboxamide/ 04.10 VentiRx Pharmaceuticals Ovarian Cancer
5 pegylated proline interferon alpha-2b/ 04.11 PharmaEssentia Corp (Taiwan) Thrombocythemia
6 H-Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Arg-Lys-Leu-Ser-Ser-Ile-Glu- Ser-Asp-Val-OH/ 04.11 NoNO Inc Acute Ischemic Stroke
7 ……./ 04.11   Post-exposure prophylaxis of anthrax disease resulting from suspected or confirmed Bacillus anthracis exposure
8 benznidazole/ 04.14 Chemo Research (Spain) Chagas Disease
9 naltrexone/clonidine combination/ 04.14 Allodynic Therapeutics Postherpetic Neuralgia
10 volasertib/ 04.14 Boehringer Ingelheim Acute Myeloid Leukemia
11 6-(1H-Indazol-6-yl)-N-(4-morpholinophenyl)imidazo-[1,2-alpha]pyrazin-8-amine bis-methanesulfonate/ 04.14 Gilead Sciences Chronic Lymphocytic


12 ciprofloxacin dry powder inhaler/ 04.17 Bayer HealthCare Pharmaceuticals Non-Cystic Fibrosis Bronchiectasis
13 teriparatide/04.18 Entera Bio Ltd. (Israel) hypoparathyroidism
14 Allopregnanolone/04.20 Sage Therapeutics Status Epilepticus
15 (R)-1-(2,2-difluorobenzo [d][1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide/04.24 Vertex Pharmaceuticals Cystic Fibrosis
16 Sotatercept/04.28 Celgene Anemias associated with myelodysplastic syndrome

& myelodysplastic/ myeloproliferative


17 ………./ 04.28   Acute Myeloid Leukemia
18 3-bromopyruvate/04.29 PreScience Labs Pancreatic Cancer
19 humanized Fc engineered monoclonal antibody against CD19/04.29 MorphoSys AG (Germany) chronic lymphocytic leukemia/small



20 a live attenuated bioengineered Listeria monocytogenes cancer immunotherapy/04.29 Advaxis Stage II to IV




21 recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase/04.29 Abeona Therapeutics Mucopolysaccharidosis

type III-A

(Sanfilippo Syndrome

Type A)

22 Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase/04.30 Abeona Therapeutics Mucopolysaccharidosis

III-B (Sanfilippo

Syndrome Type B)

23 moxifloxacin hydrochloride/04.30 Bayer HealthCare Pharmaceuticals Tuberculosis
24 Human plasma derived conagulation protein-Factor XI/04.30 Cambryn Biologics Congenital Factor XI    Deficiency
25 (3E,5E)-3,5-bis[(4-fluoro-3-nitrophenyl)methylidene]-1-(prop-2-enoyl)azepan-4-0ne/ 04.30 VivoLux AB (Sweden) Multiple Myeloma
26 18-(p-[124I]-iodophenyl)octadecyl phosphocholine/ 04.30 Cellectar Biosciences Glioma
27 Ipatasertib/ 04.30 Genentech Gastric Cancer
28 Demcizumab/ 04.30 OncoMed Pharmaceuticals Pancreatic Cancer
29 pegylated granulocyte colony stimulating factor/ 04.30 PharmaEssentia (Taiwan) patients at risk of developing myelosuppression following a radiological or nuclear incident


** “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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