Orphan Drug Designations – April 2014

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A list of the Orphan drug designations and approvals by the US Food and drug Administration in April 2014.

FDA – 10/04/2014
Generic Name: isotretinoin
Trade Name:
Orphan Designation: Treatment of congenital ichthyosis
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Patagonia Pharmaceuticals
FDA – 10/04/2014
Generic Name: pegylated proline interferon alpha-2b
Trade Name:
Orphan Designation: Treatment of myelofibrosis
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: PharmaEssentia
FDA – 10/04/2014
Generic Name: {2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl](3H-benzo[f]azepin-4-yl)}-N,N-dipropycarboxamide
Trade Name:
Orphan Designation: Treatment of ovarian cancer
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: VentiRx Pharmaceutical
FDA – 10/04/2014
Generic Name: carbetocin
Trade Name:
Orphan Designation: Treatment of Prader Willi syndrome
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Ferring Pharmaceuticals
FDA – 11/04/2014
Generic Name: pegylated proline interferon alpha-2b
Trade Name:
Orphan Designation: Treatment of thrombocythemia
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: PharmaEssentia
FDA – 11/04/2014
Generic Name:  H-Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Arg-Lys-Leu-Ser-Ser-Ile-Glu- Ser-Asp-Val-OH
Trade Name:
Orphan Designation: Treatment of acute ischemic stroke patients presenting within 3 hours of symptom onset
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: NoNO
FDA – 14/04/2014
Generic Name: volasertib
Trade Name:
Orphan Designation: Treatment of acute myeloid leukemia
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Boehringer Ingelheim Pharmaceuticals
FDA – /04/2014
Generic Name: 6-(1H-Indazol-6-yl)-N-(4-morpholinophenyl)imidazo-[1,2-alpha]pyrazin-8-amine bis-methanesulfonate
Trade Name:
Orphan Designation: Treatment of chronic lymphocytic leukemia
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Gilead Sciences
FDA – 14/04/2014
Generic Name: benznidazole
Trade Name:
Orphan Designation: Treatment of Chagas disease
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Chemo Research
FDA – 14/04/2014
Generic Name: naltrexone/clonidine combination
Trade Name:
Orphan Designation: Treatment of postherpetic neuralgia
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Allodynic Therapeutics
FDA – 17/04/2014
Generic Name: ciprofloxacin dry powder inhaler
Trade Name:
Orphan Designation: Treatment of non-cystic fibrosis bronchiectasis
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Bayer HealthCare Pharmaceuticals
FDA – 18/04/2014
Generic Name: teriparatide
Trade Name:
Orphan Designation: Treatment of hypoparathyroidism
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Entera Bio
FDA – 20/04/2014
Generic Name: allopregnanolone
Trade Name:
Orphan Designation: Treatment of status epilepticus
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Sage Therapeutics
FDA – 24/04/2014
Generic Name: (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
Trade Name:
Orphan Designation: Treatment of cystic fibrosis
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Vertex Pharmaceuticals
 FDA – 28/04/2014
Generic Name: sotatercept
Trade Name:
Orphan Designation: Treatment of anemias associated with myelodysplastic syndrome and myelodysplastic/myeloproliferative neoplasms.
Orphan Designation Status: Designated
FDA Orphan Approved: No
Celgene
FDA – 29/04/2014
Generic Name: recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase
Trade Name:
Orphan Designation: Treatment of mucopolysaccharidosis type III-A (Sanfilippo Syndrome Type A)
Orphan Designation Status: designated
FDA Orphan Approved:  No
Sponsor: Abeona Therapeutics
FDA – 29/04/2014
Generic Name: humanized Fc engineered monoclonal antibody against CD19
Trade Name:
Orphan Designation: Treatment of patients with chronic lymphocytic leukemia/small lymphocytic lymphoma
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: MorphoSys AG
FDA – 29/04/2014
Generic Name: a live attenuated bioengineered Listeria monocytogenes cancer immunotherapy
Trade Name:
Orphan Designation: Treatment of Stage II to IV invasive cervical carcinoma
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Advaxis
FDA – 30/04/2014
Generic Name: 3-bromopyruvate
Trade Name:
Orphan Designation: Treatment of pancreatic cancer
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: PreScience Labs
FDA – 30/04/2014
Generic Name: moxifloxacin hydrochloride
Trade Name:
Orphan Designation: Treatment of tuberculosis
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Bayer HealthCare Pharmaceuticals
FDA – 30/04/2014
Generic Name: Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase
Trade Name:
Orphan Designation: Mucopolysaccharidosis III-B (Sanfilippo Syndrome Type B)
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Abeona Therapeutics
FDA – 30/04/2014
Generic Name: Human plasma derived conagulation protein-Factor XI
Trade Name:
Orphan Designation: For the treatment of congenital Factor XI deficiency
Orphan Designation Status: Designated
FDA Orphan Approved: No
Sponsor: Cambryn Biologics

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