New FDA Orphan Drugs

FDA Approves Clobazam Add-On for Lennox Syndrome The benzodiazepine is already available in 100 other countries, marketed under different brand names.
News, October 24, 2011
Orphan Drug Status for Corneal Cross-Linking in Keratoconus Corneal cross-linking is the first medical therapy available in the US to slow disease progress.
News, September 14, 2011
FDA Approves First Treatment for Congenital Factor XIII Deficiency The FDA has approved the first product intended to prevent bleeding in people with congenital factor XIII deficiency.
News, February 23, 2011
FDA Approves Orphan Drug Fidaxomicin for Pediatric CDI The FDA has granted orphan drug designation for all formulations of fidaxomicin for the treatment of Clostridium difficile infection (CDI) in pediatric patients aged 16 years and younger.
News, January 10, 2011
FDA Approves Orphan Drug Status for Type 1 Diabetes Stem Cell Therapy The FDA has approved orphan drug designation for a stem cell therapy in patients with newly diagnosed type 1 diabetes mellitus.
News, May 07, 2010
FDA Approves Orphan Drug Status for Pancreatic Cancer Vaccine The FDA has approved orphan drug status for a therapeutic vaccine to treat pancreatic cancer.
News, March 18, 2010
FDA Approves Orphan Drug Status for Inhaled Ciprofloxacin for Cystic Fibrosis The FDA has approved orphan drug status for inhaled ciprofloxacin for pulmonary infection resulting from Pseudomonas aeruginosa infection in patients with cystic fibrosis.
News, March 16, 2010
Personalized Lymphoma Vaccine Is Granted Orphan Drug Status The manufacturer says it is preparing to file for approval on the basis of data that showed an improvement in disease-free survival in lymphoma.
News, January 13, 2010
High-Dose Melphalan Granted Orphan Drug Status for Neuroendocrine Liver Metastases The FDA has approved orphan drug designation for melphalan infusion in the treatment of patients with neuroendocrine tumors metastatic to the liver.
News, June 10, 2009
High-Concentration Capsaicin Patch Granted Orphan Drug Designation for PHN The FDA has approved orphan drug designation for a high-concentration capsaicin dermal patch (Qutenza) for the treatment of pain associated with postherpetic neuralgia.
News, June 09, 2009
New FDA Orphan Drugs The FDA has granted orphan drug designation to oral fludarabine phosphate for the treatment of chronic lymphocytic leukemia, clobazam for the adjunctive treatment of Lennox-Gastaut syndrome, and oral beclomethasone dipropionate for the treatment of pediatric Crohn's disease.
News, January 18, 2008
New FDA Orphan Drugs The FDA has granted orphan drug designation to A-001 for the prevention of acute chest syndrome in patients with sickle cell disease, JZP-8 for the treatment of recurrent acute repetitive seizures in patients with epilepsy, and AST-120 for the treatment of pouchitis.
News, January 11, 2008
New FDA Orphan Drugs: Sodium Phenylbutyrate, MGCD0103, Bendamustine HCl The FDA has granted orphan drug designation for sodium phenylbutyrate for the treatment of spinal muscular atrophy, MGCD0103 for the treatment of Hodgkin's lymphoma, and bendamustine HCl for the treatment of chronic lymphocytic leukemia.
News, July 26, 2020
New FDA Orphan Drugs The FDA has granted orphan drug designation for mifepristone for the treatment of Cushing's syndrome, 1-deoxynojirimycin hydrochloride for the treatment of Pompe disease, and cetuximab for the treatment of pancreatic cancer.
News, July 26, 2020
New FDA Orphan Drugs: Levulan, Vekacia, Hexarelin Analog The FDA has granted orphan drug designation for aminolevulinic acid HCl in combination with photodynamic therapy, a cyclosporine A drug, and a peptidomimetic analog of hexarelin.
News, July 26, 2020
New FDA Orphan Drugs: Ganciclovir and Tissue Repair Cells The FDA has granted orphan drug designation for ganciclovir ophthalmic gel in the treatment of acute herpetic keratitis and tissue repair cells in the treatment of dilated cardiomyopathy.
News, July 26, 2020
New FDA Orphan Drugs: Cethromycin, Maribavir, rhPDGF-BB The FDA has granted orphan drug designation for cethromycin, maribavir, and recombinant human platelet-derived growth factor (rhPDGF-BB).
News, July 26, 2020
New FDA Orphan Drugs: VariZIG, Pafuramidine Maleate, RG1068 The FDA has granted orphan drug status for varicella zoster immune globulin [human] injection, pafuramidine maleate, and synthetic human secretin.
News, July 26, 2020
New FDA Orphan Drugs: MGA031, KBPA101, TLF Oral Solution The FDA has approved orphan drug status for the humanized Fc-engineered anti-CD3 monoclonal antibody hOKT3-gamma-1 (Ala-Ala; MGA031), a fully human monoclonal antibody (KBPA101), and an oral solution formulation of talactoferrin alfa (TLF).
News, July 26, 2020
New FDA Orphan Drugs: CellCept, Prochymal, Shiga Toxin Antibodies The FDA has approved orphan drug indications for mofetil (CellCept), an adult stem cell product (Prochymal), and 2 chimeric anti-Shiga toxin antibodies (caStx1 and caStx2).
News, July 26, 2020

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