New FDA Orphan Drugs

 
 

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.
  • 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.
  • 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.
  • 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.
  • 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.
  • FDA Approves Orphan Drug Status for Pancreatic Cancer Vaccine The FDA has approved orphan drug status for a therapeutic vaccine to treat pancreatic cancer.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • New FDA Orphan Drugs: CDX-110, ATryn, ISIS 333611 The FDA has granted orphan drug designation to CDX-110 for the treatment of glioblastoma multiforme, ATryn for the treatment of hereditary antithrombin deficiency, and ISIS 333611 for the treatment of amyotrophic lateral sclerosis.
  • New FDA Orphan Drugs: KNS-760704, Colchicine, Panobinostat The FDA has granted orphan drug designation to KNS-760704 for the treatment of amyotrophic lateral sclerosis, colchicine for the treatment of familial Mediterranean fever and Behcet's Syndrome, and panobinostat for the topical treatment of cutaneous T-cell lymphoma.
  • New FDA Orphan Drugs: MB07133, VitiGam, ALS-357 The FDA has granted orphan drug designation to MB07133 for the treatment of liver cancer, VitiGam for the treatment of stage IIB to stage IV metastatic melanoma, and ALS-357 for the topical treatment of metastatic melanoma.
  • New FDA Orphan Drugs: IPI-504, Pafuramidine, Preos The FDA has granted orphan drug designation for IPI-504 for the treatment of gastrointestinal stromal tumors, pafuramidine for the treatment of African sleeping sickness, and Preos for the treatment of hypoparathyroidism.
  • 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.
  • New FDA Orphan Drugs The FDA has granted orphan drug designation for 5(S)-(2'-hydroxyethoxy)-20(S)-camptothecin for the treatment of osteosarcoma, 5,7-dihydroxy-3-(4-hydroxyphenyl)-chromen-4-one (Bio 300) for the treatment of acute radiation syndrome, and autologous cultured endothelial cells on a donor human corneal disk for the treatment of Fuch's dystrophy, bullous keratopathy, and pseudophakic bullous keratopathy.
  • 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.
  • New FDA Orphan Drugs The FDA has granted orphan drug designation for leukocyte interleukin as neoadjuvant therapy for patients with squamous cell carcinoma of the head and neck, pafuramidine maleate for the prevention of malaria, and plevitrexed for the treatment of gastric cancer.