Posts tagged: orphan
FDA approves EUSA’s orphan drug ERWINAZE for acute lymphoblastic leukemia
EUSA Pharma, a transatlantic specialty pharmaceutical company focused on oncology, oncology supportive care and critical care, today announced that the US Food and Drug Administration (FDA) has approved its orphan drug ERWINAZE (asparaginase Erwinia chrysanthemi) for the treatment of acute lymphoblastic leukemia (ALL) in patients with hypersensitivity to E. coli-derived ...
EMA recommends 4SC’s resminostat for orphan medicinal product designation to treat HL
4SC AG, a discovery and development company of targeted small molecule drugs for autoimmune diseases and cancer, today announced that the European Medicines Agency (EMA) has recommended 4SC's anti-cancer compound resminostat for designation as orphan medicinal product for the treatment of Hodgkin's Lymphoma (HL), a cancer of the lymphatic system.
Liver Cancer Drug Provectus Receives Orphan Drug Designation From FDA
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to Provectus Pharmaceuticals, Inc., for Rose Bengal, the active component in their new oncology medication PV-10. The drug is designed for the treatment of hepatocellular carcinoma (HCC), the most prevalent form of liver cancer. At present Provectus is ...
FDA grants cardiotrophin-1 Orphan Drug status for acute liver failure treatment to Digna Biotech
(Digna Biotech S.L) Biotechnological company Digna Biotech announced that the Food and Drug Administration granted cardiotrophin-1 Orphan Drug status for use in the treatment of acute liver failure.
FDA grants cardiotrophin-1 Orphan Drug status for acute liver failure treatment to Digna Biotech
Digna Biotech, a Spanish biotechnological company, announces that the Food and Drug Administration (FDA) granted cardiotrophin-1 (CT-1) Orphan Drug status for use in the treatment of acute liver failure (Designation request 11-3507). This provides more incentives to work on cures for this disease, which has an ...
FDA grants orphan drug designation for Avedro’s VibeX to treat Keratoconus
Avedro, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the Company's VibeX for use with its KXL System (UVA irradiation) for Corneal Cross-linking to treat Keratoconus.
FDA grants Orphan Drug Designation to Kamada’s AAT product for treating type 1 diabetes
Kamada LTD., a bio-pharmaceutical company engaged in the development, manufacturing and marketing of specialty life-saving therapeutics, was granted an Orphan Drug Designation for its Alpha-1 Antitrypsin (AAT) product to treat type 1 diabetes.
Penn receives $10 million to create center for orphan disease research and therapy
PHILADELPHIA - The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania announces the launching of a first-of-its-kind interdisciplinary center focused on discovering novel treatments for orphan diseases. The Center will bring together, without institutional walls, all necessary approaches to attacking and treating orphan diseases: establishing dedicated ...
Penn receives $10 million to create center for orphan disease research and therapy
(University of Pennsylvania School of Medicine) The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania announces the launching of a first-of-its-kind interdisciplinary center focused on discovering novel treatments for orphan diseases. The Center will bring together, without institutional walls, all necessary approaches to attacking and treating ...
Repligen Receives U.S. Fast Track Designation And European Orphan Medicinal Product Recommendation For RG3039 For Spinal Muscular Atrophy
Repligen Corporation (NASDAQ: RGEN) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RG3039, a potential treatment for Spinal Muscular Atrophy (SMA). Fast Track is a process designed to facilitate the development and expedite the review of drugs that treat serious diseases and ...
Genetic Finding Offers Hope For Orphan Disease: New Drugs Should Work For Rare Blood Disorder
New research conducted at UNC Lineberger Comprehensive Cancer Center, offers hope for people with a rare disorder called Chuvash polycythemia. Polycythemia is a disease characterized by excessive production of red blood cells. Symptoms include an enlarged spleen, blood clots, an increased risk of stroke, and in some cases the disease ...