Biohaven’s Taldefgrobep Alfa Receives Fast Track Designation from FDA for Treating Spinal Muscular Atrophy
Biohaven Ltd. has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its novel anti-myostatin adnectin, taldefgrobep alfa, for treating spinal muscular atrophy (SMA). ...
Atriva receives FDA Orphan Drug Designation for Zapnometinib to treat Hantavirus Infections
Atriva Therapeutics GmbH, a biopharmaceutical company pioneering the development of host-targeting antiviral therapies, announced today that its lead candidate, zapnometinib (ATR-002), has received Or ...
FDA Grants Orphan Drug Designation to ATYR1923 for Sarcoidosis Treatment
aTyr Pharma, Inc., a clinical-stage biotherapeutics company focused on the discovery and development of novel medicines based on novel biological pathways, announced today that the U.S. Food and Drug ...
European Commission has designated oral decitabine and cedazuridine as orphan drugs for the treatment of Acute myeloid leukemia (AML)
Astex Pharmaceuticals, Inc. today announces that the European Commission (EC) has granted orphan-drug designation to the oral fixed-dose combination of decitabine and cedazuridine for the treatment of ...
SpliSense Announces EMA and FDA Grant Orphan Drug Designation to SPL84-23-1 for the Cystic Fibrosis Treatment
SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other pulmonary diseases, today announced that the U.S. Food and Drug Administrati ...
NeurOp Receives FDA Orphan Drug Designation for NP10679 for Subarachnoid Hemorrhage Treatment
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to NeurOp, Inc.'s investigational candidate NP10679 to treat Subarachnoid hemorrhage (SAH). NeurOp is working on a subun ...
U.S. FDA Grants Orphan Drug Designation to Tesomet In Hypothalamic obesity (HO)
Saniona, a clinical-stage biopharmaceutical company focused on rare diseases, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) to treat hypothalamic obesity (HO) ...
Rezolute, Inc. Receives FDA Orphan Drug Designation for Ersodetug for Hypoglycemia Due to Tumor Hyperinsulinism
Rezolute, Inc., a leading late-stage biopharmaceutical company focused on developing transformative therapies for rare diseases, announced that the U.S. Food and Drug Administration (FDA) has granted ...
Vir Biotechnology Secures EMA Orphan Drug Designation for Tobevibart and Elebsiran in Treating Chronic Hepatitis Delta
Vir Biotechnology Secures EMA Orphan Drug Designation for Tobevibart and Elebsiran in Treating Chronic Hepatitis Delta Vir Biotechnology, Inc. has announced a significant milestone in the fight agains ...
FDA Grants Orphan Drug Designation to Remedy Pharma’s CIRARA for Treatment of Acute Ischemic Stroke
Remedy Pharmaceuticals, a pioneer in stroke drug development, has announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Division (OOPD) has granted Orphan Drug Designati ...