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Ionis treatment for Alexander disease receives orphan drug designation from U.S. FDA
- U.S. orphan drug status follows similar designation by the European Medicines Agency CARLSBAD, Calif., Sept. 30, 2020 /PRNewswire/ -- Ionis Pharmaceuticals,
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"- U.S. orphan drug status follows similar designation by the European Medicines Agency\n\n\nCARLSBAD, Calif., Sept. 30, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ION373 for the treatment of people with Alexander disease, a severe, progressive and debilitating rare neurodegenerative disease that can result in death. Infants and young children who develop the condition rarely survive beyond their teenage years or young adulthood. Alexander disease has been estimated to occur in about one in one million births. Orphan drug designation is granted by the FDA to drugs and biologics intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation. \n\n \n \n \n \n \n \n\n \nAlexander disease is caused by a mutation in a protein resulting in overproduction of glial fibrillary acidic protein (GFAP) in the brain. ION373 is an Ionis-owned investigational antisense medicine designed to stop the mutated gene from producing excess GFAP.\nUnder the FDA's Orphan Drug Act, orphan drug status provides incentives, including waiver of certain administrative fees, grants and tax credits for clinical trials, and seven years of market exclusivity following drug approval. Earlier this year, the European Medicines Agency (EMA) granted orphan drug designation to ION373. EMA provides regulatory, financial and market incentives to develop therapies for life threatening or chronically debilitating conditions affecting not more than five in 10,000 people in the European Union (EU) and for which there is no satisfactory method of diagnosis, prevention or treatment. \n\"Receiving FDA orphan drug status for ION373 reflects the urgent need for a novel medicine to treat Alexander disease. We look forward to working closely with regulators, clinical investigators, Alexander disease patients and their families to advance this important medicine and make it available to those who need it,\" said Frank Bennett, Ph.D., Ionis' chief scientific officer and franchise leader for neurological programs. \nION373 is one of several Ionis-owned investigational medicines designed to treat neurological diseases. Others include...