Ionis treatment for Alexander disease granted orphan drug status from EMA

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[{"type":"text","content":"CARLSBAD, Calif., Feb. 11, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that the European Medicines Agency (EMA) has granted orphan drug designation to ION373 for the treatment of people with Alexander disease, a severe, progressive and debilitating condition that can eventually result in death due to loss of control over autonomic functions like breathing. Infants and young children who develop the condition rarely survive beyond their teenage years or young adulthood. \n\n \n \n\n \nOrphan drug designation by the EMA provides regulatory and financial 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. Upon approval, drugs that have been granted orphan designation by the EMA receive market exclusivity for 10 years in the EU. \nAlexander disease affects approximately 500 people in the EU. It is caused by a mutation in a protein that creates overproduction of glial fibrillary acidic protein (GFAP) in the brain. ION373 is designed to stop the mutated gene from producing excess GFAP.\n\"We look forward to working closely with European regulators, clinical investigators, Alexander disease patients and their families to advance this important medicine and make it available to those who need it,\" said Brett P. Monia, Ph.D., Ionis' chief executive officer. \"Receiving orphan drug status reflects the urgent need for ION373, which is among a number of wholly-owned, novel drugs in the Ionis pipeline that we are prioritizing.\"\nAbout Ionis Pharmaceuticals, Inc.\nAs the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyret...

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