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Ionis receives FDA Fast Track designation for olezarsen in patients with familial chylomicronemia syndrome
FCS is a rare and debilitating genetic disease often leading to significant risk of acute, potentially fatal pancreatitisIf approved, olezarsen would be the
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"FCS is a rare and debilitating genetic disease often leading to significant risk of acute, potentially fatal pancreatitisIf approved, olezarsen would be the first approved treatment for FCS in the U.S.CARLSBAD, Calif., Jan. 31, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted olezarsen Fast Track designation for the treatment of familial chylomicronemia syndrome (FCS).\n\n \n \n \n \n \n \n\n \nFCS is a debilitating genetic disease characterized by severely high levels of plasma triglycerides and a risk of unpredictable and potentially fatal acute pancreatitis. In addition to acute pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and type 3c diabetes. There are currently no approved therapies for the treatment of FCS in the U.S.\nFast Track designation is designed to expedite the FDA's review of innovative, new drugs that demonstrate the potential to address unmet medical need.\n\"The FDA Fast Track designation for olezarsen recognizes the urgent need for an effective treatment for FCS, a debilitating rare disease affecting people with very limited treatment options and an elevated risk of painful and potentially fatal bouts of pancreatitis,\" said Richard S. Geary, Ph.D., executive vice president and chief development officer at Ionis. \"We look forward to working collaboratively with the FDA to bring forward a safe and effective treatment for FCS patients as quickly as possible.\"\nIonis fully enrolled its global Phase 3 BALANCE study of olezarsen in adult patients with FCS last year. The company plans to share data from the BALANCE study in the second half of 2023. In addition to FCS, Ionis is evaluating olezarsen in severe hypertriglyceridemia (SHTG). More information on the BALANCE study (NCT04568434) is available at www.clinicaltrials.gov.\nAbout familial chylomicronemia syndrome (FCS)\nFCS is a rare, genetic disease estimated to affect 3,000 to 5,000 people worldwide and is characterized by extremely elevated triglyceride levels. FCS can lead to many chronic health issues including severe, recurrent abdominal pain, fatigue, high risk of life-threatening pancreatitis and abnormal enlargement of the liver or spleen. In addition, people with FCS are often una...