Ionis receives FDA Fast Track designation for Olezarsen in patients with Familial Chylomicronemia Syndrome
Ionis Pharmaceuticals today announced that the United States Food and Drug Administration (FDA) has granted olezarsen Fast Track designation for the treatment of familial chylomicronemia syndrome (FCS).
FCS 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. Fast Track designation is designed to expedite the FDA’s review of innovative, new drugs that demonstrate the potential to address unmet medical need.
“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.”
Ionis 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).
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