Olezarsen sNDA accepted by the FDA for Priority Review for the treatment of severe hypertriglyceridemia (sHTG)

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[{"type":"text","content":"\n– PDUFA date set for June 30, 2026 –\n\n\n CARLSBAD, Calif.--(BUSINESS WIRE)--\nIonis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for olezarsen for severe hypertriglyceridemia (sHTG). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026.\n\n\n“Current standard of care therapies for sHTG provide limited benefit, leaving people vulnerable to recurrent and debilitating acute pancreatitis attacks with serious, long-term health consequences,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “The FDA’s Priority Review designation underscores the urgent need for additional treatment options and will enable us to bring olezarsen to patients as quickly as possible. This milestone represents a significant step toward our goal of delivering the first-ever treatment shown to reduce the risk of potentially life-threatening acute pancreatitis attacks in people with sHTG.”\n\n\nThe sNDA and Priority Review designation were based on the positive results from the Phase 3 CORE and CORE2 studies of olezarsen. In the studies, olezarsen demonstrated a highly statistically significant placebo-adjusted reduction in triglyceride levels of up to 72% and an 85% reduction in acute pancreatitis events with favorable safety and tolerability. Additionally, nearly 90% of olezarsen-treated patients achieved triglyceride levels less than 500 mg/dL, which is below the risk threshold for acute pancreatitis. The data were published in The New England Journal of Medicine and presented at the American Heart Association Scientific Sessions.\n\n\nPriority Review designation is granted to marketing applications for medicines that, if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention or diagnosis of a serious condition, with the expectation of the FDA taking action within six months, compared to 10 months under standard review. The U.S. FDA previously granted olezarsen Breakthrough Therapy designation in November 2025.\n\n\nAbout the CORE and CORE2 Studies\n\n\nCORE (NCT05079919; n=617) and CORE2 (NCT05552326; n=446), conducted with The TIMI Study Group, are Phase 3 global, multicenter, randomized, double-blind, placebo-contr...

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