Olezarsen - (CORE)

February 26, 2026: Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced the FDA accepted for Priority Review its supplemental NDA for olezarsen for severe hypertriglyceridemia (sHTG), with a PDUFA target action date of June 30, 2026. The filing and Priority Review were supported by Phase 3 CORE (NCT05079919; n=617) and CORE2 (NCT05552326; n=446) results: olezarsen achieved a highly statistically significant placebo-adjusted triglyceride reduction of up to 72%, an 85% reduction in acute pancreatitis events, and nearly 90% of treated patients reached triglycerides <500 mg/dL. CORE/CORE2 were global, randomized, double-blind, placebo-controlled trials in adults with baseline triglycerides ≥500 mg/dL receiving standard of care; dosing was 50 mg or 80 mg subcutaneously every 4 weeks for 12 months, with primary endpoint percent change in fasting triglycerides at six months versus placebo. The company noted the data were published in The New England Journal of Medicine and presented at the American Heart Association Scientific Sessions, and that olezarsen previously received FDA Breakthrough Therapy designation (November 2025).