RNA-targeted medicines company Ionis Pharmaceuticals Inc (Nasdaq: IONS) announced on Monday that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olezarsen as an adjunct to diet for adults with severe hypertriglyceridemia (sHTG), defined as triglyceride levels ≥500 mg/dL. The designation aims to expedite review of therapies for serious or life-threatening conditions showing substantial improvement over existing treatments.

The designation is based on Phase 3 CORE and CORE2 studies, where olezarsen demonstrated up to a 72% placebo-adjusted reduction in triglycerides and an 85% reduction in acute pancreatitis events, with nearly 90% of patients achieving triglyceride levels below 500 mg/dL. The trials enrolled 1,063 adults on standard-of-care therapies, including participants with triglycerides ≥880 mg/dL. Olezarsen was administered via subcutaneous injection every four weeks, with efficacy measured at six months. Data were published in The New England Journal of Medicine and presented at the American Heart Association Scientific Sessions.

Olezarsen is an investigational RNA-targeted therapy designed to reduce apoC-III production, a key regulator of triglyceride metabolism. It is already approved in the US and EU as TRYNGOLZA for familial chylomicronemia syndrome. Ionis plans to submit a supplemental new drug application to the FDA by the end of 2025.

Ionis Pharmaceuticals has over three decades of experience developing RNA-targeted medicines, with marketed therapies and a leading pipeline in neurology, cardiometabolic disease and high-need areas, advancing innovation in RNA therapies and gene editing.