December 1, 2025 — Leads & Copy —
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for its investigational medicine, olezarsen. The designation is for olezarsen when used as an adjunct to diet, to reduce triglyceride (TG) levels in adults with severe hypertriglyceridemia (sHTG), defined as TG levels greater than or equal to 500 mg/dL.
The FDA’s Breakthrough Therapy designation aims to accelerate the review of drugs intended to treat serious or life-threatening conditions, demonstrating preliminary clinical evidence of offering significant improvement over existing treatments.
The Breakthrough Therapy designation was granted based on Phase 3 CORE and CORE2 clinical study results of olezarsen. Data from the studies were published in The New England Journal of Medicine and presented at the American Heart Association Scientific Sessions.
In the CORE and CORE2 studies, olezarsen demonstrated a statistically significant, placebo-adjusted reduction in triglyceride levels of up to 72%, and an 85% reduction in acute pancreatitis events, along with favorable safety and tolerability. Nearly 90% of patients treated with olezarsen achieved triglyceride levels below 500 mg/dL, which is below the risk threshold for acute pancreatitis.
Ionis plans to submit a supplemental new drug application to the FDA before the end of the year.
According to the press release, existing therapies for sHTG often fail to sufficiently lower triglyceride levels or prevent life-threatening acute pancreatitis attacks.
Severe hypertriglyceridemia (sHTG), characterized by triglycerides ≥500 mg/dL, increases the risk of acute pancreatitis and other severe health issues, such as organ damage. Acute pancreatitis can cause severe abdominal pain requiring prolonged hospitalization and may become life-threatening, making prevention of initial attacks critical. People with a history of acute pancreatitis episodes face an even greater risk of future occurrences.
Olezarsen is designed to lower the body’s production of apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood. Olezarsen is approved in the U.S. and the European Union under the brand name TRYNGOLZA® for adults with familial chylomicronemia syndrome (FCS).
The CORE studies included 617 participants while the CORE2 study consisted of 446 participants.
Sam Tsimikas, M.D., senior vice president, global cardiovascular development, Ionis, stated that the Breakthrough Therapy designation reflects the groundbreaking nature of the CORE and CORE2 study results.
As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. Ionis currently has marketed medicines and a leading pipeline in neurology, cardiometabolic disease and select areas of high patient need.
Ionis Pharmaceuticals® and TRYNGOLZA® are trademarks of Ionis Pharmaceuticals, Inc.
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Ionis Pharmaceuticals, Inc.
Source: Ionis Pharmaceuticals, Inc.
