Key Takeaways:
The Food and Drug Administration approved Ionis Pharmaceuticals Inc.'s Tryngolza (olezarsen) as the first treatment shown to reduce both triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia, the agency said Wednesday.
"Tryngolza represents a meaningful advance for patients with severe hypertriglyceridemia who have had limited options to reduce their risk of acute pancreatitis," Dr. Norman Stockbridge, director of the FDA's Division of Cardiology and Nephrology, said.
The approval was supported by two Phase 3 trials — CORE-TIMI 72a and CORE2-TIMI 72b — enrolling 1,061 patients total. At six months, patients receiving the 50-milligram dose saw a placebo-adjusted triglyceride reduction of 62.9 percentage points in CORE and 49.2 percentage points in CORE2; the 80-mg dose produced reductions of 72.2 and 54.5 percentage points, respectively (P<.001 for all comparisons). Acute pancreatitis events were 85% lower with Tryngolza than placebo (rate ratio, 0.15; 95% confidence interval, 0.05 to 0.40; P<.001).
Severe hypertriglyceridemia is defined by fasting triglyceride levels of at least 500 milligrams per deciliter, more than three times the normal threshold of 150 mg/dL. Standard care has included lifestyle modifications and older medications that never demonstrated a statistically significant reduction in acute pancreatitis risk in their trials, the FDA said. Tryngolza is self-administered once monthly via autoinjector in either a 50-mg or 80-mg dose. The most common side effects were injection site reactions and elevated liver enzymes; the 80-mg dose was also associated with a higher rate of thrombocytopenia.
The approval positions Ionis as the first company with a labeled indication for acute pancreatitis risk reduction in sHTG, a market with no direct competitor offering comparable data. The company, which has a market capitalization of about $12.75 billion, previously won FDA approval for Tryngolza in December 2024 for familial chylomicronemia syndrome, a rare genetic form of severe hypertriglyceridemia. Investors will watch for initial prescription data and potential label expansion into broader cardiovascular outcomes, which could significantly expand the addressable patient population beyond the estimated 4 million Americans with triglyceride levels above 500 mg/dL.
This article is for informational purposes only and does not constitute investment advice.
