By VETTAPHARMA reporter – Derek Roche: The U.S. Food and Drug Administration (FDA) has approved Ionis Pharmaceuticals’ Tryngolza (olezarsen), the first treatment shown to reduce the risk of acute pancreatitis in adults with severe hypertriglyceridemia.
Approved for use alongside diet, the once-monthly subcutaneous therapy addresses a long-standing unmet need by becoming the first medicine to demonstrate both substantial triglyceride reduction and a lower rate of acute pancreatitis in this high-risk patient population.
Severe hypertriglyceridemia is defined by fasting triglyceride levels of at least 500 mg/dL and is associated with an increased risk of acute pancreatitis, a potentially life-threatening inflammation of the pancreas. While previously approved therapies lower triglyceride levels, none had demonstrated a reduction in the risk of acute pancreatitis in clinical trials.
The approval was supported by two randomized, double-blind, placebo-controlled studies (NCT05079919 and NCT05552326) involving 1,061 adults with severe hypertriglyceridemia. Participants had an average baseline triglyceride level of 1,116 mg/dL. The primary endpoint in both trials was the percent change in fasting triglyceride levels from baseline to Month 6 (average of Weeks 25 and 27) compared with placebo.
In the first study, Tryngolza reduced triglyceride levels by 63% with the 50 mg dose and 72% with the 80 mg dose compared with placebo. In the second study, triglyceride reductions were 49% and 55% for the 50 mg and 80 mg doses, respectively. An integrated analysis of both trials showed that the rate of acute pancreatitis was lower in the pooled Tryngolza group than in the placebo group, providing the first clinical evidence that a triglyceride-lowering therapy can reduce pancreatitis risk in adults with severe hypertriglyceridemia.
The most common adverse reactions were injection-site reactions and elevated liver enzymes. The prescribing information recommends liver enzyme testing before treatment initiation, following dose increases, and when clinically indicated thereafter. Potential allergic reactions, including skin redness, hives, facial swelling, chills, and difficulty breathing, have also been reported.
The FDA granted Priority Review and Breakthrough Therapy designations to Tryngolza for this indication.
Quick FAQs
1. What Is Tryngolza?
Tryngolza (olezarsen) is a once-monthly subcutaneous therapy approved for use with diet to reduce triglyceride levels and the risk of acute pancreatitis in adults with severe hypertriglyceridemia.
2. What Happened?
The FDA approved Ionis Pharmaceuticals’ Tryngolza as the first treatment shown to reduce the risk of acute pancreatitis in adults with severe hypertriglyceridemia.
3. What Studies Supported The Approval?
The approval was based on two randomized, double-blind, placebo-controlled clinical trials (NCT05079919 and NCT05552326) involving 1,061 adults with severe hypertriglyceridemia.
4. What Is Severe Hypertriglyceridemia?
Severe hypertriglyceridemia is a condition in which fasting triglyceride levels are 500 mg/dL or higher, increasing the risk of acute pancreatitis and progressive metabolic complications.
5. What Were The Key Results?
Tryngolza reduced triglyceride levels by up to 72% compared with placebo and demonstrated a lower rate of acute pancreatitis than placebo in an integrated analysis of the two pivotal trials.
6. Why Is This Important?
Tryngolza is the first FDA-approved therapy shown to reduce the risk of acute pancreatitis in adults with severe hypertriglyceridemia, establishing a new treatment option for patients at high risk of this potentially life-threatening complication.
