The US Food and Drug Administration approved olezarsen (Tryngolza) for use with diet to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia.

Severe hypertriglyceridemia is defined as fasting triglyceride levels of at least 500 mg/dL. According to the US Food and Drug Administration (FDA), guidelines recommend lowering triglyceride levels above 500 mg/dL to reduce the risk of acute pancreatitis.

The approval was supported by two randomized, double-blind, placebo-controlled trials that enrolled 1,061 adults with severe hypertriglyceridemia. Mean baseline triglyceride levels across the studies were 1,116 mg/dL. The primary endpoint was the percent change in fasting triglyceride levels from baseline to month 6 compared with placebo.

In one trial, triglyceride levels decreased by 63% and 72% with the 50-mg and 80-mg doses, respectively, compared with placebo. In the second trial, reductions were 49% and 55% with the 50-mg and 80-mg doses, respectively. A pooled analysis of the two studies found a lower incidence of acute pancreatitis among patients receiving olezarsen compared with placebo.

The most common adverse reactions were injection-site reactions and increased liver enzyme levels. The FDA recommends that health care providers consider liver enzyme testing before treatment initiation, before dose escalation, and when clinically indicated thereafter. Persistent elevations may warrant dose interruption and/or dose reduction.

Potential allergic reactions reported with treatment included skin redness, hives, facial swelling, chills, and difficulty breathing. The FDA advises that health care providers should instruct patients to discontinue olezarsen and seek medical attention if signs or symptoms of an allergic reaction occur.

Olezarsen received Priority Review and Breakthrough Therapy designations for this indication.

Source: US Food and Drug Administration