FDA Approves First Therapy to Reduce Pancreatitis Risk

In two placebo-controlled trials, triglyceride levels declined by up to 72% compared with placebo.

To approve olezarsen (Tryngolza) for reducing triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia.

Clinical Trials: Two randomized, double-blind, placebo-controlled trials with 1,061 adults were conducted to evaluate the efficacy of olezarsen.

Mean baseline triglyceride levels were 1,116 mg/dL.
In one trial, triglyceride levels decreased by 63% and 72% with 50-mg and 80-mg doses, respectively.
In the second trial, reductions were 49% and 55% with the 50-mg and 80-mg doses, respectively.
A pooled analysis indicated a lower incidence of acute pancreatitis in patients receiving olezarsen compared to placebo.

The FDA recommends liver enzyme testing before treatment initiation and ongoing monitoring due to potential adverse reactions.

The trials primarily focused on short-term efficacy and safety.
Long-term effects and safety profile of olezarsen remain to be fully established.

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