The FDA has approved olezarsen as the first and only therapy, alongside diet, to lower triglycerides and acute pancreatitis risk in patients with severe hypertriglyceridemia, according to a manufacturer press release.
Olezarsen (Tryngolza, Ionis Pharmaceuticals), an apolipoprotein C-III inhibitor, is administered once monthly by autoinjector and is available in 50 mg and 80 mg doses. Olezarsen had been granted FDA orphan drug status in February 2024 for the treatment of familial chylomicronemia syndrome, a rare form of severe hypertriglyceridemia (sHTG).
The FDA based its approval on positive results from the phase 3 CORE and CORE2 trials, published in The New England Journal of Medicine. In the studies, olezarsen delivered significant triglyceride reductions at 6 months: The 50 mg dose lowered levels by 49% to 63% vs. placebo, while the 80 mg dose achieved reductions of 55% to 72%.
At 12 months, olezarsen was linked to significant reduction in acute pancreatitis events, with the 50 mg and 80 mg doses demonstrating 91% and 76% reductions, respectively. Additionally, 86% of patients with baseline and 12-month data achieved triglyceride levels below 500 mg/dL, a key threshold associated with lower acute pancreatitis risk.
The most common adverse events for patients with sHTG included injection site reactions and liver enzyme increases.
