US biotechnology company Ionis Pharmaceuticals Inc (Nasdaq: IONS) announced on Thursday that it has received approval from the US Food and Drug Administration (FDA) for TRYNGOLZA (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronaemia syndrome (FCS).

FCS is a rare, genetic form of severe hypertriglyceridemia (sHTG) that can lead to potentially life-threatening acute pancreatitis (AP).

According to Ionis, TRYNGOLZA is the first FDA-approved treatment that significantly and substantially reduces triglyceride levels in adults with FCS and provides clinically meaningful reduction in AP events when used with an appropriate diet (less than 20 grams of fat per day). The treatment is self-administered through an auto-injector once monthly.

The approval was based on positive data from a global, multicentre, randomised, placebo-controlled, double-blind Phase 3 clinical trial in adult patients with genetically identified FCS and fasting triglyceride levels greater than or equal to 880 mg/dL.

In the study, TRYNGOLZA 80mg demonstrated a statistically significant placebo-adjusted mean reduction in triglyceride levels of 42.5% from baseline to six months. Reductions from baseline to 12 months were further improved, with TRYNGOLZA achieving a placebo-adjusted 57% mean reduction in triglycerides. TRYNGOLZA also demonstrated a substantial, clinically meaningful reduction in AP events over 12 months; one patient (5%) experienced one episode of AP in the TRYNGOLZA group compared with seven patients (30%) who experienced 11 total episodes of AP in the placebo group.