– Recommendation based on Phase 3 Balance results, which showed a significant reduction of triglycerides and substantial reduction of acute pancreatitis events with TRYNGOLZA –

– European Commission decision expected by Q4 2025 –

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) and Sobi® today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of TRYNGOLZA® (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). The positive opinion is now referred to the European Commission (EC) for an approval decision, which is expected by Q4 2025.

The CHMP opinion is based on positive data from the Phase 3 Balance study, in which TRYNGOLZA demonstrated a statistically significant reduction in triglyceride levels at six months that was sustained through 12 months. Additionally, TRYNGOLZA demonstrated a substantial and clinically meaningful reduction in acute pancreatitis events over 12 months. TRYNGOLZA showed a favorable safety and tolerability profile. Study results were published in The New England Journal of Medicine (NEJM).

FCS is a rare and genetic form of severe hypertriglyceridemia (sHTG) that prevents the body from breaking down fats and severely impairs the ability to remove triglycerides from the bloodstream. People with FCS often have triglyceride levels of more than 880 mg/dL (10 mmol/L), compared to normal levels of <150 mg/dL (1.7 mmol/L), and are at high risk of developing acute pancreatitis, which can be life-threatening. In the EU, FCS is estimated to impact up to 13 people per million.

Sobi has exclusive rights to commercialize TRYNGOLZA in countries outside the U.S., Canada and China. As Ionis' European commercial partner for Waylivra (volanesorsen), the only medicine currently approved for FCS in the EU, Sobi will leverage existing market expertise and distribution channels to enable an effective TRYNGOLZA launch in FCS, if approved.

TRYNGOLZA was approved in the United States in December 2024 and granted orphan designation in the EU. Olezarsen is also being evaluated for sHTG, a serious condition defined by dangerously high triglycerides (≥500 mg/dL), and data from the Phase 3 CORE and CORE2 studies are expected in Q3 2025.