Ionis Pharmaceuticals And Sobi Say EU Approves TRYNGOLZA For Familial Chylomicronemia Syndrome

(RTTNews) - Ionis Pharmaceuticals, Inc. (IONS) and Swedish biopharmaceutical company Sobi announced Friday that TRYNGOLZA (olezarsen) has been approved in the European Union (EU) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS).

The approval follows the positive opinion of the Committee for Medicinal Products for Human Use.

The approval is based on positive data from the Phase 3 Balance study, in which TRYNGOLZA 80 mg demonstrated a statistically significant reduction in fasting 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.

FCS is a rare and genetic form of severe hypertriglyceridemia (sHTG). People with FCS often have triglyceride levels of more than 880 mg/dL (10 mmol/L), compared to normal levels of less than 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.

TRYNGOLZA is also being evaluated for sHTG, defined by severely high triglycerides =500 mg/dL (5.65 mmol/L), and positive topline results from the Phase 3 studies were announced in September 2025.

Balance is a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of olezarsen in patients with FCS at six and 12 months.