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TRYNGOLZA® (olezarsen) recommended for approval in the EU by CHMP for familial chylomicronemia syndrome (FCS)
– Recommendation based on Phase 3 Balance results, which showed a significant reduction of triglycerides and substantial reduction of acute pancreatitis
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Recommendation based on Phase 3 Balance results, which showed a significant reduction of triglycerides and substantial reduction of acute pancreatitis events with TRYNGOLZA –\n\n\n– European Commission decision expected by Q4 2025 –\n\n\n CARLSBAD, Calif.--(BUSINESS WIRE)--\nIonis 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.\n\n\n“Building on the strong early launch of TRYNGOLZA in the U.S., the positive CHMP opinion advances our commitment to expand access to TRYNGOLZA globally,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “TRYNGOLZA has demonstrated significant reductions in triglycerides and substantial reductions in acute pancreatitis events with favorable safety and tolerability. With this robust clinical profile, combined with Sobi’s deep commercial market expertise in FCS, TRYNGOLZA has the potential to make a meaningful difference for people living with FCS in the EU.”\n\n\nThe 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).\n\n\nFCS 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","length":3131,"tagName":"div"}]