Ionis Pharmaceuticals Receives Positive CHMP Opinion for Tryngolza Approval

Ionis Pharmaceuticals has announced that the European Medicines Agency's Committee for Medicinal Products for Human Use has adopted an opinion for Tryngolza, an adjunct to diet for treating genetically confirmed familial chylomicronemia syndrome in adult patients. The positive opinion is now referred to the European Commission for approval, with a decision expected by Q4.

Title: Ionis Pharmaceuticals and Sobi Secure Positive CHMP Opinion for Tryngolza in EU

Ionis Pharmaceuticals, Inc. (IONS) and Sobi have announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for TRYNGOLZA (olezarsen) as an adjunct to diet for treating genetically confirmed familial chylomicronemia syndrome (FCS) in adult patients. The positive opinion is now referred to the European Commission for approval, with a decision expected by the fourth quarter of 2025.

The CHMP opinion is based on positive data from the Phase 3 Balance study, which demonstrated statistically significant reductions in triglyceride levels at six months and maintained these reductions for up to 12 months. Additionally, TRYNGOLZA showed a substantial and clinically meaningful reduction in acute pancreatitis events over 12 months. TRYNGOLZA was previously approved in the United States in December 2024.

FCS is a rare and genetic disorder characterized by extremely high levels of triglycerides, leading to recurrent episodes of acute pancreatitis. The condition is estimated to impact up to 13 people per million in the EU.

"This positive CHMP opinion advances our commitment to expand access to TRYNGOLZA globally," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "TRYNGOLZA has demonstrated significant reductions in triglycerides and substantial reductions in acute pancreatitis events with favorable safety and tolerability."

Sobi, which has exclusive rights to commercialize TRYNGOLZA outside the U.S., Canada, and China, will leverage its existing market expertise and distribution channels to facilitate an effective launch of TRYNGOLZA in the EU if approved.

Lydia Abad-Franch, M.D., MBA, head of research, development, and medical affairs and chief medical officer of Sobi, commented, "The approval recommendation brings us one step closer to delivering TRYNGOLZA to people living with FCS in the EU. We believe TRYNGOLZA has the potential to be an important treatment for people living with this rare and serious disease."

TRYNGOLZA is an RNA-targeted medicine designed to lower the body's production of apoC-III, a protein that regulates triglyceride metabolism. It is not yet approved for any indication in Europe.

References:
[1] https://www.nasdaq.com/articles/ionis-pharma-sobi-get-positive-chmp-opinion-tryngolza-familial-chylomicronemia-syndrome
[2] https://www.morningstar.com/news/business-wire/20250724782589/tryngolza-olezarsen-recommended-for-approval-in-the-eu-by-chmp-for-familial-chylomicronemia-syndrome-fcs