TRYNGOLZA® (olezarsen) approved in the European Union for familial chylomicronemia syndrome (FCS)

About this update from Ionis Pharmaceuticals, Inc.

[{"type":"text","content":" CARLSBAD, Calif.--(BUSINESS WIRE)--\nIonis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced 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.\n\n\nThe 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. Study results were published in The New England Journal of Medicine (NEJM).\n\n\n“The EU approval of TRYNGOLZA is a significant advance for the treatment of FCS,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “TRYNGOLZA has the potential to be a transformative treatment option for FCS patients in the EU who are at risk of debilitating and life-threatening acute pancreatitis attacks. We are proud to work with Sobi, a long-standing partner of Ionis and the FCS community, to make TRYNGOLZA available to people with FCS in the EU.”\n\n\nFCS 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.\n\n\n“TRYNGOLZA is the next step in our support for the FCS community in Europe. It has a strong safety and efficacy profile, with significant reductions in triglyceride levels and a notable decrease in acute pancreatitis events, which impact morbidity, mortality and quality of life,” said Lydia Abad-Franch, M.D., MBA, head of research, development, and medical affairs (RDMA) and chief medical officer at Sobi. “This builds on our commitment to FCS that began with Waylivra (volanesorsen), the only approved treatment for FCS in Europe until now. With the European Commission’s approval of TRYNGOLZA for FCS, we are ...

More updates from Ionis Pharmaceuticals, Inc.