Sobi's TRYNGOLZA® Receives Positive Opinion for sHTG Treatment in Europe

Sobi's TRYNGOLZA® Gains Positive CHMP Opinion for sHTG Treatment

In a significant breakthrough, Sobi® announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion endorsing the extension of indications for their investigational drug, TRYNGOLZA® (olezarsen). This recommendation aims to offer new hope for adult patients battling severe hypertriglyceridemia (sHTG), especially those with fasting triglyceride levels reaching 880 mg/dL (≥10 mmol/L).

Severe hypertriglyceridemia is not just a medical term; it represents a potentially life-threatening condition that poses serious health risks, including acute pancreatitis. This complication occurs when triglyceride levels exceed a critical threshold, substantially increasing the chances of experiencing a medical emergency. According to estimates, nearly 700,000 individuals within major EU markets are grappling with triglyceride levels at or above this perilous mark, highlighting the urgent need for effective treatment options.

The positive opinion from CHMP is anchored in robust clinical data derived from the pivotal Phase 3 CORE and CORE2 trials, conducted in partnership with the TIMI Study Group. These studies revealed that TRYNGOLZA® achieved a remarkable placebo-adjusted mean reduction in fasting triglyceride levels, with reductions reaching up to 72% at the six-month mark—effects that were sustained through the one-year follow-up.

Furthermore, participants benefited from a significant 85% reduction in adjudicated acute pancreatitis events compared to those receiving placebo. These outcomes underscore the potential of TRYNGOLZA® not only to lower triglyceride levels, but also to mitigate serious clinical complications associated with sHTG.

During the American Heart Association's Scientific Sessions 2025, findings from CORE and CORE2 were published in the esteemed New England Journal of Medicine (NEJM), solidifying the credibility of the results. Lydia Abad-Franch, MD, Head of RD and Medical Affairs and Chief Medical Officer at Sobi, expressed that this positive opinion is a direct reflection of the clinical evidence gathered, and it brings us closer to making TRYNGOLZA® a reality for patients at risk of acute pancreatitis associated with sHTG. Such complications not only involve hospital stays but can also lead to irreversible damage, significantly affecting the quality of life of individuals concerned.

In the case of sHTG, current practice guidelines advocate for interventions when triglyceride levels surpass 880 mg/dL due to the heightened risk of developing acute pancreatitis—an often critical medical emergency that can demand intensive care and extend hospital admissions.

The promising recommendation from the CHMP is viewed as a pivotal development by executives at Sobi and Ionis Pharmaceuticals, the company that initially discovered and developed TRYNGOLZA. Brett P. Monia, Ph.D., the Chief Executive Officer of Ionis, remarked on the importance of this decision in expanding access to much-needed treatment for patients in Europe.

TRYNGOLZA® works at the molecular level by targeting apolipoprotein C-III production, a key player in triglyceride metabolism, thereby addressing the very source of the problem. With its current approvals across various regions—including the UK, US, EU, and Canada—it represents a novel therapeutic approach that continues to show promise for those suffering from hypertriglyceridemia-related complications.

As Sobi awaits the final decision from the European Commission, the company remains optimistic about the future of TRYNGOLZA® and its impact on patients living with sHTG. A predicted decision from the EC is expected within the fourth quarter of 2026, which could mark the dawn of a new era in treating this often-neglected condition.

In conclusion, the positive CHMP opinion for TRYNGOLZA® marks a significant milestone in the fight against severe hypertriglyceridemia, paving the way for improved patient outcomes and highlighting the pressing need for effective interventions against life-threatening health complications.