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Tryngolza®: A New Era for Familial Chylomicronemia Syndrome Treatment

On September 19, 2025, Sobi® and Ionis Pharmaceuticals, Inc. announced significant advancements in the treatment options available for familial chylomicronemia syndrome (FCS), with the approval of Tryngolza® (olezarsen) in the European Union. This milestone represents a critical step forward for patients suffering from this rare genetic disorder characterized by high triglyceride levels and the need for better management of their condition.

Understanding Familial Chylomicronemia Syndrome (FCS)

FCS is a rare genetic condition that results in severe hypertriglyceridemia, posing a high risk of acute pancreatitis, which can be life-threatening. Patients diagnosed with FCS experience extremely elevated triglyceride levels due to the impaired function of lipoprotein lipase (LPL), thus making it difficult for them to process certain fats. These patients often require specialized dietary adjustments and ongoing management due to their condition.

In the European Union, the prevalence of FCS is estimated to affect up to 13 individuals per million. Such statistics underline the urgent need for novel therapies that can improve the quality of life for these individuals, preventing serious complications like pancreatitis.

Tryngolza®: Efficacy and Safety

The approval of Tryngolza® was based on the favorable findings from the Phase 3 Balance study, which showcased significant reductions in fasting triglyceride levels over both six and twelve months of treatment. Specifically, the 80 mg dosage of Tryngolza demonstrated statistically significant efficacy along with a positive safety profile, making it a promising option for managing triglyceride levels in FCS patients.

Dr. Lydia Abad-Franch, Chief Medical Officer at Sobi, emphasized that this approval not only enhances treatment options for the FCS community in Europe but also supports ongoing efforts in combating the serious health risks associated with high triglyceride levels, particularly the frequent occurrence of acute pancreatitis. She noted the success was built upon previous initiatives with Waylivra (volanesorsen), the prior treatment available for FCS in Europe.

The Balance study, a randomized, double-blind, placebo-controlled trial, focused on patients diagnosed with FCS and stretched over a duration of 12 months. This research provided evidence of the treatment’s significant capacity to decrease acute pancreatitis events, further enhancing its profile as a valuable therapeutic approach.

The Path Forward

The introduction of Tryngolza® marks a hopeful chapter for those affected by FCS in the EU. With Sobi holding the exclusive rights to commercialize the drug outside the U.S., Canada, and China, they aim to extend this innovative treatment to patients across Europe that have had limited options until recently.

As clinical evaluations continue, Tryngolza® is also being explored for treating severe hypertriglyceridemia (sHTG) with triglyceride levels that reach ≥500 mg/dL. With encouraging topline results anticipated in future studies, there is optimism that this drug could soon pave the way for improved health outcomes in a broader range of patients.

Conclusion

The approval of Tryngolza® (olezarsen) represents a significant development in the biopharmaceutical landscape, especially for patients grappling with FCS. It signifies dedication from companies like Sobi and Ionis to bring forth innovative solutions aimed at making a tangible difference in the lives of individuals with rare diseases. With ongoing research and supportive clinical data, this new treatment step provides not just hope, but a more manageable future for FCS patients in the EU.