Sobi's Tryngolza Receives EMA Validation for sHTG Treatment Indication Extension

Sobi® has made an important announcement regarding its novel therapy, Tryngolza® (olezarsen), which has been validated by the European Medicines Agency (EMA) for an extension of its indication. This application pertains to the treatment of adult patients suffering from severe hypertriglyceridemia (sHTG) with triglyceride levels of 880 mg/dL or above (≥10 mmol/L). Elevated triglycerides are a significant health concern, as they correlate with increased risks of severe health complications including all-cause mortality, acute pancreatitis, and cardiovascular events.

The support for this submission is derived from pivotal Phase 3 studies, CORE and CORE2, which were published in the New England Journal of Medicine in 2025. These trials provided robust evidence about the beneficial effects of olezarsen, positioning it as a critical option in the management of this severe condition.

Dr. Lydia Abad-Franch, Sobi's Chief Medical Officer, emphasized the uniqueness of Tryngolza in its ability to reduce the risk of acute pancreatitis among patients with sHTG: "Tryngolza is the only pharmacological therapy to demonstrate a significant reduction in the risk of acute pancreatitis in patients with severe hypertriglyceridemia. If approved, it could transform treatment approaches and alleviate the burden that chronic hospital visits and severe symptoms impose on patients."

The strategic importance of this application cannot be overstated as it encapsulates Sobi's commitment to providing innovative therapeutic solutions for those living with rare and severe diseases. It reflects a growing body of clinical evidence that underscores the necessity of more effective treatment options for sHTG, a condition that currently affects around 2 million individuals across the EU, with approximately 700,000 of them having dangerously high triglyceride levels.

Understanding Severe Hypertriglyceridemia (sHTG)

Severe hypertriglyceridemia is characterized by triglyceride levels exceeding 500 mg/dL and presents a medical emergency when levels reach or exceed 880 mg/dL. Patients suffering from this condition face increased risks including acute pancreatitis, a serious disorder that can result in debilitating pain, lengthy hospital stays, and long-term organ damage. Unfortunately, existing therapies and lifestyle recommendations have proven insufficient in effectively lowering triglyceride levels for every patient.

In the CORE and CORE2 studies, patients with triglyceride levels ≥500 mg/dL were randomly assigned to receive various dosages of olezarsen or placebo. The results were promising, showing a substantial decrease in triglyceride levels, thus emphasizing its potential utility in clinical practice. The trials involved 617 and 446 participants respectively and were conducted globally across multiple centers.

The Science Behind Olezarsen

Olezarsen is an RNA-targeted medicine designed to lower the production of apoC-III, a liver protein crucial in regulating triglyceride metabolism. Its efficacy has already been demonstrated in managing familial chylomicronemia syndrome, with regulatory approvals already received in the United States and Canada.

By entering into a licensing agreement with Ionis Pharmaceuticals, Sobi now holds exclusive commercialization rights for Tryngolza in countries beyond the U.S., excluding China and Canada. This further reinforces Sobi’s strategy to cater to the medical needs of patients across the globe who may benefit from this innovative treatment.

Conclusion

The EMA's validation is a key milestone for Sobi and the broader medical community striving to enhance care for patients with severe hypertriglyceridemia. Should the application ultimately lead to approval, the introduction of Tryngolza could represent a transformative step forward in the management of this complex condition and significantly improve the quality of life for affected patients. As the rhythm of clinical research progresses, the hope is that such breakthroughs will become standard lines of defense against severe metabolic disorders.