Sobi has announced that the European Medicines Agency (EMA) has validated its application to expand the indication of Tryngolza (olezarsen) for the treatment of adults with severe hypertriglyceridemia (sHTG), defined by triglyceride levels ≥880 mg/dL (≥10 mmol/L). The move marks a significant step in broadening the therapeutic scope of its RNA-targeted medicine portfolio.
The submission is supported by positive data from the Phase III CORE and CORE2 trials, published in the New England Journal of Medicine in 2025. These studies demonstrated the efficacy and safety of olezarsen in reducing triglyceride levels in patients already receiving standard-of-care therapies.
Tryngolza is currently the only pharmacological therapy to show a reduction in the risk of acute pancreatitis in patients with sHTG, a condition associated with elevated risks of cardiovascular events, hospitalisation, and mortality. Despite existing treatments and lifestyle interventions, many patients continue to face inadequate triglyceride control.
Olezarsen, developed by Ionis Pharmaceuticals, works by reducing the production of apoC-III, a liver protein that regulates triglyceride metabolism. Under a licensing agreement, Sobi holds exclusive commercialisation rights for Tryngolza in ex-US markets, excluding Canada and China.
The therapy was previously approved in the European Union in September 2025 as an adjunct to diet for adults with familial chylomicronemia syndrome (FCS). The current application reflects growing clinical evidence supporting its broader use in severe lipid disorders.
Severe hypertriglyceridemia affects approximately 2 million individuals across the EU5, with around 700,000 patients experiencing triglyceride levels ≥880 mg/dL. The condition significantly increases the risk of acute pancreatitis, a potentially life-threatening disease requiring intensive medical care.
For pharma manufacturing, the development underscores rising demand for advanced RNA-based therapeutics and highlights the growing importance of scalable, precision-driven production capabilities. Sobi continues to strengthen its rare disease pipeline by leveraging innovative technologies aimed at improving outcomes for patients with complex conditions.
