The European Medicines Agency (EMA) has validated an application to expand the approved uses of Tryngolza® (olezarsen) to include the treatment of adult patients with severe hypertriglyceridemia (sHTG), a condition characterized by extremely high levels of triglycerides in the blood. This validation, announced March 30, 2026, by Sobi®, marks a significant step toward potentially offering a recent therapeutic option for individuals at increased risk of serious health complications like pancreatitis and cardiovascular disease. Severe hypertriglyceridemia, defined as triglyceride levels of 880 mg/dL or higher, affects an estimated 700,000 people in the EU5 – France, Germany, Italy, Spain, and the United Kingdom – and represents a substantial, often under-addressed, public health concern.
The application is based on data from the Phase 3 CORE and CORE2 studies, published in the New England Journal of Medicine in 2025. These pivotal trials demonstrated Tryngolza’s efficacy in lowering triglyceride levels and, crucially, reducing the risk of acute pancreatitis, a painful and potentially life-threatening inflammation of the pancreas. Currently, managing sHTG often relies on lifestyle changes and standard therapies that don’t consistently achieve sufficient triglyceride reduction for all patients. The potential approval of this expanded indication could provide a much-needed pharmacological intervention for those who don’t respond adequately to existing treatments.
Understanding Severe Hypertriglyceridemia and its Risks
Hypertriglyceridemia, simply put, means having too many triglycerides – a type of fat – in your blood. While moderate elevations are relatively common, sHTG represents a far more dangerous scenario. Triglyceride levels at or above 880 mg/dL are associated with a buildup of chylomicrons, large particles that carry triglycerides, in the bloodstream. This accumulation isn’t just a matter of high numbers; it directly increases the risk of acute pancreatitis, a medical emergency requiring hospitalization and potentially leading to long-term organ damage. Beyond pancreatitis, elevated triglycerides are as well linked to a higher risk of atherosclerotic cardiovascular events, increasing the likelihood of heart attack and stroke.
“Preventing the first attack of pancreatitis is key,” explains Lydia Abad-Franch, MD, Head of R&D and Medical Affairs and Chief Medical Officer at Sobi. “Tryngolza is the only pharmacological therapy to demonstrate a reduction in the risk of acute pancreatitis in patients with severe hypertriglyceridemia. If approved, it could offer an significant therapeutic option to help prevent this life-threatening condition, which can place a significant burden on patients due to frequent hospitalisations, intensive treatment, persistent symptoms and reduced quality of life.”
How Tryngolza Works and Clinical Trial Results
Tryngolza, developed by Ionis Pharmaceuticals and licensed to Sobi for commercialization outside of the U.S., Canada, and China, is an RNA-targeted therapy. It works by reducing the production of apolipoprotein C-III (apoC-III), a protein produced in the liver that plays a key role in regulating triglyceride metabolism. By lowering apoC-III levels, Tryngolza helps the body clear triglycerides from the bloodstream more effectively. The drug is administered via subcutaneous injection every four weeks.
The CORE (NCT05079919) and CORE2 (NCT05552326) trials, conducted with The TIMI Study Group, involved a total of 1063 participants with sHTG. Participants were already receiving standard of care treatments for high triglycerides. The studies randomized patients to receive either 50 mg or 80 mg of olezarsen, or a placebo, over a 12-month period. The primary endpoint – the percentage change in fasting triglyceride levels from baseline at six months – showed a statistically significant reduction in the olezarsen groups compared to placebo, as detailed in the New England Journal of Medicine publication. Notably, a substantial proportion of participants (43%) had baseline triglyceride levels at or above 880 mg/dL.
Regulatory Pathway and Existing Approvals
The EMA validation confirms the completeness of Sobi’s application and initiates a formal review process. The agency will now assess the data submitted to determine whether the benefits of Tryngolza outweigh the risks for this expanded indication. A decision is expected in the coming months, though the exact timeline remains subject to the EMA’s review schedule.
Tryngolza is already approved in the European Union – since September 2025 – as a treatment for familial chylomicronemia syndrome (FCS), a rare genetic disorder that prevents the body from properly processing fats. It is also approved in the U.S. And Canada for FCS. This new application seeks to broaden its leverage to address the more common, yet still serious, condition of severe hypertriglyceridemia.
Sobi’s commitment to addressing rare diseases is reflected in its approximately 1,900 employees across Europe, North America, the Middle East, Asia, and Australia. In 2025, the company reported revenue of SEK 28 billion, and its shares are listed on Nasdaq Stockholm (STO:SOBI).
Disclaimer: This article provides information for general knowledge and informational purposes only, and does not constitute medical advice. It is essential to consult with a qualified healthcare professional for any health concerns or before making any decisions related to your health or treatment.
The EMA’s review of this application represents a crucial step toward potentially improving the lives of individuals living with severe hypertriglyceridemia. The agency’s decision, anticipated in the coming months, will determine whether Tryngolza can become a valuable tool in preventing the debilitating consequences of this often-overlooked condition. Further updates on the review process will be available on the European Medicines Agency website.
What are your thoughts on this potential new treatment option? Share your comments below, and please share this article with anyone who might uncover it helpful.
