EU Approves Tryngolza® for Familial Chylomicronemia Syndrome

Sobi® (STO: SOBI) and Ionis Pharmaceuticals announced that Tryngolza® (olezarsen) has been approved in the European Union for adults with genetically confirmed familial chylomicronemia syndrome (FCS).

The decision follows a positive opinion from the Committee for Medicinal Products for Human Use.

Clinical Evidence: The Balance Study

Approval was based on results from the Phase 3 Balance study.

• Primary endpoint: Tryngolza 80 mg significantly reduced fasting triglyceride levels at six months versus placebo.
• Durability: Reduction was sustained through 12 months.
• Secondary endpoint: Substantial, clinically meaningful reduction in acute pancreatitis events over 12 months.
• Safety: Favorable safety and tolerability profile.
• Publication: Results appeared in The New England Journal of Medicine.

Expert Perspectives

Dr. Lydia Abad-Franch, Chief Medical Officer at Sobi, said:

“Tryngolza has a strong efficacy and safety profile, lowering triglycerides and reducing acute pancreatitis events. This represents a major advance for FCS care.”

She noted that Sobi’s FCS commitment began with Waylivra (volanesorsen), the first approved treatment in Europe.

Dr. Brett P. Monia, CEO of Ionis, added:

“Tryngolza could transform treatment for EU patients at risk of life-threatening pancreatitis attacks. We are proud to partner with Sobi.”

About Familial Chylomicronemia Syndrome

• FCS is a rare genetic form of severe hypertriglyceridemia (sHTG).
• Caused by impaired lipoprotein lipase (LPL) function.
• Leads to extremely high triglyceride levels and risk of acute pancreatitis, a potentially fatal complication.
• Patients often suffer chronic issues: fatigue, abdominal pain, disability, and reduced quality of life.
• In the EU, FCS affects up to 13 people per million.

About Tryngolza® (olezarsen)

• RNA-targeted therapy designed to reduce apolipoprotein C-III, a key triglyceride regulator.
• Approved in the U.S. and EU for adults with FCS.
• Administered as an 80 mg subcutaneous injection once monthly via pre-filled autoinjector.
• Being evaluated for severe hypertriglyceridemia (sHTG) with triglyceride levels ≥500 mg/dL.
• Positive topline results from Phase 3 sHTG trials were announced in September 2025.

The Balance Study Design

• Global, multicenter, randomized, double-blind, placebo-controlled Phase 3 trial.
• Duration: 12 months.
• Enrolled patients with FCS.
• Primary endpoint: percent change in triglycerides at six months.
• Secondary endpoints: triglycerides at 12 months, lipid parameter changes, and acute pancreatitis events.
• Patients could join an open-label extension for ongoing treatment.

About Sobi

Sobi is a global biopharma focused on rare diseases.

• Workforce: ~1,900 employees across Europe, North America, Middle East, Asia, Australia.
• 2024 revenue: SEK 26 billion.
• Listed on Nasdaq Stockholm (STO: SOBI).
• Learn more at sobi.com and LinkedIn.

Commercialization Rights

Sobi has exclusive rights to commercialize Tryngolza outside the U.S., Canada, and China.

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!