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.
