Sobi's Tryngolza (Olezarsen) EU Approval: A Blockbuster in the Making for a Rare Genetic Disorder

In September 2025, Sobi and Ionis Pharmaceuticals secured a landmark approval for Tryngolza (olezarsen) in the European Union for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS), a rare genetic disorder characterized by life-threatening hypertriglyceridemia and recurrent acute pancreatitis. This regulatory milestone, following a positive opinion from the EMA's Committee for Medicinal Products for Human Use (CHMP) in July 2025, positions Tryngolza as a transformative therapy in a niche but high-need market. With robust clinical data, a differentiated mechanism of action, and Sobi's established commercial infrastructure, the drug's blockbuster potential—defined as annual sales exceeding $1 billion—is increasingly within reach, with significant implications for Sobi's growth trajectory.

EU Approval and Clinical Efficacy: A New Standard of Care

The approval of Tryngolza in the EU was underpinned by the Phase 3 Balance study, which demonstrated a statistically significant 77% reduction in fasting triglyceride levels at six months, with sustained efficacy through 12 months. Notably, the study also reported a 100% reduction in acute pancreatitis events compared to placebo, a critical outcome for patients with FCS who face a 15–30% lifetime risk of pancreatitis Tryngolza® (olezarsen) approved in the European Union for familial chylomicronemia syndrome (FCS)^[1]. As an antisense oligonucleotide targeting apolipoprotein C-III (apoC-III), Tryngolza addresses a root cause of triglyceride accumulation, offering a mechanistic advantage over existing therapies like Sobi's Waylivra (volanesorsen), which inhibits lipoprotein lipase.

The drug's once-monthly subcutaneous administration via a pre-filled autoinjector further enhances its patient-centric profile, a key differentiator in a disease where treatment adherence is often challenging due to dietary restrictions and frequent complications Tryngolza | European Medicines Agency (EMA)^[2].

Market Opportunity: Niche but Lucrative

FCS affects approximately 13 individuals per million in the EU, translating to roughly 6,500 patients across the bloc. While small in absolute terms, this population represents a concentrated market with limited therapeutic options. Prior to Tryngolza's approval, Waylivra was the only EU-approved treatment, but its adoption was constrained by complex dosing regimens and safety concerns. Tryngolza's superior efficacy and tolerability profile, coupled with its orphan drug designation, positions it to capture a dominant market share.

The competitive landscape is further tilted in Tryngolza's favor by its potential expansion into severe hypertriglyceridemia (sHTG), a broader indication with an estimated prevalence of 1 in 500 individuals. Positive Phase 3 data for sHTG, expected in September 2025, could unlock a patient population over 40 times larger than FCS, significantly amplifying its commercial potential Ionis Q2 2025 slides: TRYNGOLZA sales surge as pipeline advances^[3].

Financial Projections and Blockbuster Potential

Ionis Pharmaceuticals, which retains commercial rights in the U.S., Canada, and China, reported TRYNGOLZA net sales of $26 million in the first half of 2025, with Q2 sales tripling to $19 million. The company has raised its 2025 revenue guidance to $850 million, with TRYNGOLZA projected to contribute $75–80 million annually. While EU pricing details remain undisclosed, the drug's value-based pricing in the U.S. ($275,000 annually) and its demonstrated clinical benefits suggest a similar premium in Europe, where reimbursement decisions are often tied to health technology assessments Ionis reports second quarter 2025 financial results and highlights^[4].

For Sobi, which holds exclusive EU commercialization rights, Tryngolza represents a strategic inflection point. The company's prior experience with Waylivra—despite its commercial limitations—has equipped it with deep market insights, including payer dynamics and patient access challenges. With a projected EU launch in late 2025, Sobi is well-positioned to replicate Ionis's U.S. success, potentially capturing 60–70% of the FCS market within two years.

Strategic Implications for Sobi

The approval of Tryngolza not only diversifies Sobi's rare disease portfolio but also reinforces its reputation as a leader in RNA-targeted therapies. The drug's success could free up capital for pipeline expansion, particularly in hematology and immunology, where Sobi has ongoing programs. Additionally, the partnership with Ionis—strengthened by the recent expansion of their collaboration—provides a blueprint for future co-development opportunities, enhancing Sobi's long-term innovation pipeline.

However, challenges remain. EU member states may delay reimbursement due to cost-effectiveness debates, and competition from emerging therapies targeting apoC-III or other lipid pathways could intensify. That said, Tryngolza's first-mover advantage, coupled with its robust Phase 3 data, creates a high barrier to entry for rivals.

Conclusion: A Catalyst for Sobi's Growth

Tryngolza's EU approval marks a pivotal moment in the treatment of FCS and a significant win for Sobi. With blockbuster potential fueled by its clinical differentiation, premium pricing, and expansion into sHTG, the drug could drive Sobi's revenue growth by 20–30% annually over the next three years. For investors, this represents a compelling case of a rare disease therapy scaling into a broader market, underpinned by a partnership with Ionis and a regulatory environment increasingly supportive of innovative orphan drugs.