Recommendation for TRYNGOLZA® (olezarsen) Approval for FCS in the EU

TRYNGOLZA® Receives Positive Opinion from CHMP for FCS Approval

In a significant development for patients suffering from familial chylomicronemia syndrome (FCS), the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for TRYNGOLZA® (olezarsen) as a supplementary treatment designed to lower triglyceride levels in adults diagnosed with this rare genetic condition. This milestone brings hope to a community grappling with the often debilitating symptoms of FCS, which includes the risk of severe complications like acute pancreatitis.

Clinical Breakthrough

The positive opinion follows successful outcomes from the Phase 3 Balance study. This global, multicenter trial demonstrated that TRYNGOLZA significantly reduced triglyceride levels after six months of treatment, with sustained results observed at twelve months. Additionally, there was a notable decrease in the occurrence of acute pancreatitis among participants, a life-threatening condition that often requires emergency medical care.

Lydia Abad-Franch, MD, MBA, who leads Research, Development, and Medical Affairs at Sobi, emphasized the importance of this recommendation, stating, "The approval recommendation brings us one step closer to delivering TRYNGOLZA to individuals affected by FCS in the EU and symbolizes our ongoing commitment to this patient community. Patients with FCS face serious health risks, and we firmly believe that TRYNGOLZA could alleviate some of these burdens."

Targeting FCS

Familial chylomicronemia syndrome is characterized by extreme elevations in triglyceride levels due to genetic factors that impair lipid metabolism. Individuals with this condition frequently experience triglyceride levels exceeding 880 mg/dL, compared to normal range levels below 150 mg/dL. This impairment makes them highly susceptible to acute pancreatitis, among other complications. Estimates suggest that up to 13 individuals per million in the EU are affected by this serious disease.

As the exclusive developer of TRYNGOLZA outside of the U.S., Canada, and China, Sobi is well-prepared to leverage its expertise in the FCS market. The company previously secured approval for Waylivra, another therapy for this condition, and aims to launch TRYNGOLZA effectively upon final approval.

The Path Forward

Pending a decision from the European Commission, expected by the end of 2025, the focus for Sobi and Ionis will be on rolling out this innovative treatment across Europe. Brett P. Monia, CEO of Ionis Pharmaceuticals, reiterated their commitment to this cause, stating, "With the strong launch of TRYNGOLZA in the U.S. already underway, the favorable opinion from CHMP allows us to focus on providing this important therapy to patients in Europe as well."

Important Safety Information

While the efficacy of TRYNGOLZA is promising, patients should be aware of potential adverse reactions. Reports of injection site reactions, decreased platelet count, and hypersensitivity have been noted among a portion of participants in clinical trials. Additionally, patients are advised to monitor for signs of hypersensitivity, including bronchospasm and facial swelling.

As awareness around FCS increases, the prospective approval of TRYNGOLZA could mark a significant shift in how this serious condition is treated, offering hope for improved quality of life for those affected. Further updates are expected as the European Commission reviews CHMP's recommendation, paving the way for the hopeful introduction of this novel treatment in the EU market.