Triglyceride Drug Approved to Treat Adults with FCS
- By BSTQ Staff
Tryngolza (olezarsen) has been approved by the U.S. Food and Drug Administration to be used with diet to reduce triglycerides (TG) in adults with familial chylomicronemia syndrome (FCS), a rare, genetic disorder that prevents the body from breaking down fats (TG) in the bloodstream. This is a first-in-class approval, meaning Tryngolza uses a new mechanism of action, or works differently in the body, than other therapies currently used to treat FCS. Tryngolza is injected subcutaneously (under the skin) once per month.
The efficacy and safety of Tryngolza were evaluated in a randomized, placebo-controlled, double-blind clinical trial (NCT04568434) in 66 adult patients with FCS and fasting TG levels of at least 880 mg/dL (the average baseline TG level was approximately 2600 mg/dL). The primary endpoint was percent change in fasting TG levels from baseline to month six (average of weeks 23, 25 and 27) compared to placebo.
The average percent change in TG from baseline to month six in the Tryngolza treatment group was -42.5 percent compared to the placebo group. Median percent and absolute changes in TG levels from baseline over time demonstrated a consistent lowering effect during the 12-month treatment period.
The most common adverse reactions in patients treated with Tryngolza were injection site reactions, decreased platelet count and joint pain or stiffness (arthralgia).
References
FDA Approves Drug to Reduce Triglycerides in Adult Patients with Familial Chylomicronemia Syndrome. U.S. Food and Drug Administration news release, Dec. 19, 2024. Accessed at www.fda. gov/drugs/news-events-human-drugs/fda-approves-drug-reduce-triglycerides-adult-patients-familial-chylomicronemia-syndrome.
