FDA approves Tryngolza as adjunct to diet for familial chylomicronemia syndrome

Tryngolza (olezarsen) has been approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), which can lead to potentially life-threatening acute pancreatitis (AP), according to a press release issued by Ionis Pharmaceuticals.

Data have shown Tryngolza significantly and substantially reduces triglyceride levels in adults with FCS and reduces AP events when used with a diet including ≤20 g fat per day. Tryngolza is self-administered once monthly via an autoinjector.

The FDA approval was based on data from the global, multicenter, randomized, placebo-controlled phase 3 Balance clinical trial involving adults with genetically identified FCS and fasting triglyceride levels ≥880 mg/dL. From baseline to six months, there was a significant placebo-adjusted mean reduction in triglyceride levels of 42.5 percent. From baseline to 12 months, reductions were further improved, with a placebo-adjusted 57 percent mean reduction in triglycerides. Over 12 months, there was a clinically meaningful reduction in AP events; one patient (5 percent) experienced one AP event in the Tryngolza group, compared with seven (30 percent) who experienced 11 episodes in the placebo group. A favorable safety profile was seen for Tryngolza.

“For the first time, adults with FCS can now access a treatment that substantially reduces triglycerides and the risk of debilitating and potentially life-threatening acute pancreatitis,” Brett P. Monia, Ph.D., the chief executive officer of Ionis, said in a statement.

Approval of Tryngolza was granted to Ionis Pharmaceuticals.

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