FDA Approves Tryngolza for Familial Chylomicronemia Syndrome

On December 27, 2024, Ionis Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted approval for Tryngolza (olezarsen), a treatment designed to lower triglycerides in adults with familial chylomicronemia syndrome (FCS). FCS is known to cause high levels of triglycerides, which can result in acute pancreatitis, a condition that poses serious health risks.

Tryngolza has demonstrated significant efficacy in reducing triglyceride levels in individuals with FCS, when used in combination with a diet containing no more than 20 grams of fat per day. This medication is administered by patients themselves once a month using an autoinjector. The FDA's decision to approve this treatment was based on findings from the extensive, global Balance clinical trial, a phase 3 study that was both randomized and placebo-controlled. The trial focused on adults diagnosed with FCS, who had fasting triglyceride levels of 880 mg/dL or higher.

Participants in the study showed substantial improvements. Within six months of treatment, there was a notable average reduction in triglyceride levels by 42.5 percent, compared to those in the placebo group. After a year, this reduction increased to an average of 57 percent. Alongside the decrease in triglyceride levels, there was also a significant drop in acute pancreatitis incidents. Only one patient, constituting 5 percent of those receiving Tryngolza, experienced an episode of acute pancreatitis. In contrast, 30 percent of participants in the placebo group experienced a total of 11 such episodes.

The trial results also indicated a favorable safety profile for Tryngolza, suggesting that it not only effectively manages triglyceride levels but also does so safely. This advancement marks a significant milestone for those with FCS, offering a new therapeutic option that mitigates the risk of severe pancreatitis.

Brett P. Monia, Ph.D., CEO of Ionis Pharmaceuticals, expressed that this approval represents a critical development for FCS patients, providing them with an effective means to manage triglyceride levels and reduce the risk of acute pancreatitis, a condition that can severely impact quality of life.

Overall, the approval of Tryngolza signifies a hopeful advancement for those affected by familial chylomicronemia syndrome, offering a new method to manage this challenging condition.