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The U.S. Food and Drug Administration (FDA) has approved Tryngolza (olezarsen) as an adjunct to diet for the treatment of familial chylomicronemia syndrome (FCS), a rare genetic disorder characterized by extremely high triglyceride levels that can lead to potentially life-threatening acute pancreatitis (AP). The announcement was made in a press release from Ionis Pharmaceuticals, the developer of Tryngolza.
This milestone approval provides the first targeted therapy for adults with FCS, offering significant and sustained triglyceride reduction while also lowering the risk of acute pancreatitis when combined with a low-fat diet (≤20 g fat per day). Tryngolza is designed for monthly self-administration using an autoinjector, providing a convenient treatment option for patients managing this challenging condition.
Clinical Trial Results
The FDA’s decision was based on data from the Balance phase 3 clinical trial, a global, multicenter, randomized, placebo-controlled study. Participants included adults with genetically confirmed FCS and fasting triglyceride levels of at least 880 mg/dL.
Key findings from the trial included:
- Six-month results: A placebo-adjusted mean triglyceride reduction of 42.5 percent.
- 12-month results: A further placebo-adjusted mean triglyceride reduction of 57 percent.
- Acute pancreatitis outcomes: Over 12 months, the Tryngolza group experienced significantly fewer AP events. Only one patient (5 percent) in the treatment group had an AP episode, compared to 11 episodes reported by seven patients (30 percent) in the placebo group.
Tryngolza also demonstrated a favorable safety profile, making it a promising treatment option for FCS patients.
A Transformative Treatment Option
“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,” said Brett P. Monia, Ph.D., CEO of Ionis Pharmaceuticals. “This approval represents a significant step forward for the FCS community, which has long faced limited treatment options.”
FCS is an ultra-rare disorder caused by genetic mutations that disrupt the normal breakdown of triglycerides, leading to extreme lipid levels and frequent episodes of AP. Prior to the approval of Tryngolza, treatment options were limited to strict dietary management and supportive care during AP episodes.
Looking Ahead
With FDA approval, Ionis Pharmaceuticals will now focus on ensuring the availability of Tryngolza to patients in need. The treatment’s approval marks a turning point for individuals living with FCS, offering hope for improved quality of life and reduced disease burden.
This advancement reflects the growing potential of targeted therapies to address unmet needs in rare genetic disorders.
