FDA Approves Ionis' TRYNGOLZA for FCS Treatment

Ionis Pharmaceuticals has achieved a significant milestone with the US Food and Drug Administration (FDA) granting approval for TRYNGOLZA (olezarsen). This innovative therapy is designed to be used alongside dietary interventions to help manage triglyceride levels in adults diagnosed with familial chylomicronaemia syndrome (FCS), a hereditary condition that leads to extremely high triglyceride levels in the blood. With this approval, TRYNGOLZA becomes the first treatment sanctioned by the FDA specifically targeting triglyceride reduction in adults, marking a new horizon for patients affected by this challenging genetic disorder.

FCS is known for its association with severe hypertriglyceridemia, which can often lead to acute pancreatitis (AP), a painful and potentially dangerous condition. TRYNGOLZA offers patients a tangible reduction in the frequency of AP events when combined with a low-fat diet. Administered as a self-injection, the treatment requires only a monthly dose, providing convenience and ease for patients managing this chronic condition.

The FDA's approval was largely based on compelling results from the Phase III Balance trial, a comprehensive study that tested the efficacy and safety of olezarsen. This double-blind, multicentre trial was conducted on a global scale, involving adult participants with genetically confirmed FCS, all of whom had fasting triglyceride levels exceeding 880 mg/dL. The study found that TRYNGOLZA not only significantly reduced triglyceride levels but also substantially cut down the incidence of acute pancreatitis episodes among participants.

Brett Monia, CEO of Ionis Pharmaceuticals, highlighted the importance of this approval, stating that it represents a groundbreaking advancement for patients with FCS and their families. Monia emphasized that this approval is a transformative moment, offering patients a treatment that effectively decreases triglycerides and mitigates the risk of debilitating and potentially life-threatening acute pancreatitis. For Ionis Pharmaceuticals, the approval signifies a pivotal step in the company’s growth, marking its transition into a fully integrated commercial-stage biotechnology firm—an objective the company has been striving towards.

TRYNGOLZA received priority review status from the FDA and was previously awarded several designations, including fast track, orphan drug, and breakthrough therapy, underscoring its potential impact and urgency of need within the patient community. The company has also confirmed that TRYNGOLZA will be accessible in the US market before the close of 2024.

Beyond the US, olezarsen is currently under regulatory review in the European Union, and Ionis has plans for regulatory submissions in other countries as well. In addition to its use for FCS, olezarsen is also undergoing evaluation in three other Phase III clinical trials—named CORE, CORE2, and ESSENCE—for the treatment of severe hypertriglyceridemia. However, it is important to note that it has not yet received regulatory approval for these additional indications.

This landmark achievement by Ionis Pharmaceuticals brings new hope to those living with familial chylomicronaemia syndrome and represents a significant advance in the treatment of hypertriglyceridemia, promising to improve the quality of life for many patients worldwide.