TRYNGOLZA™ (olezarsen) approved in U.S. as first treatment for adults with familial chylomicronemia syndrome adjunct to diet

Ionis Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted approval for TRYNGOLZA™ (olezarsen). This approval marks a significant milestone in the treatment of adults diagnosed with familial chylomicronemia syndrome (FCS), a rare and severe genetic disorder characterized by extreme hypertriglyceridemia. The condition can lead to potentially life-threatening acute pancreatitis. TRYNGOLZA becomes the first FDA-approved medication specifically designed to lower triglyceride levels in adults with FCS and substantially reduce acute pancreatitis events when complemented with a low-fat diet.

TRYNGOLZA offers a new hope for those suffering from this debilitating disease, as it is self-administered once a month using an auto-injector. This ease of administration could significantly improve the quality of life for patients who previously had to rely solely on strict dietary restrictions as the primary means of managing their condition.

Brett P. Monia, Ph.D., CEO of Ionis, hailed this approval as a transformative event for patients and their families. Monia expressed gratitude toward the FCS community, patients, families, and investigators involved in clinical trials that made this advancement possible. The approval is a crucial step for Ionis itself, marking its evolution into a commercial-stage biotechnology company.

The FDA’s approval of TRYNGOLZA was supported by data from the Balance clinical trial, a global, multicenter, double-blind study. This Phase 3 trial involved participants with genetically confirmed FCS and fasting triglyceride levels of at least 880 mg/dL. The results demonstrated a significant reduction in triglyceride levels among those treated with TRYNGOLZA. After six months, there was a placebo-adjusted mean reduction of 42.5% in triglyceride levels. This reduction improved to 57% after 12 months. The treatment also resulted in a substantial decrease in acute pancreatitis episodes compared to the placebo group.

The safety profile of TRYNGOLZA was favorable, with the most common side effects being injection site reactions, decreased platelet count, and joint pain. The results of the Balance study have been published in The New England Journal of Medicine.

Alan Brown, M.D., a Balance trial investigator, noted that the approval of TRYNGOLZA represents a pivotal moment for those living with FCS. Until now, the primary approach was managing the disease through rigorous dietary and lifestyle changes. The new treatment offers a significant reduction in triglycerides and decreases the risk of acute pancreatitis, providing a much-needed alternative for patients and physicians.

FCS is a rare genetic disorder that disrupts the body's ability to break down fats due to impaired lipoprotein lipase (LPL) function. This results in elevated triglyceride levels, which can lead to acute pancreatitis, among other health issues like fatigue and severe abdominal pain. The condition affects approximately 3,000 people in the U.S., many of whom remain undiagnosed.

TRYNGOLZA is anticipated to be available in the U.S. by the end of the year. Ionis is dedicated to assisting patients in accessing their prescribed medications by offering comprehensive support services tailored to the FCS community through their Ionis Every Step™ program. This initiative includes insurance and affordability assistance, as well as resources on disease and nutrition education.

TRYNGOLZA was granted Priority Review by the FDA and has also received Fast Track, Orphan Drug, and Breakthrough Therapy designations. The medication is under review in the European Union, and regulatory submissions are planned for other countries. Additionally, olezarsen, the active ingredient in TRYNGOLZA, is being evaluated in three other Phase 3 clinical trials for the treatment of severe hypertriglyceridemia, though it has not yet received regulatory approval for these indications.