TRYNGOLZA™ (olezarsen) U.S. Approval: First Familial Chylomicronemia Syndrome Treatment for Adults

CARLSBAD, CA, USA I December 19, 2024 – Ionis Pharmaceuticals, Inc. has announced the U.S. Food and Drug Administration's (FDA) approval of TRYNGOLZA™ (olezarsen) for adults with familial chylomicronemia syndrome (FCS). This marks a significant milestone, as TRYNGOLZA becomes the first FDA-approved medication to substantially lower triglyceride levels in those affected by this rare genetic condition, which is characterized by severe hypertriglyceridemia (sHTG) and can lead to life-threatening acute pancreatitis (AP).

TRYNGOLZA is designed to be used alongside a strict diet of no more than 20 grams of fat per day. The drug is self-administered through an auto-injector once a month. Dr. Brett P. Monia, CEO of Ionis, highlighted this development as a pivotal moment not only for patients but also for Ionis, marking its transition into a fully integrated commercial-stage biotechnology firm. Monia expressed gratitude to the FCS community and clinical trial participants who played a role in bringing this treatment to fruition.

The FDA's decision was based on the successful outcomes of the global Phase 3 Balance clinical trial. This study was a multicenter, randomized, placebo-controlled, double-blind trial involving adults with genetically confirmed FCS and triglyceride levels of 880 mg/dL or higher. Participants treated with TRYNGOLZA showed a significant mean reduction in triglyceride levels, with a 42.5% decrease after six months and a 57% reduction after 12 months compared to the placebo group. Additionally, the incidence of AP was considerably reduced, with only one patient in the TRYNGOLZA group experiencing an AP episode, compared to seven in the placebo group.

Safety profiles for TRYNGOLZA were favorable, with the most common side effects being injection site reactions, decreased platelet count, and joint pain. Results from the Balance study have been published in The New England Journal of Medicine.

Dr. Alan Brown, a clinical professor and trial investigator, noted the significance of this approval as it provides a long-awaited treatment option for FCS patients who previously relied solely on rigorous dietary restrictions to manage their condition. FCS affects roughly 3,000 individuals in the U.S., with many remaining undiagnosed. This condition, caused by impaired lipoprotein lipase (LPL) function, leads to extremely high triglyceride levels and frequent AP episodes, along with chronic fatigue and abdominal pain.

The approval of TRYNGOLZA offers new hope for those living with FCS. Lindsey Sutton Bryan, co-founder of the FCS Foundation, emphasized the transformative impact this treatment could have on patients who have struggled with severe pain and fatigue throughout their lives.

Ionis Pharmaceuticals is committed to supporting the FCS community through its Ionis Every Step™ program, providing resources and services such as patient education and insurance assistance. TRYNGOLZA has received various designations, including Fast Track, Orphan Drug, and Breakthrough Therapy, highlighting its importance and potential impact.

This approval reflects Ionis's commitment to leveraging RNA-targeted therapies to address unmet needs in rare diseases. With a robust pipeline in neurology, cardiology, and other fields, Ionis aims to continue delivering innovative solutions for serious health conditions.