Ionis Pharmaceuticals (IONS) Announces FDA Approval of Tryngolza™ for Treatment of Familial Chylomicronemia Syndrome

On December 19, 2024, Ionis Pharmaceuticals, Inc. made a significant announcement about the approval of Tryngolza™ (olezarsen) by the U.S. Food and Drug Administration (FDA). This approval marks a groundbreaking milestone as Tryngolza™ is now the first-ever treatment approved for adults suffering from familial chylomicronemia syndrome (FCS) in the United States as an adjunct to their diet regimen.

FCS is a rare genetic form of severe hypertriglyceridemia that can result in life-threatening acute pancreatitis events. Tryngolza™ has demonstrated effective reduction in triglycerides and a substantial decrease in acute pancreatitis occurrences when used in conjunction with a low-fat diet (<20 grams of fat per day). This self-administered treatment, administered monthly through an auto-injector, has been a result of Ionis’ long-standing commitment to the FCS community and collaboration with patients, families, and researchers. The FDA approval was established based on the positive outcomes from the global Phase 3 Balance clinical trial, a randomized, placebo-controlled study in adults with genetically confirmed FCS and elevated fasting triglyceride levels. Tryngolza™ exhibited a notable reduction in triglyceride levels alongside a significant decrease in acute pancreatitis events over a period of 12 months. Ionis Pharmaceuticals views the FDA approval of Tryngolza™ as a transformative event for patients, signifying a crucial advancement in the treatment landscape for FCS. This achievement also reflects Ionis’ evolution into a fully integrated commercial-stage biotechnology company, ready to deliver innovative medicines to individuals battling serious illnesses. In addition to the FDA approval in the U.S., Ionis is currently pursuing regulatory approvals for olezarsen in other regions. Tryngolza™, the first in a series of anticipated launches, is slated to be available to patients in the U.S. before the end of the year. Ionis aims to provide comprehensive support through its Ionis Every Step™ program, assisting patients with personalized services and resources to navigate their treatment journey effectively. Ionis Pharmaceuticals will hold a webcast at 6:45 pm ET today to discuss the implications and significance of the FDA approval. Interested parties can access the webcast through their official website. For more detailed information about Tryngolza™ and the FDA approval process, please visit the official Ionis Pharmaceuticals website or consult the Prescribing Information for Tryngolza™.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Ionis Pharmaceuticals’s 8K filing here.

About Ionis Pharmaceuticals

Ionis Pharmaceuticals, Inc discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy.