FDA Approves Ionis’s TRYNGOLZA for Familial Chylomicronemia Syndrome, a Rare Metabolic Disorder

Approval Date:
December 20, 20244

Treatment Name:
TRYNGOLZA (olezarsen)

Indication:
Familial chylomicronemia syndrome (FCS), a rare and life-threatening metabolic disorder characterized by extremely high levels of triglycerides.

Significance:
TRYNGOLZA is the first-ever FDA-approved treatment for adults with FCS, significantly reducing triglyceride levels and acute pancreatitis events4.

Clinical Trial:
Approval was based on the Phase 3 Balance clinical trial, which demonstrated a favorable safety profile and efficacy in reducing triglycerides and pancreatitis events4.

Availability:
TRYNGOLZA will be available in the U.S. as an adjunct to diet for adults with FCS, regardless of genetically or clinically confirmed diagnosis4.

Sources:

4. https://ir.ionis.com/news-releases/news-release-details/tryngolzatm-olezarsen-approved-us-first-ever-treatment-adults