Olezarsen (Tryngolza; Ionis) has been approved by the FDA for the treatment of familial chylomicronemia syndrome (FCS), a rare, inherited disorder characterized by severe hypertriglyceridemia.
FDA officials based their decision in part on results from the double-blind, phase 3 BALANCE trial (NCT04568434), which demonstrated that olezarsen greatly reduced fasting trigylyceride (TG) levels compared with placebo from baseline to 6 months in 66 patients with FCS and fasting TG levels of at least 880 mg/dL.
The average percent change in TG from baseline to month 6 in the Tryngolza treatment group was –42.5% compared with the placebo group, according to a statement from the FDA. Median percent and absolute changes in TG levels from baseline over time demonstrated a consistent lowering effect during the 12-month treatment period.
This is a first-in-class approval, making Tryngolza the first agent available in the United States to treat FSC.
“With no treatment options previously available, we were limited to relying only on extremely strict diet and lifestyle changes as the sole preventative treatment option,” Alan Brown, MD, FNLA, FACC, FAHA, clinical professor of medicine, Rosalind Franklin University of Medicine and Science, and BALANCE trial investigator, said a in press release about the approval. “The FDA approval of Tryngolza is an important moment for people living with FCS, their families and physicians who now, for the first time, have a treatment that significantly lowers triglycerides and decreases the risk of potentially life-threatening acute pancreatitis events, as an adjunct to a low-fat diet. I am excited to have a medicine I can prescribe to my patients that has been shown to change the course of their disease.”
Reference
- Ionis. Tryngolza (olezarsen) approved in US as first-ever treatment for adults living with familial chylomicronemia syndrome as an adjunct to diet. Press release. Published December 19, 2024. Accessed January 2, 2025. https://www.multivu.com/ionis-pharmaceuticals/9295551-en-tryngolza-olezarsen-fda-approval