Ionis Pharmaceuticals announces FDA acceptance of olezarsen NDA

Ionis Pharmaceuticals announced that the U.S. Food and Drug Administration has accepted for Priority Review the New Drug Application for olezarsen, an investigational RNA-targeted medicine for the treatment of adults with familial chylomicronemia syndrome. The FDA has set an action date of December 19, 2024 and indicated they are not currently planning an advisory committee meeting for olezarsen. he application to the FDA was based on positive results from Balance, a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 study. The study results were presented at the 2024 American College of Cardiology Annual Meeting and published simultaneously in The New England Journal of Medicine. The U.S. FDA granted olezarsen Fast Track designation for the treatment of FCS in January 2023, as well as Orphan Drug designation and Breakthrough Therapy designation in February 2024. Ionis is planning additional regulatory filings for the treatment of FCS in the European Union this year.