Ionis announces olezarsen FCS New Drug Application accepted for Priority Review and enrollment in Phase 3 sHTG program completed

Core Insights - Ionis Pharmaceuticals has completed Phase 3 enrollment for olezarsen, targeting severe hypertriglyceridemia, with results anticipated in the second half of 2025 [1][3] - The FDA has accepted the New Drug Application for olezarsen for familial chylomicronemia syndrome (FCS) under Priority Review, with an action date set for December 19, 2024 [1][12] - Olezarsen is designed to lower apoC-III production, a protein that regulates triglyceride metabolism, and is being evaluated for both FCS and severe hypertriglyceridemia [4][12] FDA Review Process - Priority Review designation indicates the potential for significant improvements in treatment, with FDA action expected within six months [2] - The application is based on positive results from the Balance Phase 3 study, presented at the 2024 ACC Annual Meeting and published in NEJM [2] Clinical Trials and Patient Demographics - Three Phase 3 trials (CORE, CORE2, ESSENCE) for severe hypertriglyceridemia have enrolled over 1,000 patients with triglyceride levels ≥500 mg/dL and over 1,400 patients with levels between 150 mg/dL and <500 mg/dL [3][7] - Severe hypertriglyceridemia affects over three million people in the U.S., posing risks of pancreatitis and cardiovascular issues [7][17] Market Need and Treatment Landscape - There are currently no FDA-approved therapies for FCS, and patients rely on restrictive diets for management [6][15] - Existing treatment guidelines recommend lifestyle changes and medications for triglyceride levels ≥500 mg/dL, but many patients require additional therapeutic options [17] Company Overview - Ionis Pharmaceuticals has a strong pipeline in RNA-targeted medicines and has developed five marketed medicines, focusing on serious diseases [18]