What is the story about?
What's Happening?
Ionis Pharmaceuticals has announced positive results from its Phase 3 CORE and CORE2 studies of olezarsen, a treatment for severe hypertriglyceridemia (sHTG). The studies demonstrated a significant reduction in fasting triglycerides and acute pancreatitis events among participants. Olezarsen achieved a placebo-adjusted mean reduction in fasting triglycerides of up to 72% and an 85% reduction in acute pancreatitis events. The studies involved nearly 1,100 patients who were on standard lipid-lowering therapy. Olezarsen showed a favorable safety profile, with injection site reactions being the most common adverse event. Ionis plans to submit a supplemental new drug application to the FDA by the end of the year.
Why It's Important?
The results from the olezarsen studies are significant as they offer a new treatment option for patients with sHTG, a condition characterized by high triglyceride levels and increased risk of acute pancreatitis. Current therapies and lifestyle changes often fail to adequately manage these risks. With approximately 3 million people affected by sHTG in the U.S., olezarsen could transform the treatment landscape, providing relief to those at high risk of life-threatening pancreatitis attacks. The success of olezarsen also marks a potential milestone for Ionis Pharmaceuticals, enhancing its portfolio of RNA-targeted medicines.
What's Next?
Ionis Pharmaceuticals plans to submit a supplemental new drug application to the FDA by the end of the year, aiming for approval to bring olezarsen to market. The company will present detailed data at an upcoming medical conference, which could further validate the drug's efficacy and safety. If approved, olezarsen would be Ionis's third independent launch in two years, marking a significant step in expanding its reach in the cardiometabolic disease sector.
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