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

Ionis Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for olezarsen, a new RNA-targeted medicine intended for treating familial chylomicronemia syndrome (FCS) in adults. The FDA has granted a Priority Review for olezarsen, setting an action date for December 19, 2024, without planning an advisory committee meeting.

FCS is a rare genetic disorder that causes significant physical and emotional distress. It is characterized by extremely high triglyceride levels due to a deficiency in the enzyme lipoprotein lipase (LPL), which impairs the breakdown of chylomicrons, the particles primarily composed of triglycerides. This condition leads to severe health issues, including a high risk of acute pancreatitis (AP). Managing FCS is challenging, as affected individuals often rely on highly restrictive diets due to the lack of effective FDA-approved treatments in the U.S.

Brett Monia, CEO of Ionis, highlighted the urgent need for a treatment for FCS. He emphasized the potential of olezarsen to lower triglyceride levels and reduce the frequency of life-threatening acute pancreatitis events. Monia expressed the company's commitment to working closely with the FDA to expedite the review process and deliver this breakthrough medicine to patients by the end of 2024. Additionally, Ionis is progressing with its Phase 3 trials evaluating olezarsen for severe hypertriglyceridemia (sHTG).

The NDA submission for olezarsen was supported by positive outcomes from the Balance study, a global Phase 3 trial that was randomized, double-blind, and placebo-controlled. The results were presented at the 2024 American College of Cardiology Annual Meeting and published in The New England Journal of Medicine. The FDA had previously granted olezarsen Fast Track, Orphan Drug, and Breakthrough Therapy designations for FCS, underscoring its potential significance. Ionis also plans to file for regulatory approval in the European Union within the year.

Beyond FCS, olezarsen is under investigation for treating severe hypertriglyceridemia (sHTG) in three Phase 3 trials—CORE, CORE2, and ESSENCE. These trials completed enrollment in early 2024. CORE and CORE2 involved over 1,000 patients with fasting triglyceride levels of at least 500 mg/dL, while ESSENCE enrolled more than 1,400 patients with varying triglyceride levels who were either diagnosed with or at risk for atherosclerotic cardiovascular disease. The data from these trials are expected in the second half of 2025.

sHTG is a prevalent condition characterized by triglyceride levels of 500 mg/dL and above, caused by genetic factors and secondary conditions like diet, lifestyle, and certain medications. In the U.S., over three million individuals are estimated to live with sHTG, which places them at a high risk for pancreatitis and other severe health complications. Current treatment guidelines recommend lifestyle modifications and triglyceride-lowering medications, but these are often insufficient for many patients, indicating a significant need for new therapeutic options like olezarsen.

Ionis Pharmaceuticals has a three-decade history of developing innovative medicines. The company has five marketed products and a robust pipeline focused on neurology, cardiology, and other areas of high unmet medical need. As a pioneer in RNA-targeted therapies, Ionis continues to advance in the field of RNA medicines and gene editing, driven by a commitment to improving patient outcomes.

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