Ionis announces olezarsen FCS New Drug Application accepted for Priority Review and enrollment in Phase 3 sHTG program completed
25 Jun 2024
Phase 3Priority ReviewNDA
– Olezarsen PDUFA date set for December 19, 2024 for treatment of familial chylomicronemia syndrome –
– Phase 3 enrollment completed in CORE, CORE2 and ESSENCE evaluating olezarsen for the treatment of severe hypertriglyceridemia, with results expected in 2H 2025 –
CARLSBAD, Calif., June 25, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for olezarsen, an investigational RNA-targeted medicine for the treatment of adults with familial chylomicronemia syndrome (FCS). The FDA has set an action date of December 19, 2024 and indicated they are not currently planning an advisory committee meeting for olezarsen.
"FCS is a debilitating, rare, genetic disease that causes significant physical, emotional and financial burden with no approved treatment options in the U.S.," said Brett Monia, Ph.D., chief executive officer of Ionis. "The Priority Review underscores the urgent need people living with FCS have for a medicine that may help lower triglyceride levels and reduce incidence of life-threatening acute pancreatitis events. We look forward to partnering closely with the FDA during the review process as we work to bring this potentially breakthrough medicine to patients before the end of 2024. Separately, we are pleased with the progress of our now fully enrolled Phase 3 program investigating olezarsen in the much more common severe hypertriglyceridemia population."
Priority Review is a designation for medicines that have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease, with the expectation of the FDA taking action within six months, compared to 10 months under standard review. The 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 (ACC) Annual Meeting and published simultaneously in The New England Journal of Medicine (NEJM).
In addition to FCS, Ionis is evaluating olezarsen for the treatment of severe hypertriglyceridemia (sHTG) in three Phase 3 clinical trials – CORE, CORE2 and ESSENCE, all three of which completed enrollment in the first half of 2024. CORE and CORE2 enrolled more than 1,000 sHTG patients with fasting triglyceride levels ≥500 mg/dL; ESSENCE enrolled more than 1,400 high triglyceride patients with fasting triglyceride levels ≥150 mg/dL to Ionis.com and follow us on X (Twitter) and LinkedIn.
Ionis Forward-looking Statements
This press release includes forward-looking statements regarding olezarsen, Ionis' business, and the therapeutic and commercial potential of Ionis' commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions, or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended Dec. 31, 2023, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at www.Ionis.com.
Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc.
Ionis Investor Contact:
D. Wade Walke, Ph.D.
[email protected] 760-603-2331
Ionis Media Contact:
Hayley Soffer
[email protected] 760-603-4679
SOURCE Ionis Pharmaceuticals, Inc.
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