FDA grants fast track designation for olezarsen in familial chylomicronemia syndrome
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The FDA has granted fast track designation for olezarsen in the treatment of familial chylomicronemia syndrome, according to a press release from Ionis Pharmaceuticals.
Olezarsen is an investigational ligand-conjugated antisense drug developed to inhibit the production of apolipoprotein C-III in patients with elevated triglyceride levels, including those with familial chylomicronemia syndrome (FCS).
The FDA’s decision comes after Ionis completed enrollment in 2022 for its global phase 3 BALANCE study of olezarsen in adult patients with FCS.
“The FDA fast track designation for olezarsen recognizes the urgent need for an effective treatment for FCS, a debilitating rare disease affecting people with very limited treatment options and an elevated risk of painful and potentially fatal bouts of pancreatitis,” Richard S. Geary, PhD, executive vice president and chief development officer at Ionis, said in the release. “We look forward to working collaboratively with the FDA to bring forward a safe and effective treatment for FCS patients as quickly as possible.”
According to the release, the designation is designed to facilitate development and expedite the review of therapies for serious or life-threatening diseases in which there is an unmet need. Benefits to the fast-track status include more frequent interactions with the FDA throughout the development program and eligibility for accelerated approval, priority review and rolling review.
Ionis plans to share data from the BALANCE study in the second half of 2023, the release stated.
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