The Food and Drug Administration (FDA) has granted Fast Track designation to olezarsen for the treatment of familial chylomicronemia syndrome (FCS).

In FCS, mutations in the lipoprotein lipase gene lead to an accumulation of chylomicrons in plasma resulting in severe hypertriglyceridemia. Olezarsen is an investigational ligand-conjugated antisense medication designed to inhibit the production of apolipoprotein C-III, a protein produced in the liver that regulates triglyceride metabolism in the blood.

The Company is currently evaluating the efficacy and safety of olezarsen in patients with FCS in the phase 3 BALANCE study ( Identifier: NCT04568434). Data are expected to be available in the second half of 2023.

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“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,” said Richard S. Geary, PhD, executive vice president and chief development officer at Ionis. “We look forward to working collaboratively with the FDA to bring forward a safe and effective treatment for FCS patients as quickly as possible.”

Olezarsen is also being investigated in patients with hypertriglyceridemia and atherosclerotic cardiovascular disease (established or at increased risk for), and/or with severe hypertriglyceridemia ( Identifier: NCT05355402).


Ionis receives FDA Fast Track designation for olezarsen in patients with familial chylomicronemia syndrome. News release. Ionis Pharmaceuticals, Inc. Accessed January 31, 2023.

This article originally appeared on MPR