FDA grants orphan drug designation to olezarsen for familial chylomicronemia syndrome
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The FDA has granted orphan drug designation to Ionis Pharmaceuticals’ olezarsen, an investigational ligand-conjugated antisense drug for the treatment of familial chylomicronemia syndrome, according to a company release.
A rare genetic hyperlipidemia, familial chylomicronemia syndrome (FCS) is characterized by extremely elevated triglyceride levels and increased risk for recurrent acute pancreatitis, abdominal pain, hepatosplenomegaly, eruptive xanthomas, lipemia retinalis and type 3c diabetes. Olezarsen, previously granted FDA fast track status in January 2023, was developed to inhibit the production of apolipoprotein C-III in patients with elevated triglyceride levels.
“People living with FCS are in urgent need of a medicine that may help stabilize their triglyceride levels and reduce the risk of life-threatening [acute pancreatitis] attacks,” Sam Tsimikas, MD, senior vice president of global cardiovascular development at Ionis, said in the release. “If approved, olezarsen has the potential to be the standard of care in the U.S. for the treatment of FCS. We are deeply committed to advancing this important medicine that has the potential to be transformative for people and families living with FCS.”
The company noted that olezarsen is also being investigated for the treatment of severe hypertriglyceridemia in phase 3 clinical trials.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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