Research & Development
Ionis Pharmaceuticals' Olezarsen receives US FDA Orphan Drug designation for familial chylomicronemia syndrome
16 February 2024 -

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), a United States-based biotechnology company, announced on Thursday that it has received Orphan Drug designation from the US Food and Drug Administration (FDA) for the investigational medicine Olezarsen intended for the treatment of familial chylomicronemia syndrome (FCS), a rare, genetic disease characterized by extremely elevated triglyceride levels.

Olezarsen, an RNA-targeted investigational LIgand Conjugated Antisense (LICA) medicine, is being assessed for people at risk of disease because of elevated triglyceride levels, which includes those with FCS. It is aimed at inhibiting the body's production of apoC-III.

The US FDA has granted fast track designation to Olezarsen for the treatment of FCS in January 2023. The company is also assessing the product for the treatment of severe hypertriglyceridemia (sHTG) in Phase three clinical trials.

Sam Tsimikas, MD., Ionis senior vice president, global cardiovascular development, said, 'People living with FCS are in urgent need of a medicine that may help stabilise their triglyceride levels and reduce the risk of life-threatening AP attacks. If approved, olezarsen has the potential to be the standard of care in the US 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.'