The US Food and Drug Administration (FDA) has granted orphan drug designation to Ionis Pharmaceuticals’ olezarsen, an investigational ligand conjugated antisense medicine for familial chylomicronemia syndrome (FCS).

This rare genetic disease is characterised by extremely high triglyceride levels and can lead to recurrent acute pancreatitis (AP).

Olezarsen is an RNA-targeted therapy aimed at reducing the production of apolipoprotein C-III (ApoC-III), a protein that regulates triglyceride metabolism.

It is currently undergoing evaluation for individuals at risk of diseases caused by elevated triglyceride levels, including FCS.

In January 2023, the US regulator awarded fast track designation to olezarsen for FCS.  

Ionis is also investigating its potential to treat severe hypertriglyceridemia (sHTG) in Phase III clinical trials.

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FCS patients experience triglyceride levels that are significantly higher than normal, leading to life-threatening conditions such as AP and potential damage to vital organs.

No currently FDA-approved therapies exist for FCS, and standard treatments for lowering triglycerides are largely ineffective in FCS patients.

Sufferers have to adhere to strict diets to manage their condition.

In December 2023, Ionis signed a licensing agreement with Otsuka Pharmaceutical, granting the latter the rights to market donidalorsen, another ligand-conjugated antisense therapy for hereditary angioedema, in Europe.

Ionis Pharmaceuticals global cardiovascular development senior vice-president Sam Tsimikas stated: “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.”

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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