RNAi Therapeutic Gets Orphan Drug Status for Familial Chylomicronemia Syndrome

The Food and Drug Administration (FDA) has granted Orphan Drug designation to ARO-APOC3 (Arrowhead Pharmaceuticals), an investigational treatment for familial chylomicronemia syndrome (FCS).

In FCS, mutations in lipoprotein lipase lead to an accumulation of chylomicrons in plasma resulting in severe hypertriglyceridemia. ARO-APOC3 is a subcutaneously administered RNAi therapeutic that targets apolipoprotein C-III (apoC-III), which according to the Company, is expected to reduce VLDL synthesis and assembly, enhance the breakdown of triglyceride rich lipoproteins, and improve clearance of VLDL and chylomicron remnants.

A phase 1, placebo controlled trial in 63 healthy volunteers with elevated triglycerides and patients with severe hypertriglyceridemia and FCS was initiated in March 2019. The trial is investigating the safety, tolerability, pharmacokinetics and pharmacodynamics of the treatment.

There is currently no therapy in the US approved to treat FCS. Last year the FDA issued a Complete Response Letter to Akcea and Ionis Pharmaceuticals rejecting their New Drug Application for a possible treatment.

For more information visit arrowheadpharma.com.