FDA Grants Breakthrough Therapy Designation to Plozasiran for Familial Chylomicronemia Syndrome

Plozasiran, an investigational RNA interference (RNAi) therapeutic, has been granted breakthrough therapy designation by the FDA as an adjunct to diet for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS).

This rare genetic disease is characterized by extremely elevated triglyceride levels, which can lead to acute and potentially fatal pancreatitis. There are currently no FDA-approved therapies specifically targeting FCS, leaving a clinical need for patients with the disease.

The designation was awarded based on results from the PALISADE Phase 3 clinical trial. The trial demonstrated that plozasiran reduced triglycerides by 80% from baseline at month 10 and lowered the risk of developing acute pancreatitis by 83% in patients with genetically confirmed or clinically diagnosed FCS. The randomized, placebo-controlled study enrolled 75 participants across 18 countries, with patients receiving either 25 mg or 50 mg of plozasiran every 3 months.

Plozasiran is a first-in-class therapeutic designed to reduce production of apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism. By lowering APOC3 levels, the treatment aims to reduce triglycerides and mitigate the severe complications associated with FCS.

The developer of plozasiran plans to submit a New Drug Application to the FDA by the end of 2024 and seek regulatory approvals globally. To date, plozasiran has demonstrated a favorable safety profile, with adverse events consistent with the comorbidities and conditions of the study population.

Reference

Arrowhead Pharmaceuticals Receives FDA Breakthrough Therapy Designation for Plozasiran. Arrowhead Pharmaceuticals. September 10, 2024. Accessed December 24, 2024. https://arrowheadpharma.com/news-press/arrowhead-pharmaceuticals-receives-fda-breakthrough-therapy-designation-for-plozasiran