Plozasiran FDA Approval Status
Last updated by Judith Stewart, BPharm on Jan 20, 2025.
FDA Approved: No
Generic name: plozasiran
Company: Arrowhead Pharmaceuticals, Inc.
Treatment for: Familial Chylomicronemia Syndrome
Plozasiran is a first-in-class investigational RNA interference (RNAi) therapeutic in development for the treatment of familial chylomicronemia syndrome.
- Familial chylomicronemia syndrome (FCS) is a rare, genetic hyperlipidemia characterized by an excessive increase in plasma triglyceride levels due to the accumulation of chylomicrons, which leads to extremely high triglyceride levels. Severe elevations in triglycerides can lead to serious symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues.
- Plozasiran works in the treatment of FCS by reducing the production of apolipoprotein C-III (APOC3). APOC3 increases triglyceride levels in the blood by inhibiting breakdown of triglyceride rich lipoproteins (TRLs) by lipoprotein lipase and uptake of TRL remnants by hepatic receptors in the liver. Reducing APOC3 reduces triglycerides and restores lipids to more normal levels.
- A New Drug Application (NDA) has been accepted by the U.S. Food and Drug Administration (FDA) for plozasiran in the treatment of FCS based on positive results from the Phase 3 PALISADE study with supportive confirmatory evidence from the Phase 2 clinical studies of the SUMMIT Program. The FDA has assigned November 18, 2025 as the Prescription Drug User Fee Act (PDUFA) action date.
- Plozasiran in the treatment of FCS has also been granted Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation by the FDA.
Development timeline for plozasiran
Further information
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