Arrowhead Pharmaceuticals Secures FDA Approval for Plozasiran to Treat Familial Chylomicronemia Syndrome
Saturday, January 18, 2025
Arrowhead Pharmaceuticals has announced the acceptance of its New Drug Application (NDA) by the U.S. Food and Drug Administration (FDA) for plozasiran, an investigational RNA interference therapeutic for familial chylomicronemia syndrome (FCS).
FCS is a rare genetic disorder often caused by mutations affecting triglyceride metabolism. It leads to extremely high triglyceride levels, exceeding 880 mg/dL, which may result in severe complications such as acute pancreatitis, diabetes, and chronic pain. Current treatment options are limited.
Plozasiran is a first-in-class investigational RNAi therapy targeting apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism. By reducing APOC3 levels, plozasiran aims to lower triglycerides and normalise lipid profiles.
It has demonstrated efficacy in reducing triglycerides across several clinical studies in patients with FCS, severe hypertriglyceridemia, and mixed hyperlipidaemia.
Plozasiran is currently under investigation and has not yet received approval for use in any condition.
This rare genetic condition leads to severe triglyceride elevations and associated complications. The FDA has set a Prescription Drug User Fee Act (PDUFA) target date of 18 November 2025, without plans for an advisory committee meeting at this stage.
Arrowhead also intends to submit approval applications for plozasiran to other regulatory authorities globally in 2025. The NDA is supported by results from the PALISADE Phase 3 clinical trial and earlier Phase 2 studies under the SUMMIT programme.
The PALISADE trial met its primary endpoint, demonstrating significant reductions in triglyceride levels and improvements in key secondary outcomes, including reductions in apolipoprotein C-III and the incidence of acute pancreatitis.
Patients in the plozasiran 25 mg group experienced a median 80% reduction in triglycerides, while the pooled plozasiran group showed an 83% reduction in pancreatitis risk compared to placebo.
The treatment was generally well tolerated, with commonly reported side effects including abdominal pain, nasopharyngitis, and nausea.
Results from PALISADE were presented at leading medical conferences, including the American Heart Association Scientific Sessions 2024 and the European Society of Cardiology Congress 2024, with publications in Circulation and The New England Journal of Medicine.
Source: arrowheadpharma.com
