Arrowhead Pharma Gets FDA Approval for Plozasiran in Chylomicronemia Syndrome

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.

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!