Plozasiran led to an 80% median reduction in baseline of fasting triglyceride level in a Phase III trial. Image credit: Shutterstock/ peterschreiber.media.

The US Food and Drug Administration (FDA) has accepted a new drug application (NDA) for Arrowhead Pharmaceutical’s plozasiran in the same indication, a month after the first treatment was approved by the regulator for the treatment of familial chylomicronemia syndrome (FCS).

The Prescription Drug User Fee Act (PDUFA) action date for Arrowhead’s candidate is 18 November 2025, with the FDA adding that it currently does not intend to hold an advisory committee meeting.

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FCS is a rare metabolic disease that prevents the body from breaking down fats due to a mutation in lipoprotein lipase. The resulting elevated levels of triglycerides can lead to chronic abdominal pain, diabetes, hepatic steatosis, and acute and potentially fatal pancreatitis.

Arrowhead’s NDA is supported by data from the Phase III PALISADE (NCT04998201) trial, along with supportive confirmatory evidence from Phase II studies in the SUMMIT programme. Plozasiran lowered triglyceride levels and reduced acute pancreatitis risk, leading the trial to meet primary and all multiplicity-controlled key secondary endpoints.

Plozasiran is an RNA interference (RNAi) therapeutic that cuts the production of apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism.

Last month, Ionis Pharmaceuticals’ Tryngolza (olezarsen) became the first treatment approved for FCS by the FDA. Similar to plozasiran, Tryngolza is also an RNA-based drug designed to reduce the body’s production of APOC3. Direct comparisons between the two drugs have not been made in clinical trials. High-dose Tryngolza led to a 44% placebo-adjusted drop in triglyceride levels from baseline at six months, with this rising to 59% in months six to 12. For 25mg plozasiran, the median reduction in baseline fasting triglyceride level was 80%.

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Tryngolza is forecast to generate $303m in annual revenue by 2030, according to GlobalData’s Pharma Intelligence Center.

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Arrowhead’s CEO Chris Anzalone said: “We are laser-focused on our ongoing work to ensure an efficient and successful commercial launch this year, pending FDA review and approval.”

In addition to targeting FDA approval, Arrowhead stated that it intends to submit applications for plozasiran in FCS treatment to additional regulatory authorities this year.

The pharma company also has further lipid-based plans for plozasiran and is investigating the drug in severe hypertriglyceridemia and mixed hyperlipidemia. Moreover, Arrowhead placed a target on the obesity treatment space by advancing two RNAi-based candidates to clinic in August 2024.