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news.Arrowhead Pharmaceuticals has submitted a new drug application (NDA) for plozasiran to the US Food and Drug Administration (FDA) to treat familial chylomicronemia syndrome (FCS).
The application is supported by the SUMMIT programme's studies and the Phase III PALISADE trial’s positive outcomes. Credit: Tatiana from Pixabay.
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This rare genetic disease currently lacks FDA-approved treatments, making the submission a significant milestone.
Plozasiran, also known as ARO-APOC3, is an investigational RNA interference (RNAi) therapeutic. It aims to reduce the production of apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism.
The company plans to extend its regulatory submissions beyond the US, targeting additional approvals for plozasiran in 2025.
Arrowhead president and CEO Chris Anzalone said: “The NDA submission for investigational plozasiran represents an important milestone for Arrowhead as we advance multiple potential new medicines developed using our proprietary Targeted RNAi Molecule (TRiM) platform for patients in diverse therapeutic areas including cardiometabolic, pulmonary, neuromuscular, and liver diseases.
“We believe in the potential of RNAi to make a meaningful impact on patients and this first NDA submission is the culmination of over 15 years of innovation and commitment by so many talented Arrowhead employees and the investigators, patients, and caregivers that helped make our vision a reality.”
The application is supported by the SUMMIT programme’s studies and the Phase III PALISADE trial’s positive outcomes.
PALISADE met its primary and key secondary endpoints, demonstrating significant reductions in APOC3, triglycerides and acute pancreatitis incidence.
In this study, plozasiran showed a sustained decrease in triglycerides, with a median reduction of 80% in the 25mg group. Moreover, there was an 83% reduction in the risk of acute pancreatitis development in the pooled 25mg and 50mg plozasiran groups compared to the placebo.
Till date, plozasiran has been generally well-tolerated. The most commonly reported treatment-emergent adverse events in the PALISADE study for the 25mg dose, which is proposed for marketing approval, included Covid-19, abdominal pain, nasopharyngitis and nausea.
