Arrowhead Pharmaceuticals announced that the FDA accepted its New Drug Application (NDA) for plozasiran, a treatment for the rare genetic disorder familial chylomicronemia syndrome (FCS). The FDA set a target action date of November 18, 2025, and currently does not plan to convene an advisory committee meeting. Arrowhead plans to submit applications for plozasiran to other regulatory bodies in 2025.
This NDA acceptance is a crucial step towards potentially providing a much-needed treatment option for individuals with FCS. FCS patients experience extremely high triglyceride levels, putting them at significant risk for acute pancreatitis and other debilitating complications that severely impact their quality of life. Current treatment options are limited, making plozasiran’s potential approval a significant advancement in FCS management.
The NDA submission hinges on positive results from the Phase 3 PALISADE study, supported by data from Phase 2 trials. PALISADE demonstrated that plozasiran significantly reduced triglycerides, apolipoprotein C-III, and the incidence of acute pancreatitis. Specifically, the study showed a median triglyceride reduction of 80% and an 83% reduction in acute pancreatitis risk. The 25 mg dose, proposed for marketing approval, was generally well-tolerated, with common adverse events including abdominal pain, COVID-19, nasopharyngitis, and nausea.
The FDA’s acceptance of the NDA for plozasiran marks a significant milestone for Arrowhead. A potential approval would represent a major breakthrough for FCS patients, offering a new therapeutic option to manage this severe and rare disease. It also positions Arrowhead to potentially expand into the broader markets of severe hypertriglyceridemia and mixed hyperlipidemia, which the company is actively investigating in ongoing Phase 3 trials.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
