18 January 2025 – Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for plozasiran, an investigational therapy designed to treat familial chylomicronemia syndrome (FCS)—a rare, genetic disorder marked by extremely high triglyceride levels and elevated acute pancreatitis risk.

The FDA also assigned a Prescription Drug User Fee Act (PDUFA) action date of 18 November 2025 for evaluation, and indicated that no advisory committee meeting is planned, suggesting confidence in the submission’s clarity and quality.

Arrowhead highlighted that the NDA filing was supported by positive outcomes from its Phase 3 PALISADE study, demonstrating consistent trendlowering triglycerides and clinical benefit in individuals genetically confirmed—or clinically diagnosed—with FCS. The acceptance marks a significant inflection point for both Arrowhead and the rare disease community it aims to serve.

Integrated Insight

The FDA’s acceptance of plozasiran’s NDA is more than procedural—it reflects robust clinical performance and strong regulatory readiness. A clear PDUFA date in November 2025 gives stakeholders a solid timeline for decision-making and strategic preparation for potential approval.

This development is especially meaningful in the area of rare metabolic disorders, where treatment options are scarce and the burden on patients is high. FCS, characterized by catastrophic pancreatitis and chronic complications, has seen no approved targeted therapies—making plozasiran’s potential approval a landmark event.

Arrowhead’s NDA acceptance may also enhance investor and partner confidence, possibly catalysing commercialization deals and international regulatory filings in the months ahead.