Arrowhead’s plozasiran nears FDA approval for rare fat-processing disorder

Arrowhead Pharmaceuticals has taken a key step forward with its experimental drug plozasiran. The company recently submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for the treatment of Familial Chylomicronemia Syndrome (FCS), a rare genetic disorder that affects the body's ability to process certain fats. A decision on approval is now expected by November 18, 2025.

The biotech firm, known for its RNA interference (RNAi) technology, focuses on therapies for cardiometabolic diseases. Its lead candidate, plozasiran, aims to lower dangerously high triglyceride levels by targeting the ApoC3 gene.

Plozasiran is being developed for two conditions: FCS, and Severe Hypertriglyceridemia (sHTG), a broader metabolic issue that can lead to high levels of triglycerides in the blood. If approved, it would address a significant unmet need for patients with limited treatment options.

Arrowhead’s financial position remains strong, with around $900 million in cash and equivalents on hand. This provides a solid foundation for ongoing research and development. Beyond plozasiran, the company is advancing other programs, including fazirsiran for Alpha-1 antitrypsin deficiency and early-stage obesity treatments like ARO-INHBE and ARO-ALK7. Investors have shown interest in the company’s progress. As of January 15th, Arrowhead’s stock (ARWR) traded at $66.25, with a trailing price-to-earnings ratio of 478.56 and a forward P/E of 111.11. Analysts, including Baird, have highlighted the potential of plozasiran, with further FDA decisions anticipated in the second half of 2026. The next major milestone for Arrowhead will be the FDA’s review of plozasiran for FCS. Additional data from trials in sHTG are also expected soon, which could further shape the drug’s future.

The FDA’s decision on plozasiran will determine whether Arrowhead can bring its first RNAi therapy for FCS to market. A positive outcome would expand treatment options for patients with severe triglyceride disorders. The company’s pipeline and financial stability position it for continued growth in cardiometabolic and obesity-related therapies.