Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) recently made significant strides in the development of its investigational drug, plozasiran, for the treatment of familial chylomicronemia syndrome (FCS), a severe and rare genetic disorder characterized by persistently high triglyceride levels. The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for plozasiran, marking a critical step forward in its potential approval process. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of November 18, 2025, and has indicated that it is not currently planning to convene an advisory committee meeting regarding the application.
Recent data from clinical studies were also highlighted at the American Heart Association Scientific Sessions 2024 (AHA24). In presentations, Arrowhead Pharmaceuticals shared findings from the Phase 3 PALISADE study, as well as from open-label extensions of the Phase 2 MUIR and SHASTA-2 studies. The PALISADE study aimed to assess the efficacy and safety of plozasiran in patients diagnosed with FCS and demonstrated promising results. Concurrently, data from these studies were published in the AHA journal, Circulation, further establishing the credibility of the findings.
In addition to its regulatory and scientific advancements, Arrowhead Pharmaceuticals launched a new disease awareness campaign
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