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Stony Brook Heart Institute Administers the World’s First Commercial Plozasiran Therapy for Patients with a Rare Lipid Disorder

Published on: Dec 30, 2025

The Center for Advanced Lipid Management at the Stony Brook Heart Institute has reached a historic global milestone by becoming the first center worldwide to administer a commercial dose of plozasiran, a newly FDA-approved RNA-interference therapy for Familial Chylomicronemia Syndrome (FCS).

FCS is an ultra-rare genetic disorder characterized by extremely elevated triglyceride levels often 10 to 100 times above normal placing patients at high risk for recurrent, severe pancreatitis. Approximately 6,500 individuals in the United States are affected by genetic or clinical FCS. Plozasiran, an RNAi therapy targeting apolipoprotein C-III, demonstrated substantial and sustained reductions in triglyceride levels along with fewer pancreatitis episodes in the PALISADE Phase 3 trial, marking a transformative breakthrough for patients who previously had limited or no effective treatment options.

“This represents a landmark moment for patients living with FCS around the world,” said Dr. Tahmid Rahman, Co-Director of the Center for Advanced Lipid Management at the Stony Brook Heart Institute. “Our early involvement in clinical research and our specialized, referral-level infrastructure enabled us to deliver this therapy within days of FDA approval.”

The Center for Advanced Lipid Management integrates advanced diagnostic expertise with coordinated, precision-based care to rapidly identify rare and complex lipid disorders and deliver cutting-edge, disease-modifying treatments such as plozasiran. Dr. On Chen, Co-Director of the Center, emphasized that the program serves as a Center of Excellence for genetic and rare lipid disorders, including familial hypercholesterolemia, elevated lipoprotein(a), Familial Chylomicronemia Syndrome, and severe inherited hypertriglyceridemia. The program also provides comprehensive cardiovascular care for patients with coronary artery disease, prior heart attack or stroke, and peripheral arterial disease, while offering advanced cardiovascular risk assessment and early intervention strategies for preventive care.

Patients benefit from access to emerging and transformative therapies, including novel biologics, RNA-based treatments, and clinical trials for individuals whose cholesterol or triglyceride levels remain uncontrolled with standard therapies positioning Stony Brook Medicine as a regional and national leader in advanced lipid care.

Source: https://news.stonybrook.edu/newsroom/press-release/medical/stony-brook-heart-institute-delivers-worlds-first-commercial-plozasiran-treatment-for-patients-with-rare-lipid-disease/

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