Stony Brook Heart Institute’s Center for Advanced Lipid Management has become the first medical center worldwide to deliver a commercial dose of plozasiran, an RNA-interference therapy recently approved by the U.S. Food and Drug Administration (FDA) for Familial Chylomicronemia Syndrome (FCS).
FCS is a rare genetic disorder that causes extremely high triglyceride levels, significantly increasing the risk of recurrent and severe pancreatitis. According to estimates, about 6,500 people in the United States are affected by either genetic or clinical forms of FCS. Until now, patients with this condition had limited effective treatment options.
Plozasiran targets apolipoprotein C-III and was shown in the PALISADE Phase 3 trial to provide significant and sustained reductions in triglyceride levels as well as fewer episodes of pancreatitis.
“This is a historic moment for patients with FCS everywhere,” said Dr. Tahmid Rahman, Co-Director of the Center for Advanced Lipid Management at the Stony Brook Heart Institute. “Our early research involvement and specialized, referral-level clinical infrastructure allowed us to deliver this therapy within days of FDA approval.”
Dr. On Chen, also Co-Director of the Center for Advanced Lipid Management at Stony Brook Heart Institute, stated: “Stony Brook’s Advanced Lipid Management Center combines specialized diagnostic expertise with coordinated, precision care to rapidly identify rare and complex lipid disorders and deliver state-of-the-art, disease-modifying therapies such as plozasiran. The Center of Excellence program specializes in genetic and rare lipid disorders, including familial hypercholesterolemia, elevated lipoprotein(a), Familial Chylomicronemia Syndrome, and severe inherited hypertriglyceridemia. The program also provides comprehensive care for patients with coronary artery disease, prior heart attack or stroke and peripheral arterial disease. For patients focused on prevention, the program offers advanced cardiovascular risk assessment using state-of-the-art imaging and testing to deliver tailored, early intervention strategies.”
The center provides access to emerging therapies—including new biologics and RNA-based treatments like plozasiran—as well as clinical trials for individuals whose cholesterol or triglycerides are not controlled by standard treatments.
Hal Skopicki, MD, PhD, Co-Director of the Stony Brook Heart Institute and Ambassador Charles A. Gargano Chair of Cardiology added: “This milestone further reinforces Stony Brook Medicine’s role as a destination center for patients and referring physicians seeking comprehensive evaluation and access to the most advanced therapies through our Heart Institute’s Center of Excellence programs.”
The Stony Brook Heart Institute operates within Stony Brook University Hospital on Long Island. It offers multidisciplinary programs focused on prevention, diagnosis, and treatment of cardiovascular diseases through collaboration among cardiologists, surgeons, clinicians, nurses, therapists, technologists and scientists. The institute features 24-hour cardiac catheterization laboratories and advanced surgical suites while conducting translational and clinical research.
For more information about services or appointments at the Center for Advanced Lipid Management visit https://heart.stonybrookmedicine.edu/LipidProgram.