Embracing Newly FDA Approved Therapies for FCS Management

The recent approval of multiple APOC3‑targeted agents has transformed the therapeutic landscape for familial chylomicronemia syndrome. Building on trial evidence, Nihar R. Desai, MD, explains that both olezarsen and plozasiran now have US Food and Drug Administration indications for FCS, offering clinicians unprecedented choice in targeted therapy. Olezarsen is administered monthly, whereas plozasiran is dosed quarterly, and this difference in dosing frequency may influence patient preference, adherence, and clinic workflow.

Desai reviews in more detail the simulation work based on the Balance and Palisade trials, emphasizing that, under multiple modeled scenarios and sensitivity analyses—including those restricted to genetically confirmed FCS—the probability of achieving triglyceride levels less than 500 mg/dL was consistently higher with plozasiran than with olezarsen. These modeled differences mirror the numerically greater triglyceride reductions observed in Palisade and are framed as clinically relevant, particularly given the association of this threshold with reduced pancreatitis risk.

Anthony Giamo, MD, underscores that real‑world treatment decisions should integrate trial data, modeled efficacy, dosing schedules, safety profiles, and individual patient characteristics and preferences. While acknowledging the limitations inherent to simulation studies and the lack of direct head‑to‑head comparisons, both experts view APOC3 inhibitors as transformative for FCS care. Clinicians are encouraged to use all available evidence to tailor therapy in a way that maximizes triglyceride lowering and minimizes pancreatitis risk, while remaining attentive to new data that may further refine agent selection.