Perspectives on Ongoing Unmet Need and the Future of FCS Care

Despite major therapeutic advances, substantial unmet need remains in the diagnosis and management of familial chylomicronemia syndrome (FCS).

In their closing reflections, Nihar R. Desai, MD, and Anthony Giamo, MD, note that many patients first present to hospital-based internists with acute pancreatitis and markedly elevated triglycerides, where distinguishing FCS from multifactorial chylomicronemia can be challenging. They emphasize the importance of early referral to lipid specialists and point to resources such as the National Lipid Association’s directory of lipidologists to facilitate expert evaluation and confirmatory testing.

The experts also highlight real‑world obstacles related to coverage, access, and cost of newly approved APOC3‑targeted therapies. Transitioning from controlled trial settings and modeling exercises to broad clinical impact requires that patients can reliably obtain and continue treatment. In addition, long‑term data on pancreatitis reduction, quality of life, and potential cardiovascular outcomes are still needed, and there is strong interest in head‑to‑head comparisons and expanded indications.

Looking forward, advances in FCS are expected to inform the management of broader populations with severe or mixed hypertriglyceridemia. The mechanistic insights gained from APOC3 inhibition may spur development of additional agents and rational combination strategies targeting triglyceride‑driven disease. The faculty characterize this moment as an inflection point in preventive cardiology and lipidology, in which a historically refractory condition now has effective targeted therapies, and ongoing education, research, and systems‑level efforts will be critical to fully realizing their potential for patients.