RX Review: Delays, Clinical Features, and Raising Awareness for FCS

As the cardiology community welcomes a second US Food and Drug Administration-approved therapy for familial chylomicronemia syndrome (FCS) in plozasiran (Redemplo), it has reignited conversations surrounding diagnosis and awareness for the rare form of dyslipidemia.

In part 2 of our 7-part video series on updates in FCS, host Viet Le, DMSc, PA-C, a preventive cardiology PA at Intermountain Health and the former president of the Academy of Physician Associates in Cardiology, welcomes Christie Ballantyne, MD, chief of cardiovascular research at Baylor College of Medicine and principal investigator of the PALISADE trial, begin segment by discussing why individuals with FCS often face long delays before receiving an accurate diagnosis.

Ballantyne explains many patients first present with symptoms common to a wide range of metabolic conditions, including abdominal discomfort, fatigue, and lab findings consistent with general hypertriglyceridemia. Because these symptoms are nonspecific, clinicians frequently default to conventional therapies such as fibrates or high-dose omega-3 fatty acids. When these interventions fail to improve triglyceride levels, the lack of response may be attributed to nonadherence rather than an underlying genetic disorder. As a result, patients can spend years cycling through ineffective treatments while episodes of pancreatitis accumulate.

The discussion shifts to the clinical signs that should raise suspicion for FCS. Ballantyne points out persistent triglyceride levels above 1000 mg/dL, particularly in the absence of secondary causes, should prompt renewed evaluation. Additional red flags include early onset, recurrent pancreatitis, eruptive xanthomas, lipemia retinalis, and poor response to standard therapies. Le and Ballantyne reiterate a comprehensive lipid profile should be performed in any patient with unexplained pancreatitis. Early recognition is especially important in 2025, given the emergence of effective therapies. Ballantyne emphasizes patients who historically endured years of uncontrolled triglycerides can now benefit from agents designed to correct disease mechanisms more directly.

The segment concludes by reinforcing the need for clinician education and systematic clinical assessment. Ballantyne stresses timely diagnosis not only prevents morbidity but also allows patients to access new treatments such as olezarsen (Tryngolza) and plozasiran. These therapies expand the possibilities for meaningful triglyceride reduction, making awareness more important than ever. Le and Ballantyne agree increased recognition will likely lead to earlier referrals, clearer treatment pathways, and a measurable improvement in patient outcomes.

Relevant disclosures for Ballantyne include Arrowhead Pharmaceuticals, Ionis, Merck, Novartis, Novo Nordisk, New Amsterdam, Esperion, AstraZeneca, Eli Lilly, and others. Relevant disclosures for Le include Amarin, Bayer, Esperion, Idorsia, Janssen, Novo Nordisk, Novartis, Lexicon Pharmaceuticals, and Pfizer.

References:

Arrowhead Pharmaceuticals. Arrowhead Pharmaceuticals Announces FDA Approval of REDEMPLO® (plozasiran) to Reduce Triglycerides in Adults with Familial Chylomicronemia Syndrome (FCS) - Arrowhead Pharmaceuticals, Inc. Arrowhead Pharmaceuticals, Inc. Published November 19, 2025. Accessed November 25, 2025. https://arrowheadpharma.com/news-press/arrowhead-pharmaceuticals-announces-fda-approval-of-redemplo-plozasiran-to-reduce-triglycerides-in-adults-with-familial-chylomicronemia-syndrome-fcs/.

Virani SS, Morris PB, Agarwala A, et al. 2021 ACC Expert Consensus Decision Pathway on the Management of ASCVD Risk Reduction in Patients With Persistent Hypertriglyceridemia: A Report of the American College of Cardiology Solution Set Oversight Committee. J Am Coll Cardiol. 2021;78(9):960-993. doi:10.1016/j.jacc.2021.06.011

US Food and Drug Administration. FDA approves Tryngolza (olezarsen). U.S. Food and Drug Administration. Published December 19, 2024. Accessed November 25, 2025. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-reduce-triglycerides-adult-patients-familial-chylomicronemia-syndrome.