On Chen and Tahmid Rahman explain the real-world safety, dosing advantages, and pharmacist-driven identification strategies for patients who may benefit from plozasiran therapy.
In an interview with Pharmacy Times, On Chen, MD, clinical associate professor in medicine at Stony Brook Medicine, and Tahmid Rahman, MD, associate director of the Center for Advanced Lipid Management at Stony Brook Medicine, discuss real-world considerations as plozasiran (Redemplo; Arrowhead Pharmaceuticals) and related therapies enter clinical practice for familial chylomicronemia syndrome (FCS).
Chen explains that earlier therapies, such as volanesorsen (Waylivra; Ionis Pharmaceuticals), were limited by safety concerns and lack of FDA approval in the United States. In contrast, plozasiran features a GalNAc moiety, a liver-targeting sugar modification that improves drug delivery and significantly reduces adverse effects, resulting in a safer and more practical treatment option for clinicians and pharmacists.
Chen also notes the availability of other agents in the same therapeutic class, such as olezarsen (Tryngolza; Ionis Pharmaceuticals), which similarly interferes with RNA pathways to reduce triglyceride production. Across clinical trials and real-world experience, these agents have demonstrated favorable safety profiles, with minimal intolerance reported. Aside from occasional injection site reactions or rare hypersensitivity events, pharmacists should expect few significant adverse effects. Rahman highlights an additional advantage of plozasiran—its quarterly dosing schedule—which improves convenience compared with monthly injectable alternatives. He references PALISADE trial data demonstrating strong safety outcomes, reinforcing confidence in long-term use.
The discussion also emphasizes the critical role pharmacists play in identifying patients who may have undiagnosed or misdiagnosed FCS. Rahman explains that pharmacists can flag patients who remain uncontrolled despite therapy with fibrates, omega-3 fatty acids, or niacin, prompting conversations about newer treatment options. In inpatient settings, pharmacists can identify patterns such as recurrent pancreatitis admissions and persistently elevated triglyceride levels, facilitating timely referrals to specialized lipid clinics.
Chen concludes by outlining hallmark signs of FCS that pharmacists should watch for, including triglyceride levels exceeding 1000 mg/dL, recurrent abdominal pain, pancreatitis episodes, and characteristic skin findings. Early recognition and referral can expedite access to advanced therapies and reduce the risk of life-threatening complications.
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