Key Takeaways
- Familial chylomicronemia syndrome (FCS) is a rare, often underdiagnosed genetic disorder causing extremely high triglycerides and recurrent pancreatitis, with traditional therapies frequently insufficient.
- Plozasiran, a small interfering RNA targeting APOC3, offers substantial and safe triglyceride reduction with convenient quarterly dosing, improving the management of FCS.
- Successful use of plozasiran depends on multidisciplinary systems, with pharmacists playing a central role in identifying high-risk patients, navigating access (PAs/benefits), and supporting education and monitoring.
On Chen, MD, and Tahmid Rahman, MD, faculty at Stony Brook Medicine’s Center for Advanced Lipid Management, join Pharmacy Focus to discuss the world’s first commercial administration of plozasiran (Redemplo; Arrowhead Pharmaceuticals), a small interfering RNA therapy targeting APOC3 mRNA, which restores lipoprotein lipase activity and produces dramatic triglyceride reductions in patients with familial chylomicronemia syndrome (FCS).
Chen and Rahman highlight the critical role of coordinated multidisciplinary systems—particularly pharmacists, nurses, and administrative staff—in pre-identifying high-risk patients, navigating prior authorization and benefit verification, and ensuring proper administration and monitoring. Pharmacists are positioned as key players in spotting undiagnosed FCS through medication patterns, very high triglyceride levels, and recurrent pancreatitis admissions, and in facilitating referrals to specialized lipid centers. Looking ahead, Chen and Rahman anticipate rapid expansion of RNA-based therapies across broader lipid disorders, including low-density lipoprotein cholesterol and lipoprotein(a), with pharmacists central to integrating these treatments into routine cardiovascular care.
