Volanesorsen May Reduce Disease Burden in Familial Chylomicronemia Syndrome
Volanesorsen (Waylivra) is being investigated for familial chylomicronemia syndrome (FCS), a genetic disease in which a deficiency in lipoprotein lipase prevents breakdown of triglycerides. Volanesorsen targets apoC-III synthesis to reduce the extremely high triglyceride levels observed in patients with Fcs. In the phase 3 APPROACH trial, the volanesorsen arm had a 77% reduction in triglyceride levels compared with an 18% increase in the placebo arm (P <.001). The ReFOCUS study surveyed patients who used volanesorsen for at least 3 months in the APPROACH extension study. Patients reported significant reductions in steatorrhea, pancreatic pain, and worry about pancreatic attacks. They also reported marked improvement in quality of life and function. Although the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee recommend approval of volanesorsen by a vote of 12 to 8, the FDA recently rejected the manufacturer’s application.
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Arca M, Hsieh A, Soran H, Rosenblit P, O’Dea L, Stevenson M. The effect of volanesorsen treatment on the burden associated with familial chylomicronemia syndrome: the results of the ReFOCUS study. Expert Rev Cardiovasc Ther. 2018;16(7):537-546. doi: 10.1080/14779072.2018.1487290.