The results of the Phase three study of WAYLIVRA (volanesorsen) in patients with familial chylomicronemia syndrome (FCS) were recently published in the New England Journal of Medicine (NEJM) (Witzum, et al., 2019). FCS patients have severely elevated triglycerides (often >2000 mg/dl). This drug is now approved in the EU and being considered for approval by the U.S. Food and Drug Administration (FDA). To date, it is the only medicine shown to be able to provide the level of triglyceride reduction these patients need.
As the results of this and other studies have already been published, I will not discuss them. Rather, I will briefly comment on one facet of this medicine that I find interesting. FCS is caused by homozygous loss of function of plasma lipoprotein lipase that degrades triglycerides. Prior to our work, it was believed that the sole activity of the target, ApoC3, was to inhibit this lipoprotein lipase. Since the lipoprotein lipase is functionally absent in FCS patients, it was assumed that volanesorsen would be ineffective. On the other hand, there is evidence that suggested Apoc3 has additional roles in the clearance of triglycerides. So, by pursuing an appropriate clinical trial, we uncovered new roles for this interesting molecular target.
For more information on WAYLIVRA, please visit www.WAYLIVRA.eu.
To read the full release highlighting WAYLIVRA’s approval in the EU, click here.