Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

52 week RCT (n=66) found antisense-mediated inhibition of hepatic APOC3 mRNA with volanesorsen resulted in 77% decrease (p<0.001) in mean triglyceride levels (-19.3 mmol/L) vs. 18% increase with placebo (+1.0 mmol/L). Thrombocytopenia and injection-site reactions were common ADRs

Familial chylomicronemia syndrome (FCS) is a rare genetic disorder that is caused by loss of lipoprotein lipase activity and characterised by chylomicronemia and recurrent episodes of pancreatitis. There are no effective therapies.

 

Volanesorsen (Waylivra) was granted conditional marketing authorisation in EU in May 2019 as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. There are plans to launch in Germany this year followed by additional European countries in 2020.