(Currently in development by Akcea)
Volanesorsen is an investigational drug in development for the treatment of FCS. It is designed to reduce the production of Apolipoprotein (ApoC-III), a protein produced in the liver that plays a central role in the regulation of plasma triglycerides. High levels of ApoC-III slow down the metabolism and/or removal of triglycerides from the bloodstream, which can lead to high levels of triglycerides in the blood.
Volanesorsen has been studied in Phase 3 clinical trials: the APPROACH Study in patients with FCS and the COMPASS Study in patients with severe hypertriglyceridemia. Both studies met their primary
endpoints and substantially reduced triglycerides in the respective patient populations, with a 77% reduction in triglycerides in the APPROACH study. Together, the combined study data from APPROACH and COMPASS showed patients had a significant reduction in new events of pancreatitis when treated with volanesorsen.
Akcea currently offers a volanesorsen Early Access Program for FCS. To learn more, email volanesorsenEAP@akceatx.com