New Therapeutic Approaches to Familial Chylomicronemia Syndrome (FCS)

Abstract

The understanding of FCS has advanced tremendously over the years, enabling us to find better alternatives in an otherwise undertreated population. The treatment revolves around an extremely low-fat diet, although patients find this difficult to maintain. There are many promising emerging approaches to FCS such as apoC3 inhibitors, ANGPTL3 inhibitors, LPL gene therapy, MTP inhibitors, DGAT inhibitors, apoC2 mimetics, and apoE mimetics. However, due to the rarity of FCS, this effort is slowed by the shortage of these rare study subjects and a regulatory process that at times discourages development in rare disease, which may easily derail such efforts. Several agents remain in development and may never prove useful for FCS, and despite several setbacks we remain hopeful that significant relief is on the way for our FCS patients.