With a cleaner safety profile and better efficacy than marketed agents, Regeneron’s first-in-class evinacumab is poised to become the standard of care for homozygous familial hypercholesterolemia and other rare lipid disorders, including for the hardest to treat patients with no LDL receptor function.
The candidate is unlikely to challenge other drug classes, like PCSK9 inhibitors, in larger hypercholesterolemia populations, however, given the availability of effective and cheap treatment options for those patients.
Homozygous familial hypercholesterolemia (HoFH) is an ultra-rare genetic condition that affects roughly 1,300 patients in the U.S. Genetic defects result in dysfunctional LDL receptors in the liver, rendering them unable to clear LDL-C from the body.
Until 2012, the most common treatment was maximally tolerated lipid-lowering therapies like statins -- which have little effect given the lack of LDL receptor function -- and apheresis, a three- to four-hour procedure done every 10-14 days to filter LDL from the blood.
In December 2012 and January 2013, two HoFH drugs came to market: Juxtapid lomitapide from Aegerion Pharmaceuticals Inc., followed by