Regeneron Pharmaceuticals shared positive results from its Phase III trial of a candidate drug for children with homozygous familial hypercholesterolemia (HoFH), a rare, life-threatening condition characterized by elevated circulating levels of low-density lipoprotein cholesterol (LDL-C) and accelerated, premature atherosclerotic cardiovascular disease (ACVD.
The latest Phase III trial results evaluated Evkeeza to treat HoFH, an inherited disease that is also the most severe form of familial hypercholesterolemia. HoFH affects one in 160,000 to 300,000 people worldwide, and around 1,300 people in the United States. Those diagnosed with the disease, including patients as young as teenagers, are at high risk for premature atherosclerotic disease and life-threatening cardiac events.
Evekeeza (evinacumab) was developed using Regeneron’s VelocImmune technology, which uses a genetically-engineered mouse platform with a humanized immune system to create fully human antibodies. It’s a fully human monoclonal antibody that binds to and blocks ANGPTL3, inhibiting lipoprotein lipase and endothelial lipase and regulating LDL-C and other circulating lipids.
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