Regeneron Pharmaceuticals (REGN) presented new data Wednesday showing a drug it developed can treat patients with a genetic disease that causes very high cholesterol levels.
In homozygous familial hypercholesterolemia, or HoFH, patients can have levels of low-density lipoprotein of 500 milligrams per deciliter or more, five times normal levels, and can have heart attacks or other cardiovascular problems in their 20s. The disorder, which results from having two non-functioning copies of the LDL receptor gene that is involved in removing cholesterol from the blood, afflicts about 1,300 patients in the United States.
