Regeneron's Evkeeza, carrying big price tag, wins FDA approval in ultra-rare cholesterol disease

Regeneron has been battling against rival Amgen with their mass-market PCSK9 cholesterol drugs, and now the company is underway with a new launch to match its rival in the ultra-rare disease homozygous familial hypercholesterolemia (HoFH).

Regeneron’s Evkeeza scored approval on Thursday as an add-on to other lipid-lowering therapies in patients 12 and older with HoFH, which affects approximately 1,300 patients in the U.S. The company has a "dedicated and experienced team in place" to support the rollout, a spokesman said. The drug is given once per month through an intravenous infusion.