Regeneron Pharmaceuticals Inc said on Wednesday the U.S. Food and Drug Administration had approved the expanded use of its drug in children aged 5 to 11 years to treat an ultra-rare disease that causes high cholesterol levels.
The drug, Evkeeza, was approved in February 2021 as an additional treatment with other lipid-lowering therapies, for patients aged 12 years and above with homozygous familial hypercholesterolemia (HoFH).
HoFH is an inherited condition that affects about 1,300 people in the United States. It causes elevated levels of low-density lipoprotein (LDL) cholesterol and types of premature cardiovascular diseases.
Lipid-lowering therapies are the most commonly used drugs for high levels of LDL cholesterol, sometimes called the 'bad' cholesterol.
The expanded approval makes Regeneron's Evkeeza the first drug to treat kids under 10 with the condition.
The list price of the drug varies by patient weight, and is expected to be roughly $450,000 per year on average, Regeneron said in 2021.
The company recorded $48 million in sales of Evkeeza in the United States in 2022.
Regeneron's patient support program offers financial assistance to eligible patients who need help with out-of-pocket cost for Evkeeza.