US FDA approves the use of Regeneron's cholesterol drug in young children

The FDA has approved the expanded use of Regeneron Pharmaceuticals Inc's drug in children aged 5 to 11 years to treat an ultra-rare disease that causes high cholesterol levels. Evkeeza was approved in February 2021 as a supplement to other lipid-lowering therapies for patients aged 12 and up with homozygous familial hypercholesterolemia (HoFH). HoFH is an inherited disorder that affects approximately 1,300 people in the United States.

It causes high levels of low-density lipoprotein (LDL) cholesterol as well as various types of premature cardiovascular disease. The most commonly used drugs for high levels of LDL cholesterol, also known as "bad" cholesterol, are lipid-lowering therapies. With the expanded approval, Regeneron's Evkeeza becomes the first drug to treat the condition in children under the age of ten.

The drug's list price varies by patient weight and is expected to be around $450,000 per year on average, according to Regeneron in 2021. In 2022, the company reported Evkeeza sales of $48 million in the United States. Regeneron's patient support programme provides financial assistance to eligible patients who require assistance with Evkeeza out-of-pocket costs.