Less than a year after selling off the rights to cholesterol drug Evkeeza outside of the United States, Regeneron has gained priority review status from the FDA for its use in children ages 5 to 11 with the ultra-rare genetic disorder homozygous familial hypercholesterolemia (HoFH).
The award comes with an action date of March 30, 2023. If approved, Evkeeza would become the first drug to treat kids with the condition who are younger than age 10.
HoFH, which affects about 1,300 people in the U.S., causes dangerously high levels of LDL (bad) cholesterol, which can trigger heart attacks as early as the teen years.
Evkeeza won its original FDA nod last February to treat patients 12 and older, becoming the first angiopoietin-like 3 (ANGPTL-3) treatment on the market. The once-a-month infused drug is used alongside other lipid-lowering therapies such as statins.
In January, Regeneron sold its ex-U.S. rights to the med to rare disease specialist Ultragenyx. The drug carries a hefty price tag at $450,000 per year and its sales have climbed slowly this year, from $8 million in the first quarter to $11 million in the second quarter and then to $14 million in the most recent three-month period.
The FDA granted the priority review based on a trial of 14 patients ages 5 to 11 with an average LDL-C level of 264, which is more than twice the desired 110 for children with HoFH. Over 24 weeks, patients on their normal regimen of lipid-lowering drugs plus Evkeeza saw an average reduction of 48% in their LDL-C levels. Most adverse events were mild, with no patients discontinuing their treatment.
Regeneron already has a cholesterol-lowering drug on the market, Sanofi-partnered Praluent, which was originally approved in 2015 and then gained its green light for HoFH in April of last year, though only for adults. Regeneron’s 2021 sales of Praluent in the U.S. reached $170 million last year.
Praluent is a PCSK9 drug, like Amgen’s Repatha, which was originally sanctioned by the FDA in 2015 and tacked on an HoFH green light in 2021 for patients age 10 and older. Amgen reported sales of Repatha at $1.2 billion last year, up 26% from 2020.
Regeneron’s hope with Evkeeza is that it can distinguish itself with its different mechanism of action as an ANGPTL-3.