FDA expands use of Regeneron's HoFH drug Evkeeza to kids 5 and up

At Regeneron, it’s all about the kids—at least this week.

One day after gaining a pediatric approval in Europe for Dupixent, Regeneron has earned an FDA nod for the use of Evkeeza in children ages 5 through 11 with homozygous familial hypercholesterolemia (HoFH).

The ultra-rare genetic condition is characterized by dangerously high levels of bad cholesterol, with treatment options limited for younger children.

Evkeeza was initially approved in February of 2021 as a first-in-class adjunctive treatment for HoFH patients 12 and older. Now it becomes the first angiopoietin-like 3 (ANGPTL3) inhibitor for kids as young as 5.  

The endorsement is based on a small single-arm trial showing the drug demonstrated a 48% reduction in low-density lipoprotein cholesterol (LDL-C) at week 24 when added to other lipid-lowering drugs. The trial enrolled just 20 kids ages 5 to 11.

Despite treatment with other lipid-lowering therapies, children entered the study with an average LDL-C level of 264 mg/dL, which is more than twice the <110 mg/dL target for pediatric patients with the disorder. The drug's safety profile was consistent with its profile in adults and other pediatric patients 12 and older in previous trials.

HoFH affects approximately 1,300 in the United States and is the most severe form of familial hypercholesterolemia (FH). The condition occurs when two copies of the FH-causing genes are inherited from each parent. Those with HoFH are at risk for cardiac events in their teen years. The condition often goes undiagnosed.

Upon its approval two years ago, Evkeeza was noted for its list price of $450,000 on average, with dosage determined by the weight of the patient. Regeneron’s sales of the drug reached just $48 million last year, climbing from $8 million in the first quarter to $15 million in the fourth.

In January of 2022, Regeneron sold rights to Evkeeza outside of the U.S. to Ultragenyx. Regeneron has another HoFH therapy, PCSK9 drug Praluent, which saw a sales drop from $170 million in 2021 to $130 million last year.

Amgen’s PCSK9 Repatha, which gained its indication for HoFH for those age 10 and up in April of 2021 and treats several other conditions, rang up sales of $1.3 billion last year, a 16% increase from 2021. 

“Evkeeza exemplifies the promise of genetics-based research to transform treatment paradigms,” George Yancopoulos, M.D., Ph.D., Regeneron’s chief scientific officer, said in the company's release.

On Tuesday, the European Commission signed off on Regeneron and Sanofi's Dupixent to treat kids age 6 months and up with atopic dermatitis (eczema). The FDA endorsed Dupixent for this age group in June of last year.