Regeneron, Ultragenyx's Evkeeza helps reduce bad cholesterol in kids 5 to 11 with rare disorder in study

Hailshadow/iStock via Getty Images

Regeneron Pharmaceuticals (NASDAQ:REGN) said a phase 3 trial of Evkeeza (evinacumab) met its main goal in children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH).

HoFH is a rare inherited condition and is a type of of familial hypercholesterolemia — a disorder in which it is difficult for the body to remove low-density lipoprotein-cholesterol (LDL-C), also called ‘bad’ cholesterol, from the blood.

The company said the trial met its main goal showing children who received Evkeeza to other lipid-lowering therapies reduced their LDL-C by 48% at week 24 on average.

Regeneron added that despite treatment with other lipid-lowering therapies, 14 children entered the trial with an average LDL-C level of 264 mg/dL, more than twice the target (<130 mg/dL) for children with HoFH.

After 24 weeks of treatment, 79% of patients reduced their LDL-C by at least half, said the company.

Regeneron noted that an absolute 132 mg/dL reduction in LDL-C from baseline, on average.

The company said there were 2 severe adverse events (AEs) (aortic stenosis and tonsilitis),which were considered unrelated to treatment. Evkeeza was generally well-tolerated with all patients completing the trial.

The most common AEs were throat pain, abdominal pain, diarrhea, headache and nasopharyngitis.

Evkeeza is already approved in the U.S., as evinacumab-dgnb, and EU as an adjunct therapy for certain patients aged 12 years and older with HoFH.

Regeneron is responsible for developing and distributing Evkeeza in the U.S. and is collaborating with Ultragenyx (RARE) to develop and sell Evkeeza outside the U.S.