ANGPTL3 inhibitor approved by FDA for young children with HoFH

The FDA has approved the use of the ANGPTL3 inhibitor evinacumab as an adjunct to other lipid-lowering therapies for the treatment of children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH).

The FDA has extended the indication of evinacumab as an adjunct to other lipid-lowering therapies for the treatment of children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). In Feb 2021, evinacumab was approved by the FDA in HoFH patients of 12 years and older .

In a single-arm, open-label phase 3 extension trial, treatment with evinacumab (15 mg/kg every four weeks administered by IV) reduced LDL-c by 48% at week 24 on average in pediatric patients with HoFH (n=20). Levels of ApoB, non-HDL-c and total cholesterol were also reduced.

The safety profile that was seen in these 20 pediatric patients was similar to the safety profile observed in adults and pediatric patients of 12 years and older, with the additional adverse reaction of fatigue (in 3 [15%] patients).

Source: press release Regeneron, March 22, 2023