The U.S. Food and Drug Administration (FDA) has approved evinacumab-dgnb ANGPTL3 antibody (Evkeeza®) to treat homozygous familial hypercholesterolemia (HoFH) in children as young as 1-year-old, according to a Regeneron® press release.

Evinacumab-dgnb was approved for use in children with HoFH aged 12 years and up in 2021, then children aged 5 to 11 in 2023. This latest approval for use in even younger children "was supported by clinical efficacy and safety among six children living with HoFH (including pharmacokinetic data among four of these patients) who took part in either the U.S. expanded access program or ex-U.S. compassionate use program for Evkeeza."

Access CardioSmart resources on FH, including an infographic specifically addressing HoFH and how it is diagnosed and treated.

Keywords: ACC Advocacy, Homozygous Familial Hypercholesterolemia, United States Food and Drug Administration