Posted by Michael Wonder on 26 Sep 2025
Evkeeza (evinacumab-dgnb) ANGPTL3 antibody approved in the US for children as young as 1 year old with ultra-rare form of high cholesterol
26 September 2025 - Regeneron Pharmaceuticals today announced that the US FDA has approved Evkeeza (evinacumab-dgnb) ANGPTL3 antibody as an adjunct to diet and exercise and other lipid-lowering therapies for the treatment of children from age 1 to less than 5 years old with homozygous familial hypercholesterolaemia.
The extended indication for Evkeeza was supported by clinical efficacy and safety data among 6 children living with homozygous familial hypercholesterolaemia (including pharmacokinetic data among 4 of these patients) who took part in either the US expanded access program or ex-US compassionate use program for Evkeeza.
