FDA approval for PCSK9i in children with heterozygous FH of 10 years and olderNews - Sep. 27, 2021
The PCSK9 inhibitor evolocumab has now been approved by the FDA for the treatment of children with heterozygous familial hypercholesterolemia (HeFH) to lower LDL-c levels.
This approval is based on findings in the_HAUSER-RCT_phase 3b trial, in which the safety and efficacy of evolocumab was examined in pediatric patients, 10-17 of age, with HeFH. LDL-c was reduced by a mean of 38% (95%CI: 31-45%, P<0.0001) at week 12 from baseline with monthly treatment with evolocumab compared to placebo. Secondary endpoints of non-HDL-c, total cholesterol and ApoB were all reduced in patients treated with evolocumab after 24 weeks from baseline compared to placebo.
No new safety risk was reported. Most common treatment-emergent adverse events included nasopharyngitis, headache, oropharyngeal pain, influenza and upper respiratory tract infection.
Evolocumab was already approved in homozygous FH patients of 13 years and older.