FDA approval for PCSK9i in children with heterozygous FH of 10 years and older
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. Secondary endpoints of non-HDL-c, total cholesterol and ApoB were all reduced in patients treated with evolocumab after 24 weeks from baseline.
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.