EVKEEZA (evinacumab) - Paediatric patients aged 6 months to 4 years with homozygous familial hypercholesterolaemia (HoFH)

The clinical benefit of EVKEEZA (evinacumab) 150 mg/ml concentrate for solution for infusion is substantial in the MA indication.

Considering:

• the absence of clinical study data for the paediatric population under 5 years of age;
• the data previously submitted and analysed concerning the other populations for which evinacumab is indicated (children aged 5 to 11 years [non-comparative CL-17100 study and extrapolation data] and adults and adolescents [comparative ELIPSE-HoFH study]);
• reassuring use data from compassionate access programmes in children under 5 years of age and an extrapolation analysis of the efficacy of evinacumab in this population based on a pharmacokinetics (PK) and PK/PD (pharmacodynamics) simulation model;
• the absence of any new safety signals and the relatively favourable safety profile of evinacumab;

the Committee deems that EVKEEZA (evinacumab) 150 mg/ml concentrate for solution for infusion provides a minor clinical added value (CAV IV) in the current care pathway for the treatment of children aged 6 months to 4 years with homozygous familial hyper-cholesterolaemia.