PRALUENT (alirocumab) - Children and adolescents aged 8 years and over with heterozygous familial hypercholesterolaemia (HFHe)

The Committee deems that the clinical benefit of PRALUENT (alirocumab) 75 mg, 150 mg and 300 mg solution for injection is substantial in paediatrics only, in children and adolescents aged 8 years and over with 
heterozygous familial hypercholesterolaemia (HFHe) inadequately controlled (LDL-c > 130 mg/L) with oral lipid-lowering treatment at the maximum tolerated dose, as anadjunct to diet, and:

• in combination with an optimised lipid-lowering therapy;
• or as monotherapy in the event of contraindication or known intolerance to both 
  statins and ezetimibe. 

Considering: 

• evidence of the superiority of PRALUENT (alirocumab) compared to placebo in terms of the reduction in LDL-c levels at 24 weeks (biological primary endpoint) in a comparative, randomised, double-blind study, with a moderate effect size (- 33.8%; CI97.5% = [- 46.4; - 21.2]; p < 0.0001);
• reduction in LDL-c levels at 24 weeks (biological primary endpoint) in a comparative, random ised, double-blind study, with a moderate effect size (- 33.8%; CI97.5% = [- 46.4; - 21.2]; p < 0.0001);
•  the absence of data relative to a potential effect of alirocumab on morbidity and mortality in children and adolescents aged 8 years and over;
•  the paediatric safety profile judged to be acceptable but with persistent uncertainties concerning the impact of the marked reduction in LDL-c on the neurocognitive functions and growth of the children;
• the absence of treatment compliance and quality of life data;
• the currently partially met medical need; 

the Committee deems that PRALUENT (alirocumab) 75 mg, 150 mg and 300 mg solution for injection provides no clinical added value (CAV V) in the current care pathway for the treatment of heterozygous familial hypercholesterolaemia in children and adolescents aged 8 years and over