Favourable opinion for reimbursement of EVKEEZA (evinacumab) in the treatment of paediatric patients aged 5 to 11 years with homozygous familial hypercholesterolaemia (HoFH) as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies.
Clinical Benefit
Substantial
The Committee deems that the clinical benefit of EVKEEZA (evinacumab) 150 mg/ml concentrate for solution for infusion is substantial in the MA indication.
Clinical Added Value
moderate
Considering:
the seriousness of the cardiovascular diseases promoted by HoFH,
the rarity of this disease with a low prevalence, particularly in children,
the medical need to have access to effective and well-tolerated medicines in children with HoFH,
the partial response of EVKEEZA (evinacumab) to the medical need, due to:
the demonstrated impact of EVKEEZA (evinacumab) on LDL-c reduction but in the absence of comparative data and a fully demonstrated impact on morbidity and mortality,
the lack of evidence of an impact of EVKEEZA (evinacumab) on quality of life and the organisation of care,
EVKEEZA (evinacumab) is unlikely to have an additional impact on public health.
Considering all these elements, the Committee deems that the clinical benefit of EVKEEZA (evinacumab) 150 mg/ml concentrate for solution for infusion is substantial in the MA indication.
The Committee issues a favourable opinion for inclusion of EVKEEZA (evinacumab) 150 mg/ml concentrate for solution for infusion in the list of covered drugs for inpatients approved for use in the indication and at the MA dosages.
Listen
