Favourable opinion for maintenance of reimbursement for the treatment of adults and adolescents aged 12 years and older 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) remains substantialin the MA indication.
Clinical Added Value
moderate
Therapeutic improvement in the care pathway.
Considering:
the final results of the open-label extension study (OLE) with longer follow-up of evinacumab treatment in 116 patients (median follow-up of 2 years with around 25% of patients having a treatment follow-up period of ≥ 2.5 years), which demonstrate maintenance of the efficacy of evinacumab on LDL-c reduction and support the safety profile judged to be favourable for evinacumab with no new safety signals observed;
the inadequately met medical need to have access to effective and well-tolerated medicines reducing the need for LDL apheresis, in patients not controlled despite optimised lipid-lowering treatment, in particular those with a biallelic mutation on the LDLR gene devoid of functional LDLR receptors;
the Transparency Committee deems that EVKEEZA (evinacumab) provides a moderate clinical added value (CAV III) in the care pathway for the treatment of adults and adolescents aged 12 years and older with homozygous familial hypercholesterolaemia (HoFH) as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies.
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