Alirocumab (3) – Praluent®

• Clinical trials
  • The randomised, controlled trial EFC14643 included children and adolescents aged between 8 and 17 years with heterozygous familial hypercholesterolaemia who, despite statin treatment with or without additional lipid-modifying therapy, or in cases of statin intolerance despite treatment with other (non-statin-based) lipid-modifying therapies.

a) Children and adolescents with heterozygous familial hypercholesterolaemia aged between 8 and 17 years, for whom dietary and pharmacological options for lowering lipids have not been fully utilised

• The additional benefit is not proven.
• The registration study EFC14643, which was presented by the pharmaceutical manufacturer only as supplementary evidence, is not suitable for the present benefit assessment of alirocumab for patient group a), as there was no scope within the study to adjust lipid-modifying therapy and the appropriate comparator therapy was therefore not implemented.

b) children and adolescents with heterozygous familial hypercholesterolaemia aged between 8 and 17 years, in whom dietary and pharmacological options for lowering lipid levels have been exhausted

• An additional benefit is not proven.
• The EFC14643 registration trial, which was presented by the pharmaceutical manufacturer only as supplementary evidence, is not suitable for the present benefit assessment of alirocumab for patient group b), as evolocumab and LDL apheresis were not administered in the study and the appropriate comparator therapy was therefore not implemented.

Courtesy translation only, please refer to the German original.