Lanadelumab (3) – Takhzyro®

• Clinical trials
  • The pharmaceutical manufacturer conducted the single-arm SPRING trial, which formed the basis for the marketing authorisation (24 children aged 2 to 11 years with a documented diagnosis of HAE (type I or II) over 12 weeks), which is not suitable for assessing additional benefit due to the lack of a comparison with the appropriate comparator therapy.

Children aged 2 to < 12 years with recurrent attacks of hereditary angioedema

• For children aged 2 to < 12 years, for the routine prophylaxis of recurrent attacks of hereditary angioedema, additional benefit of lanadelumab over the appropriate comparator therapy is not proven.
• In its dossier for the assessment of the additional benefit of lanadelumab, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy.
• Overall, no additional benefit can be inferred for children aged 2 to < 12 years for the routine prophylaxis of recurrent attacks of hereditary angioedema compared with the appropriate comparator therapy.
• Overall assessment
  • Against this background, an additional benefit is not proven for children aged 2 to < 12 years for the routine prophylaxis of recurrent attacks of hereditary angioedema.

Courtesy translation only, please refer to the German original.