Caplacizumab (2) – Cablivi®

• Clinical trials
  • The HERCULES trial is a completed, multicentre, double-blind, placebo-controlled Phase IIIstudy with a parallel-group design, which investigated the efficacy and safety of caplacizumab when administered as part of a combination therapy (daily plasma exchange and immunosuppressive therapy) in adult aTTP patients.

Adolescents aged 12 to < 18 years and weighing at least 40 kg who are suffering from an episode of acquired thrombotic thrombocytopenic purpura (aTTP)

• No clinical trials are available in adolescents aged 12 to < 18 years and weighing at least 40 kg with an episode of aTTP in which caplacizumab was used and investigated.
• The pharmaceutical manufacturer cites two case reports for the specified patient population of adolescents. These case reports are not taken into account for the benefit assessment, as it is not possible to assess the reliability of the findings or the extent of the additional benefit on the basis of case reports.
• For the benefit assessment of caplacizumab in adolescents aged 12 to < 18 years and weighing at least 40 kg with an episode of acquired thrombotic thrombocytopenic purpura (aTTP), the pharmaceutical manufacturer refers, amongst other things, to the HERCULES study as part of an evidence transfer from adults to adolescents with aTTP.
• The evidence transfer sought by the marketing authorisation holder from the adult HERCULES study to the adolescent study population under assessment is not accepted, as results on patient-relevant endpoints for the adolescent study population are lacking; consequently, it is not possible within the scope of this benefit assessment to perform evidence transfer from the HERCULES study in adults to adolescents.
• Consequently, the G-BA classifies the extent of the additional benefit of caplacizumab in the treatment of adolescents aged 12 years and over and weighing at least 40 kg who are suffering from an episode of acquired thrombotic thrombocytopenic purpura (aTTP), when used in combination with plasmapheresis and immunosuppression, is non-quantifiable. An additional benefit exists in accordance with Section 35a(1), sentence 11, first half-sentence, of SGB V, but is non-quantifiable because the scientific evidence does not permit this.

Courtesy translation only, please refer to the German original.