Avapritinib (6) – Ayvakyt®

• Clinical trials
  • The NAVIGATOR study is a single-arm, multicentre Phase I study of avapritinib, divided into a dose-escalation phase (Part 1) and an expansion phase (Part 2).
  • The CS3007-101 trial is a non-randomised Phase I/II trial of avapritinib, conducted exclusively in China.

Adult patients with inoperable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation

• The additional benefit is not proven.
• Consequently, there are no suitable data available overall for assessing the additional benefit of avapritinib. Consequently, no additional benefit of avapritinib has been demonstrated for patients with inoperable or metastatic gastrointestinal stromal tumours (GIST) who carry the platelet-derived growth factor receptor alpha (PDGFRA)-D842V mutation, compared with the appropriate comparator therapy, does not provide proof.
• mortality
  • The single-arm studies NAVIGATOR and CS3007-101 submitted by the pharmaceutical manufacturer do not allow for a comparison with the appropriate comparator therapy and are therefore not suitable for assessing the additional benefit of avapritinib as monotherapy.
• morbidity
  • The single-arm studies NAVIGATOR and CS3007-101 submitted by the pharmaceutical manufacturer do not allow for a comparison with the appropriate comparator therapy and are therefore not suitable for assessing the additional benefit of avapritinib as monotherapy.
• Side effects
  • The single-arm studies NAVIGATOR and CS3007-101 submitted by the pharmaceutical manufacturer do not allow for a comparison with the appropriate comparator therapy and are therefore not suitable for assessing the additional benefit of avapritinib as monotherapy.
• Conclusion
  • The single-arm studies NAVIGATOR and CS3007-101 submitted by the pharmaceutical manufacturer do not allow for a comparison with the appropriate comparator therapy and are therefore not suitable for assessing the additional benefit of avapritinib as monotherapy.
  • The results of the VOYAGER study, which were already submitted in the dossier for the initial assessment and are presented comparatively in the results section, a propensity score (PS)-adjusted indirect comparison with the NAVIGATOR and BLU-285-1002 studies and the retrospective study by Cassier et al, 2012, were not analysed in the present procedure and are therefore not suitable for assessing additional benefit.
  • Consequently, there are no suitable data available overall for the assessment of the additional benefit of avapritinib.

Courtesy translation only, please refer to the German original.