Baloxavir marboxil (2) – Xofluza®

• Clinical trials
  • The BLOCKSTONE trial is a randomised, double-blind, placebo-controlled trial (RCT) comparing baloxavir marboxil with placebo, which was conducted exclusively in Japan.

a) Adults and adolescents aged 12 years and over who have been exposed to influenza and are not at risk of influenza-related complications

• For adults and adolescents aged 12 years and over who have been exposed to influenza and are not at risk of influenza-related complications, there is an indication of a considerable additional benefit of baloxavir marboxil compared with a ‘wait-and-see’ approach.
• This assessment is based on the results of the BLOCKSTONE RCT involving adults and adolescents aged 12 years and over who have been exposed to influenza and are not at risk of influenza-related complications.
• Overall, the certainty of evidence for the identified additional benefit is classified as ‘indication’.
• mortality
  • In the BLOCKSTONE study, no statistically significant difference was observed between the treatment groups for the endpoint of overall survival.
• morbidity
  • The endpoint ‘symptomatic influenza infection’ was defined as fever or at least one other influenza symptom (cough, sore throat, runny nose/nasal congestion, headache, chills, muscle or joint pain, or fatigue), together with a positive RT-PCR test.
  • For the endpoint of symptomatic influenza infection – and thus, in the overall assessment of the morbidity category – a statistically significant advantage was observed in favour of baloxavir marboxil compared with a ‘wait-and-see’ approach in both selected analyses.
• quality of life
  • The endpoint of health-related quality of life was not assessed in the BLOCKSTONE study.
• Side effects
  • No statistically significant difference was observed between the treatment groups for the endpoints of serious adverse events (SAEs) and discontinuation due to adverse events.
  • A summary of the results shows no difference between baloxavir marboxil and a ‘wait-and-see’ approach in the ‘side effects’ category.
• Overall assessment / Conclusion
  • The double-blind, randomised, controlled BLOCKSTONE study was presented to assess the extent of the additional benefit of baloxavir marboxil.
  • Results are available on mortality, morbidity and side effects. The study did not assess health-related quality of life.
  • For the endpoint of overall survival, there was no statistically significant difference between baloxavir marboxil and a ‘wait-and-see’ approach.
  • In the overall analysis of the results in the morbidity category for the endpoint of symptomatic influenza infection, a statistically significant advantage was observed in favour of baloxavir marboxil compared with a ‘wait-and-see’ approach.
  • In the overall analysis of the endpoints in the ‘side effects’ category for the endpoints ‘serious adverse events’ (SAEs) and ‘discontinuation due to adverse events’, no statistically significant difference was observed between baloxavir marboxil and watchful waiting.
  • In summary, for adults and adolescents aged 12 years and over who have been exposed to influenza and are not at risk of influenza-related complications, the overall analysis of the results regarding mortality, morbidity and side effects, baloxavir marboxil offers considerable additional benefit compared with a ‘wait-and-see’ approach.

b) Adults and adolescents aged 12 years and over who have been exposed to influenza and are at risk of influenza-related complications

• For adults and adolescents aged 12 years and over who have been exposed to influenza and are at risk of influenza-related complications, the additional benefit of baloxavir marboxil compared with the appropriate comparator therapy is not proven.
• For the patient group ‘adults and adolescents aged 12 years and over who have been exposed to influenza and are at risk of influenza-related complications’, the pharmaceutical manufacturer did not submit any study that would have been suitable for assessing the additional benefit of baloxavir marboxil in this therapeutic indication compared with the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.