Isavuconazol (2) – Cresemba®

• Clinical trials
  • Study 9766-CL-0107 is a single-arm, open-label, multicentre Phase II study designed to investigate the safety, pharmacokinetics and efficacy of isavuconazole in children and adolescents aged 1 to ≤ 17 years with invasive aspergillosis or mucormycosis.

Children and adolescents aged 1 to ≤ 17 years with invasive aspergillosis

• For children and adolescents aged 1 to ≤ 17 years with invasive aspergillosis, there is a hint of a non-quantifiable additional benefit, as the scientific evidence does not permit quantification.
• The strength of the evidence is therefore classified as ‘hint’.
• mortality
  • The primary endpoint of the study was overall mortality up to day 42. By day 42, two people had died: one with confirmed or probable invasive aspergillosis and one with possible invasive fungal disease.
• morbidity
  • In study 9766-CL-0107, morbidity was assessed using the secondary efficacy endpoints of clinical, radiological and mycological response, as well as the composite efficacy endpoint of overall response, which comprises the aforementioned individual components.
  • Due to existing uncertainties regarding patient relevance and/or the validity of the data collection, none of these endpoints could be used for the benefit assessment.
  • The endpoint ‘clinical response’, as a component of the composite endpoint ‘overall response’, comprises the complete or partial resolution of infection-related signs, symptoms and physical findings (successful clinical response).
  • The validity of the ‘clinical response’ endpoint is assessed as unclear.
  • Due to major uncertainties regarding its validity, the data for the ‘clinical response’ endpoint cannot be assessed as a whole and are therefore not used for the benefit assessment.
• quality of life
  • No data on health-related quality of life were collected in study 9766-CL-0107.
• Side effects
  • Adverse events (AEs) and serious adverse events (SAEs) were recorded from the time the informed consent form was signed until 60 days after the end of treatment (EOT). The operationalisation is considered valid. Due to the single-arm study design, no conclusions regarding the extent of the additional benefit can be drawn from the data.
• Overall assessment
  • For the present benefit assessment of the treatment of invasive aspergillosis in children and adolescents aged 1 year and over, the results of the pivotal, single-arm, open-label, multicentre Phase II trial 9766-CL-0107, which investigated the safety, pharmacokinetics and efficacy of isavuconazole in children and adolescents aged 1 to ≤ 17 years with invasive aspergillosis or mucormycosis.
  • In its overall assessment, the G-BA classifies the extent of the additional benefit as non-quantifiable, as the scientific evidence does not permit quantification.
• Overall assessment
  • In the overall assessment, there is therefore a hint of a non-quantifiable additional benefit for isavuconazole in the indication ‘invasive aspergillosis in children and adolescents aged 1 to ≤ 17 years’, as the scientific evidence does not permit quantification.

Courtesy translation only, please refer to the German original.