Sofosbuvir / Velpatasvir (3) – Epclusa®

• Clinical trials
  • The pharmaceutical manufacturer has submitted the results of the non-randomised, multicentre, single-arm study GS-US-342-1143 for patients aged 3 to under 6 years.

Children with chronic hepatitis C aged 3 to < 6 years

• mortality
  • No deaths occurred.
• morbidity
  • 87.9% of patients aged 3 to < 6-year-old patients with genotype 1 or 4, and 62.5% of patients with genotype 2 or 3 treated with sofosbuvir/velpatasvir, achieved a sustained virological response 12 or 24 weeks after the end of treatment (SVR12, SVR24).
  • For the combination therapies designated as appropriate comparator therapies, in children aged 3 to < 12 years, ledipasvir/sofosbuvir (relevant for patients with genotypes 1, 4, 5 or 6) achieved a sustained virological response 12 and 24 weeks after the end of treatment in ≥ 95 per cent of patients.
  • For the appropriate comparator therapy, glecaprevir/pibrentasvir, SVR12 rates of 98.3% were observed in patients with genotypes 1, 4, 5 or 6, and 90% in patients with genotypes 2 or 3.
• Health-related quality of life
  • Health-related quality of life was assessed in the study using the PedsQL (Pediatric Quality of Life Inventory) at the start of the study and at week 24 following the end of treatment.
  • This resulted in an average change in the total score of –1.9 points by week 24 over the course of the study.
  • Due to the lack of comparative data, the results cannot be adequately interpreted.
• Side effects
  • Overall, one withdrawal due to adverse events was observed.
  • No serious adverse events (SAEs) or severe adverse events occurred in the study.
• Overall assessment
  • The single-arm study GS-US-342-1143 presented here is not suitable for assessing additional benefit due to the lack of comparison with the appropriate comparator therapy; this would only be possible, at best, if there were very large effects compared with the appropriate comparator therapy.
  • Given the high proportion of patients who experienced therapy discontinuation and the associated proportion of imputed values, the available data from the study allow only a very limited interpretation of the results.
  • A sustained virological response 12 or 24 weeks after the end of treatment was achieved with sofosbuvir/velpatasvir in 87.9% of patients with genotype 1 or 4 and in 62.5% of patients with genotype 2 or 3.
  • No deaths occurred. One adverse event occurred that led to therapy discontinuation. The available data on health-related quality of life are insufficient to allow for meaningful interpretation.
  • Overall, no additional benefit can be inferred on the basis of the data presented.

Courtesy translation only, please refer to the German original.