Sucroferric Oxyhydroxide (2) – Velphoro®

• Clinical trials
  • The pharmaceutical manufacturer has submitted the study PA-CL-PED-01, which forms the basis for the extension of the marketing authorisation, for the benefit assessment. This is a multicentre, open-label, randomised study comparing sucrroferric oxyhydroxide with calcium acetate for the control of serum phosphate levels in children and adolescents with CKD.

Children and adolescents aged 2 years and over with chronic kidney disease (CKD) at stages 4–5 (defined by a GFR < 30 ml/min/1.73 m²) or CKD requiring dialysis

• For children and adolescents aged 2 years and over with chronic kidney disease (CKD) at stages 4–5 (defined by a GFR < 30 ml/min/1.73 m²) or CKD requiring dialysis, the additional benefit of sucrroferric oxyhydroxide compared with the appropriate comparator therapy is not proven.
• On balance, the G-BA therefore concludes that the additional benefit of sucrroferric oxyhydroxide compared with the appropriate comparator therapy is not proven.
• Morbidity – serum phosphate concentration
  • The primary endpoint of the study was the trend in serum phosphate concentration in the intervention arm up to the end of the titration phase.
  • Secondary endpoints were the comparison of serum phosphate concentrations in the intervention and control arms, as well as side effects (including deaths).
  • For the analysis following the treatment period specified in the study protocol, data were available for only 39 per cent of the children and adolescents treated with sucrroferric oxyhydroxide and 10 per cent of those treated with calcium acetate.
  • Based on the data on the discontinuation rate in both arms and phases (titration and maintenance), it can be estimated that in the intervention arm less than half, and in the control arm less than a quarter, of the randomised participants were treated for the duration of at least 24 weeks required for chronic conditions.
• Side effects
  • Adverse events were cited as the most common cause of premature discontinuation of treatment with the study medication; these occurred in 12 children and adolescents in the intervention arm (≙ 18 per cent) and 6 children and adolescents in the control arm (≙ 32 per cent).
  • Furthermore, lack of adherence or withdrawal of consent led to discontinuation in 16 children and adolescents in the intervention arm (≙ 24 per cent) and 6 children and adolescents in the control arm (≙ 32 per cent).
• Overall view
  • In the PA-CL-PED-01 study, there was a high rate of early therapy discontinuation, which differed between the intervention and control arms, meaning that, overall, significantly fewer than 50% of the children and adolescents included in the study could be treated and assessed over the period of at least 24 weeks required for chronic conditions.
  • Furthermore, the study presented did not collect any patient-relevant endpoints relating to morbidity or quality of life.
  • Furthermore, due to the single-comparator design using calcium acetate, the target population for the new therapeutic indication is not fully represented.
  • Based on the available data, conclusions regarding additional benefit can only be drawn in comparison with calcium acetate.

Courtesy translation only, please refer to the German original.