Ravulizumab (3) – Ultomiris®

• Clinical trials
  • The ALXN1210-PNH-304 trial is an ongoing single-arm Phase III trial.
  • The M07-005 trial is a single-arm Phase I/II trial.

a) Paediatric patients weighing 10 kg or more with paroxysmal nocturnal haemoglobinuria (PNH) and high disease activity, characterised by clinical symptoms of haemolysis

• An additional benefit is not proven.
• In the absence of direct comparative studies of ravulizumab against the appropriate comparator therapy, the pharmaceutical manufacturer presents the two single-arm studies ALXN1210-PNH-304 (with ravulizumab as the intervention) and M07-005 (with eculizumab as the intervention) and presents a purely descriptive comparison of the results of these two studies.
• The 12-week treatment duration in the M07-005 study is too short for comparison with the ALXN1210-PNH-304 study, given the chronic nature of the condition and the recommended lifelong treatment for patients.
• The comparison of the two studies is therefore not suitable for the present assessment of patient group a).

b) Paediatric patients weighing 10 kg or more with paroxysmal nocturnal haemoglobinuria (PNH) who have been receiving eculizumab for ≥ 6 months and are clinically stable

• An additional benefit is not proven.
• In the absence of direct comparative studies of ravulizumab against the appropriate comparator therapy, the pharmaceutical manufacturer presents the two single-arm studies ALXN1210-PNH-304 (with ravulizumab as the intervention) and M07-005 (with eculizumab as the intervention) and presents the results of these two studies in a purely descriptive manner.
• As all patients in study M07-005 belong to patient group a), no comparative data against the appropriate comparator therapy are available for the assessment of patient group b).

Courtesy translation only, please refer to the German original.