Ravulizumab (2) – Ultomiris®

Atypical hemolytic uremic syndrome (aHUS)

Characteristics

Start date 01.08.2020 – Marketing authorisation: 26.06.2020
Resolution 21.01.2021
INN Ravulizumab
Brand name Ultomiris®
Pharm. company Alexion Pharma Germany GmbH
G-BA Procedure ID D-557
ATC code L04AJ02 IMMUNOSUPPRESSANTS (L04A)
ICD-10 codes (AIS) D59.3Hemolytic-uremic syndrome
Alpha-ID codes (AIS) I1866Hemolytic-uremic syndrome
DDD 59 mg P
Therapeutic area Hematopoietic diseases Atypical haemolytic uremic syndrome (aHUS)
Reason for procedure New therapeutic indication

Therapeutic indication of the resolution

Ultomiris is indicated in the treatment of patients with a body weight of 10 kg or above with atypical haemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.
Subpopulation Indication Comparator
Patients weighing 10 kg or more with atypical haemolytic uraemic syndrome (aHUS) who have not previously been treated with complement inhibitors or have received eculizumab for at least 3 months and have shown a response to eculizumab. Eculizumab

Studies and Results

No. of studies
(best subpopulation) 		2 (Studie 311, Studie 312)
Study design
(best subpopulation) 		Single-arm + ITC (PID/PSM)
Meta analysis
(best subpopulation) 		no

  • Clinical trials
    • The single-arm, multicentre trials 311 and 312, which are currently still ongoing, are the pivotal trials for ravulizumab in this therapeutic indication.
    • Study 311 included adult patients with aHUS who had not previously received complement inhibitors.
    • Study 312 enrolled children and adolescents under 18 years of age with aHUS, weighing 5 kg or more, in two cohorts.
    • The single-arm, multicentre studies C10-003 and C10-004 included paediatric (body weight of at least 5 kg) and adult patients with aHUS, respectively.

Patients weighing at least 10 kg with atypical haemolytic uraemic syndrome (aHUS) who had not previously been treated with complement inhibitors or who had received eculizumab for at least 3 months and had demonstrated a response to eculizumab

  • For ravulizumab in the treatment of patients weighing 10 kg or more with atypical haemolytic uraemic syndrome (aHUS) who have not previously been treated with complement inhibitors or who have received eculizumab for at least 3 months and have demonstrated a response to eculizumab, an additional benefit is not proven.
  • An additional benefit of ravulizumab compared with the appropriate comparator therapy is not proven.
  • For the patient population that has received eculizumab for at least 3 months and has demonstrated a response to eculizumab, the pharmaceutical manufacturer has not provided any comparative data.
  • Overall, all the comparisons presented are subject to limitations relevant to the assessment, in particular due to the limited comparability of the studies and the lack of randomisation.
  • Overall assessment
    • In summary, therefore, the data submitted for the assessment of ravulizumab for the treatment of patients weighing 10 kg or more with atypical haemolytic uraemic syndrome (aHUS) who have not previously been treated with complement inhibitors or who have received eculizumab for at least 3 months and have demonstrated a response to eculizumab, are not suitable for inferring any additional benefit of ravulizumab over the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.

Associated procedures



<< List of all resolutions