Ravulizumab (3) – Ultomiris®

Paroxysmal hemoglobinuria, pediatric patients

Characteristics

Start date 01.10.2021 – Marketing authorisation: 01.09.2021
Resolution 18.03.2022
INN Ravulizumab
Brand name Ultomiris®
Pharm. company Alexion Pharma Germany GmbH
G-BA Procedure ID D-720
ATC code L04AJ02 IMMUNOSUPPRESSANTS (L04A)
ICD-10 codes (AIS) D59.5Paroxysmal nocturnal hemoglobinuria [Marchiafava-Micheli]
Alpha-ID codes (AIS) I118016PNH (paroxysmal nocturnal hemoglobinuria)
DDD 58.9 mg P
Therapeutic area Hematopoietic diseases Paroxysmal nocturnal hemoglobinuria (PNH)
Reason for procedure New therapeutic indication

Therapeutic indication of the resolution

Ultomiris is indicated in the treatment of adult and paediatric patients with a body weight of 10 kg or above with paroxysmal nocturnal haemoglobinuria (PNH):

- in patients with haemolysis with clinical symptom(s) indicative of high disease activity.

- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months

Subpopulation Indication Comparator
a) Paediatric patients weighing 10 kg or more with paroxysmal nocturnal haemoglobinuria (PNH) with high disease activity, characterised by clinical symptoms of haemolysis. Eculizumab
b) Paediatric patients with a body weight of 10 kg or more with paroxysmal nocturnal haemoglobinuria (PNH) who have been receiving eculizumab for ≥ 6 months and are clinically stable Eculizumab

Studies and Results

No. of studies
(best subpopulation) 		0 (Data not accepted)
Study design
(best subpopulation) 		Data not accepted (Dossier: Single-arm + no comparison)
Reason for dividing into subpopulations (G-BA) Previous treatment

  • 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.

Associated procedures



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