SOLIRIS (éculizumab) - Atypical haemolytic uraemic syndrome (aHUS)

Opinions on drugs - Posted on Apr 25 2023

Reason for request

Reassessment

Reassessment.

Key points

Favourable opinion on the maintenance of reimbursement in the “treatment of atypical haemolytic uraemic syndrome”.

Role in the care pathway?

The new data are not of a nature to modify the previous conclusions of the Committee on the role of eculizumab in the therapeutic strategy:

SOLIRIS (eculizumab), a C5 complement inhibitor, remains a first-line treatment for aHUS in children (weighing 5 kg or more) and in adults. In the absence of a direct comparison with ravulizumab, the second C5 complement inhibitor with a MA in this indication, eculizumab remains the reference treatment for aHUS.

Additional clinical data remain necessary to better define the population that could benefit most from SOLIRIS (eculizumab) according to the disease aetiology and those patients in whom discontinuation of treatment with eculizumab may be suggested.

The Summary of Product Characteristics (SmPC) and the Risk Management Plan (RMP) must be respected, particularly the sections “4.2 Posology and method of administration” and “4.4 Special warnings and precautions for use” (see paragraph 8.2.4 Discussion).

The use of this drug in pregnant or breastfeeding women must comply with the SmPC (http://lecrat.fr/).

Clinical Benefit

Substantial

The clinical benefit provided by SOLIRIS 300 mg (eculizumab), concentrate for solution for infusion; remains significant in the MA indication.

Clinical Added Value

important

In view of:

the partially met medical need in a serious disease that is life-threatening if not treated,
the clinical data from 4 non-comparative phase II studies conducted in small numbers of paediatric, adolescent and adult patients with atypical haemolytic uraemic syndrome (aHUS) that showed:
- a significant percentage of patients at week 26 with no sign of TMA (thrombotic microangiopathy), with a normalisation of haematological and renal parameters, and the long-term maintenance of clinical responses,
- reduced use of plasma therapy and dialysis in all of these studies,

despite the absence of data on patients’ quality of life, while this is strongly impacted in this disease,

the fact that SOLIRIS (eculizumab) remains the reference treatment for aHUS in the absence of a comparative study versus ravulizumab (ULTOMIRIS),

the Committee considers that SOLIRIS 300 mg (eculizumab), concentrate for solution for infusion, still provides significant clinical added value (CAV II) in the management of atypical haemolytic uraemic syndrome (aHUS) which includes ULTOMIRIS (ravulizumab).