Favourable opinion for reimbursement only “as an add-on to standard therapy, including first-line immunosuppressants, for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive, who remain symptomatic.”
Unfavourable opinion for reimbursement in the other situations covered by the MA indication.
Clinical Benefit
Substantial
The Committee deems that the clinical benefit of RYSTIGGO (rozanolixizumab) is substantial only as an add-on to standard therapy, including first-line immunosuppressants, for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive, who remain symptomatic.
Insufficient
The Committee deems that the clinical benefit of RYSTIGGO (rozanolixizumab) is insufficient to justify public funding in the other clinical situations of the MA.
Clinical Added Value
minor
Considering:
evidence of the superiority of rozanolixizumab compared to placebo:
in terms of the change from baseline to D43 in MG-ADL score (primary endpoint), with a mean change (SD) of - 2.59 points (95% CI [-4.091; -1.249]); p<0.001),
on the clinical ranked secondary endpoints of disease severity assessed at D43 (Myasthenia Gravis Composite score (MG-C), QMG score, changes in “Muscle Weakness Fatigability”, “Physical Fatigue” and “Bulbar Muscle Weakness” components of the MG symptoms PRO score),
but in view of:
comparative efficacy data limited to the short term (6 weeks),
the absence of robust data on quality of life, despite this being a relevant endpoint in this disease, with this having been assessed as an exploratory endpoint,
the safety profile reported only in the short term (median follow-up of around 7 months) and associated long-term uncertainties,
the lack of comparison versus rituximab or eculizumab despite the fact that this would have been possible; as well as the absence of comparison versus efgartigimod alfa, ravulizumab or zilucoplan due to their concomitant development,
the Committee deems that, like ULTOMIRIS, VYVGART and ZILBRYSQ (with the exclusion of rituximab and SOLIRIS), RYSTIGGO (rozanolixizumab) provides a minor clinical added value (CAV IV) in the care pathway as an add-on to standard therapy, including first-line immunosuppressants, for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive, who remain symptomatic.