MABTHERA (rituximab) - Moderate to severe pemphigus vulgaris (PV) in adults

The clinical benefit provided by MABTHERA100 mg and 500 mg (rituximab), concentrate for solution for  infusion, remains significant in the MA indication.

In view of:

• the results previously provided by the phase III study (ML22196), which demonstrated the superiority of a short course (3-6 months) of the combination rituximab + prednisone at a low dose, compared to prednisone alone at a standard dose for a prolonged course (12-18 months), in terms of complete remission after 24 months, in adults with moderate to severe pemphigus vulgaris (PV) (89.5% vs 27.8%, p < 0.0001), despite the methodological weaknesses of the study (open study, with no control of alpha risk, analysis of a subgroup of patients);
• the results of a new phase III study (PEMPHIX), with good quality methodology, comparative versus mycophenolate mofetil (MMF), in combination with brief corticosteroid therapy in each group, in adults with active moderate to severe PV requiring oral corticosteroid therapy, which demonstrated:
  • the superiority of rituximab compared to MMF after 52 weeks of treatment with regard to the percentage of patients obtaining a complete response (with discontinuation of corticosteroid therapy) maintained for at least 16 weeks with a significant effect: 40.3% vs 9.5% respectively (p <0.0001),
  • the superiority of rituximab compared with MMF after 52 weeks of treatment, with also a significant effect, on the following hierarchical endpoints:
    • cortisone sparing,
    • number of relapses during treatment,
    • time period until the first relapse,
    • time period until complete lasting remission was obtained,
  • the superiority of rituximab compared to MMF with regard to the variation in the DLQI (quality of life) score, although the difference observed is not clinically relevant (-2.87 points);
• the unchanged tolerance profile, marked mainly by the reactions associated with the infusion and infections;

the Committee considers that MABTHERA 100 mg and 500 mg (rituximab) now provides moderate clinical added value (CAV III) in the management of moderate to severe pemphigus vulgaris in adults, which comprises long-term corticosteroid therapy and short-term corticosteroid therapy combined with an immunosuppressant (mycophenolate mofetil, methotrexate or azathioprine).