Rituximab was found to be superior to mycophenolate mofetil (MMF) in adult patients with severe pemphigus vulgaris, according to new phase 3 trial data.

The PEMPHIX study evaluated the efficacy and safety of rituximab compared with MMF in patients with moderate-to-severely active pemphigus vulgaris (confirmed diagnosis of pemphigus vulgaris within the previous 24 months) requiring 60mg to 120mg per day of oral prednisone or an equivalent (N=135). Patients were randomized to receive rituximab or MMF in combination with 60mg or 80mg oral prednisone, with the aim of tapering to 0mg/day by week 24. The primary end point of the study was the percentage of patients who achieved sustained complete remission (defined as achieving healing of lesions with no new active lesions), as measured by the Pemphigus Disease Area Index (PDAI) Activity Score, at Week 52.

Results showed that 40.3% of patients treated with rituximab achieved sustained complete remission without the use of steroids for 16 consecutive weeks or more, compared with 9.5% of patients who received MMF (P<.0001). In addition, rituximab treatment was associated with lower cumulative oral corticosteroid doses, fewer flares, a greater likelihood of sustained complete remission and improvement in health-related quality of life (all secondary end points).

With regard to safety, adverse events were found to be consistent with those previously observed in other trials involving rituximab. The ongoing study also includes a 48-week safety follow-up period.


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Rituximab (Rituxan; Genentech), a CD20-directed cytolytic monoclonal antibody, was approved for the treatment of severe pemphigus vulgaris in June 2018. “The approval of [rituximab] for the treatment of pemphigus vulgaris was the first major advancement in the treatment of this rare, serious disease in more than 60 years,” said Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development. “The PEMPHIX study showed that 40% of people in the study could achieve complete remission from painful blistering without the need for corticosteroids for 16 weeks or more and that [rituximab] may be a superior treatment option to mycophenolate mofetil.”

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