Rituximab Granted FDA Approval to Treat Pemphigus Vulgaris

The FDA has approved Genentech’s innovator rituximab (Rituxan) for the treatment of adults with moderate to severe pemphigus vulgaris (PV). This expanded indication makes the drug the first biologic approved for the treatment of PV, a rare autoimmune disease that affects the skin and mucous membranes with progressive blistering. The life-threatening condition affects approximately 30,000 to 40,000 people in the United States.

The FDA had previously granted the biologic priority review, breakthrough therapy designation, and Orphan Drug Designation for the treatment of PV. In total, the reference product is now licensed to treat 6 diseases: non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis, and PV.

“Today’s decision by the FDA provides the first approved treatment option in more than 60 years for patients with [PV], a potentially life-threatening disease,” said Sandra Horning, MD, chief medical office and head of global product development at Genentech, in a statement.

The approval was based on data from a randomized, controlled, open-label, multicenter study—the Ritux 3 trial—conducted in France. The study compared the EU-approved rituximab product plus short-term corticosteroids (collectively, the Ritux 3 regimen) to corticosteroids alone as a first-line treatment in patients with moderate to severe pemphigus.

The primary endpoint of the study was complete remission (defined as complete epithelialization and absence of new and/or established lesions) at 24 months without the use of steroids for 2 or more months, and was achieved by 90% of patients treated with the Ritux 3 regimen versus 28% of patients treated with corticosteroids alone.

Based on the results of the trial, the International Bullous Disease Consensus Group developed international diagnostic and management guidelines for PV. A preliminary survey based on the European Dermatology Forum and the European Academy of Dermatology and Venereology guidelines was sent to a panel of international experts to determine the level of consensus in regard to the treatment of PV with rituximab and short-term corticosteroids.

After the results were initially discussed during the annual American Academy of Dermatology conference, a second survey was sent out to achieve greater international consensus. In total, 39 experts participated in the first round of the survey, while 54 experts from 21 different countries participated in the second round. The international consensus was to recommend reference rituximab and corticosteroids as first-line therapy options for moderate to severe PV.