A Phase III trial evaluating the use of rituximab (Rituxan, Genentech) in patients with pemphigus vulgaris (PV), a rare, chronic and potentially fatal, blistering skin disease has commenced. Rituximab is approved for numerous autoimmune disorders, including rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis.
The randomized, double-blind, double-dummy, active-comparator, multicenter, international study evaluated the efficacy and safety of rituximab compared to mycophenolate mofetil in patients with moderate-to-severely active PV. Patients in the rituximab arm received 1000 mg rituximab given by intravenous infusion on day 1 and 15, with repeat rituximab administration on day 168 and 182 provided specific safety criteria had been met.
The current standard of care includes high doses of corticosteroids taken for several weeks and corticosteroids in combination with the off-label use of corticosteroid-sparing immunosuppressive drugs for many months, which can cause significant, long-term side effects.
“Current treatments for pemphigus vulgaris can cause significant, long-term side effects,” said Victoria P. Werth, MD, professor of dermatology and medicine at the University of Pennsylvania and lead investigator for the PEMPHIX trial. “It is our hope that the PEMPHIX trial will demonstrate the efficacy and safety of rituximab for this disease and provide patients with a much needed, FDA-approved treatment option.”
