Rituxan to be Tested for Treatment of Pemphigus Vulgaris

Drug shows promise treating chronic and potentially life-threatening skin disease.

Drug shows promise treating chronic and potentially life-threatening skin disease.

A new clinical trial investigating the use of Rituxan in patients with pemphigus vulgaris (PV) was recently announced by the scientists at Genentech. PV is a rare, chronic, and potentially life-threatening skin disease that involves blistering of the skin.

Independent studies conducted to prove the safety and efficacy of Rituxan in patients with PV showed the drug may provide substantial improvement in PV remission rates. The current standard of care for patients with PV is to provide corticosteroids for several weeks and in combination with the off-label use of corticosteroid-sparing immunosuppressive drugs for many months.

The treatment can cause significant, long-term side effects. However, independent studies show that Rituxan can reduce the need for this treatment. If approved, Rituxan could be the first FDA-approved biologic treatment for the autoimmune disorder, which impacts approximately 30,000 people in the United States.

The drug was previously granted orphan drug status by the FDA.

“We are taking the knowledge gained from the efficacy demonstrated by Rituxan in B cell-mediated autoimmune diseases such as rheumatoid arthritis and ANCA-associated vasculitis and applying it to research for pemphigus vulgaris,” said Paul Brunetta, MD, associate group medical director of Genentech Immunology. “The ongoing investigation of Rituxan for the treatment of pemphigus vulgaris demonstrates Genentech’s commitment to follow the science and address the unmet medical needs of patients.”

A randomized, double-blind, double-dummy, active-comparator, multicenter, international study called PEMPHIX will evaluate the safety and efficacy of Rituxan in patients with moderate-to-severe PV. Patients in the Rituxan arm of the study will receive 1000mg rituximab given through intravenous infusion on Day 1 and 15, with repeat administration of the drug on Day 168 and 182 given safety criteria has been met.

“It is our hope that the PEMPHIX trial will demonstrate the efficacy and safety of Rituxan for this disease and provide patients with a much-needed, FDA-approved treatment option,” said Victoria P. Werth, MD, lead investigator for the PEMPHIX trial.

Rituxan is currently approved for treatment of rheumatoid arthritis, granulomatosis with polyangiitis, and microscopic polyangiitis. With positive results from the PEMPHIX trial, the drug could be approved for the treatment of patients with PV, effectively getting rid of the current treatment method that causes many unfavorable side effects.