Genentech has initiated a phase III clinical trial investigating the use of Rituxan (rituximab) in patients with pemphigus vulgaris (PV), a rare, chronic and potentially fatal, blistering skin disease. The aim of the study, called PEMPHIX, is to determine the efficacy and safety of Rituxan for the treatment of PV. 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.
Results of independent studies have shown that Rituxan may provide substantial improvement in PV remission rates and successful tapering and/or cessation of corticosteroid therapy. If approved, Rituxan could be the first FDA-approved biologic treatment for the autoimmune disorder, which impacts about 30,000 people in the U.S. Rituxan for PV also has been granted Orphan Drug designation by the FDA.
Pemphigus vulgaris is the most common type of a group of autoimmune disorders collectively called pemphigus. It is a painful, disfiguring and potentially fatal disease characterized by progressive blistering of the skin and/or the mucous membranes. It affects women and men equally and occurs primarily in adults ages 30 to 60. PV has a mortality rate between 5 and 10% percent, mostly due to complications of therapy.
“Current treatments for pemphigus vulgaris can cause significant, long-term side effects,” said Victoria P. Werth, M.D., 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 Rituxan for this disease and provide patients with a much-needed, FDA-approved treatment option.”