FDA Grants Priority Review for Genentech’s Rituxan for Rare Autoimmune Condition

Rare Daily Staff

The U.S. Food and Drug Administration granted Genentech priority review status for its application to expand approved uses of its rheumatoid arthritis drug Rituxan to include it as a treatment for pemphigus vulgaris, a life-threatening, autoimmune condition with limited treatment options.

Pemphigus vulgaris causes progressive painful blistering of the skin and mucous membranes. The rare, life-threatening condition is the most common type of a group of autoimmune disorders collectively called pemphigus, which affect 30,000 to 40,000 people in the United States. 

The Priority Review designation is granted to drugs that represent significant improvements to the treatment of a disease. Under the designation, the FDA seeks to act on an application within 6 months compared to ten months under standard review. The FDA previously granted Breakthrough Therapy designation and Orphan Drug designation for Rituxan in pemphigus vulgaris.

The company’s submission of a supplemental biologics license application is based on data from a randomized trial conducted in France that evaluated Rituxan plus a tapering regimen of low dose oral corticosteroid treatment compared to a standard dose of corticosteroid alone as a first-line treatment in patients with newly diagnosed moderate to severe pemphigus. 

These results, published in The Lancet in March 2017, show that Rituxan provides substantial improvement in pemphigus vulgaris remission rates and successful tapering and/or cessation of corticosteroid therapy. Results of the study show that Rituxan provides substantial improvement in pemphigus vulgaris remission rates and successful tapering and/or cessation of corticosteroid therapy. Genentech is currently conducting another phase 3 study in pemphigus vulgaris that is evaluating Rituxan plus a tapering regimen of corticosteroid compared to Cellcept, an immunosuppressant.

February 16, 2018