FDA Grants Breakthrough Therapy Designation for Rituxan®in Pemphigus Vulgaris

Genentech, a member of the Roche Group has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to Rituxan^® (rituximab) for pemphigus vulgaris, a rare, serious and life-threatening condition characterized by progressive painful blistering of the skin and mucous membranes. FDA Breakthrough Therapy Designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases and to help ensure that patients receive access to medicines as soon as possible. Genentech is currently enrolling a Phase III study in pemphigus vulgaris (PEMPHIX, NCT02383589), a disease for which there are limited treatment options.

"People with pemphigus vulgaris need more options and we look forward to working with the FDA to make Rituxan available to patients with this potentially deadly disease," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "We are committed to developing therapies that target B cells in areas of unmet need across a range of immune and malignant diseases, including new antibody constructs that enhance efficacy, safety or both."

This is the fifteenth Breakthrough Therapy Designation granted to Genentech medicines since 2013.

Breakthrough Therapy Designation status was granted based on data from a Roche-supported randomized trial conducted in France (Comparison Between Rituximab Treatment and General Corticotherapy Treatment in Patients With Pemphigus), which evaluated Rituxan plus oral corticosteroid (CS) treatment compared to CS as a first-line treatment in patients with moderate to severe pemphigus. Results of the study, published in The Lancet, show that Rituxan may provide substantial improvement in pemphigus vulgaris remission rates and successful tapering and/or cessation of CS therapy.

In 2015, the FDA granted Orphan Drug Designation to Rituxan for the treatment of pemphigus vulgaris.