FDA grants breakthrough therapy designation to Rituxan for pemphigus vulgaris

The FDA has granted breakthrough therapy designation status to Rituxan for treating pemphigus vulgaris, Genentech has announced.

Pemphigus vulgaris is a rare autoimmune, intraepidermal blistering disease of the skin and mucous membranes that can be life-threatening, and is estimated that three in every 100,000 people are diagnosed with the disease, according to a press release from Genentech.

The status was granted based on data from a Roche-sponsored randomized trial conducted in France which measured results of Rituxian (rituximab) plus oral corticosteroid treatment compared to corticosteroid as first-line treatment for patients with moderate-to-severe pemphigus vulgaris.

Results published in The Lancet showed that rituximab “may provide substantial improvement in pemphigus vulgraris remission rates and successful tapering and/or sensation of [corticosteroid] therapy,” according to the release.

“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,” Sandra Horning, MD, chief medical officer and head of global product development, Genentech, stated in the release. “We are committed to developing therapies that target B cells in areas of unmet need across a range of immune and malignant disease, including new antibody constructs that enhance efficacy, safety or both.”

Breakthrough therapy designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases to help ensure that patients receive access to medicines as soon as possible, according to the release.

Genentech also announced it is enrolling patents in a phase 3 study of patients with moderate-to-server pemphigus vulgaris requiring 60-120 mg per day of oral prednisone or the equivalent and will compare treatment of rituximab with mycophenolate mofetil.

Reference: www.genentech.com