In March, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to rituximab (Rituxan) for pemphigus vulgaris (PV).
This morning, Genentech, the developer of the drug, announced that its Supplemental Biologics License Application (sBLA) was accepted, and the regulatory authority granted Priority Review for the use of rituximab for the treatment of the rare skin disease.
There are few approved therapeutic options for patients with this condition, which is characterized by progressive painful blistering of the skin and mucous membranes. In PV, antibodies develop and progress against the patient’s epidermal proteins, leading to debilitating blisters which typically present in the mouth first.
According to the International Pemphigus & Pemphigoid Foundation
, PV affects 30,000 to 40,000 people in the United States. Current standard-of-care for PV includes the off-label use of corticosteroids and other immunosuppressive and anti-inflammatory medications, like azathloprine, mycophenolate, cyclophosphamide, and cyclosporine.
“We are committed to developing medicines for rare diseases with limited treatment options, such as pemphigus vulgaris,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development in a press release
. “We look forward to continued work with the FDA to hopefully provide patients with a new treatment for this serious and potentially life-threatening disease.”
Data from a Phase 3 randomized trial
(NCT02383589) conducted in France was the impetus of the sBLA submission. The study evaluated rituximab plus a tapering regimen of low dose oral corticosteroid (CS) treatment in comparison to a standard dose of CS alone as a first-line treatment in patients with newly-diagnosed moderate to severe PV. Approximately 124 participants enrolled in more than 60 centers worldwide, and results of the study exhibited rituximab’s ability to provide significant improvement in PV remission rates and successful tapering and/or cessation of CS therapy.
In the study, it was reported that common side effects of patients administered rituximab included: infusion reactions, chills, infections, body aches, tiredness, and low white blood cell counts. Rituximab can also cause the following severe side effects: tumor lysis syndrome (TLS); heart, kidney, stomach, and serious bowel syndrome problems; serious infections; and low blood cell counts.
Genentech has initiated another Phase 3 study
(NCT02383589) in PV which is evaluating rituximab plus a tapering regimen of CS compared to Cellcept (Pemphix).
For more from the FDA, including applications, designations, and approvals, follow Rare Disease Report