FDA Approves Rituximab for Pemphigus Vulgaris


The rare condition affects 30,000-40,000 US patients annually. Rituximab becomes its first indicated biologic treatment.

fda, pemphis vulgaris, rituximab, genentech, roche

The US Food and Drug Administration (FDA) has approved rituximab (Rituximab) for the treatment of adults with a rare, life-threatening autoimmune disease.

The therapy has been approved to treat pemphigus vulgaris (PV), a condition characterized by progressive blistering of the skin and mucous membranes. With the approval, rituximab has become the first biologic therapy approved by the FDA for PV, and the first major advancement in treatment of the condition in 6 decades, according to Genentech.

Along with PV, rituximab is approved for the treatment of autoimmune diseases rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyganiitis. It was previously granted Priority Review, Breakthrough Therapy Designation, and Orphan Drug Designation by the FDA for its indication for adults with PV.

Its approval was based on data from the Ritux 3 trial, a randomized, controlled study conducted in France that tested the European Union (EU)-approved rituximab product in adult patients. Researchers compared rituximab plus short-term corticosteroids (CS) to CS monotherapy as a first-line treatment for patients recently diagnosed with moderate to severe PV.

Researchers tested for a primary endpoint of complete remission at 24 month without the use of steroids for 2 months or more. Study results showed that 90% of PV patients treated with the combination regimen reached endpoint, versus just 28% of PV patients treated with CS alone.

Sandra Horning, MD, chief medical officer and head of Genentech Global Product Development, expressed pride in revitalizing a longtime stagnant market for treatment of the rare disease.

“Today’s decision by the FDA provides the first approved treatment option in more than 60 years for patients with pemphigus vulgaris, a potentially life-threatening disease,” Horning said. “We are pleased to offer Rituxan as a new and effective therapy to patients with this serious condition.”

PV reportedly affects about 30,000-40,000 Americans each year, according to the International Pemphigus & Pemphigoid Foundation. Rituximab's benefit for pediatric patients has not been proven.

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