FDA Approves Genentech’s Rituxan for Rare Autoimmune Condition

Rare Daily Staff

The U.S. Food and Drug Administration approved Genentech’s Rituxan for adults with moderate to severe pemphigus vulgaris, a rare, serious, potentially life-threatening, autoimmune condition characterized by progressive painful blistering of the skin and mucous membranes.

Rituxan is the first biologic therapy approved by the FDA for PV and the first major advancement in the treatment of the disease in more than 60 years. The FDA previously granted Priority Review, Breakthrough Therapy Designation and Orphan Drug Designation to Rituxan for the treatment of PV. It is the fourth autoimmune disease Rituxan is now approved to treat.

The FDA approval is based on data from the Ritux 3 trial, a Roche-supported, randomized, controlled trial conducted in France. The study compared the Ritux 3 regimen or rituximab plus short-term corticosteroids to corticosteroids alone as a first-line treatment in patients with newly diagnosed moderate to severe pemphigus.

The primary endpoint of the study was complete remission at month 24 without the use of steroids for two or more months. Results of the study showed that 90 percent of PV patients treated with the Ritux 3 regimen met the endpoint, compared to 28 percent of PV patients treated with CS alone. The results were published in The Lancet in March 2017.

June 8, 2018