The U.S. Food and Drug Administration (FDA) has approved Genentech’s Rituxan (rituximab) for the treatment of adults with moderate to severe pemphigus vulgaris (PV), a rare, serious, potentially life-threatening condition characterized by progressive painful blistering of the skin and mucous membranes.Genentech is a member of the Roche Group.
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. With the latest FDA decision, Rituxan now is approved to treat four autoimmune diseases.
“(Thursday’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,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We are pleased to offer Rituxan as a new and effective therapy to patients with this serious condition.”
The FDA approval is based on data from the Ritux 3 trial, a Roche-supported, randomized, controlled trial conducted in France that used Roche-manufactured, European Union (EU)-approved rituximab product as the clinical trial material.
The study compared the Ritux 3 regimen (EU-approved rituximab product plus short-term corticosteroids [CS]) to CS 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. These results supported the efficacy of Rituxan in treating patients with moderate to severe PV, while tapering off of CS therapy. The results were published in The Lancet in March 2017.
Recently an international panel of experts, the International Bullous Disease Consensus Group, provided new recommendations on the diagnosis and management of pemphigus in the Journal of the American Academy of Dermatology. Based on existing European treatment guidelines, a Delphi survey process was used to help achieve international expert consensus. The consensus includes the recommendation to use an anti-CD20 monoclonal antibody (Rituxan) and corticosteroids as first line therapy options for moderate to severe pemphigus.
Pemphigus vulgaris is an autoimmune blistering disease affecting the skin and mucous membranes. This rare, potentially life-threatening condition accounts for up to 80 percent of cases of pemphigus, a group of autoimmune disorders which affect 30,000 to 40,000 people in the United States.
*Complete remission defined as complete epithelialization and absence of new and/or established lesions.
Source: Genentech. “FDA Approves Genentech’s Rituxan For Pemphigus Vulgaris.” Pharmaceutical Processing, 8 June 2018, www.pharmpro.com/news/2018/06/fda-approves-genentechs-rituxan-pemphigus-vulgaris.