On Thursday, June 7th, the FDA approved Rituxan for the treatment of adults with moderate to severe pemphigus vulgaris (PV).
Rituxan is the first biologic therapy approved by the FDA for PV and the first major advancement in the treatment of PV 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 this decision, Rituxan is now approved to treat four autoimmune diseases.
“It is our hope that this announcement will open the door to approval for other indications in our diseases and usher in a renewed focus on available treatments,” said Marc Yale, Executive Director of the International Pemphigus & Pemphigoid Foundation.
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. (Complete remission defined as complete epithelialization and absence of new and/or established lesions.)
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. These results were published in The Lancet in March 2017.
An international panel of experts called the International Bullous Disease Consensus Group recently 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.
The Role of the IPPF
The IPPF aims to serve as a primary source of information for you regarding this approved treatment and is available to help answer your questions in the upcoming months. If you are considering Rituxan as a potential therapy, please consult your healthcare provider. Inform them of your medical history, and ask about the potential side effects.
The IPPF’s Peer Health Coaches (PHC) are pemphigus and pemphigoid patients who help more than 1,200 patients and caregivers each year. These specially trained PHCs reduce patient anxiety and uncertainty while providing unbiased disease and treatment knowledge. You can find our PHCs engaging the community through social media, emails, phone calls, and in-person support. The goal of our PHC program is to ensure we help every person who needs assistance in the shortest amount of time possible.
Genentech Access Solutions
Genentech is the drug company that produces Rituxan (rituximab). Genentech Access Solutions is a resource for people considering Rituxan as a treatment option. It may be worth contacting Access Solutions directly regardless of whether or not you have health insurance.
Access Solutions may be able to help by:
- Checking your insurance coverage and costs
- Helping you find ways to pay for your medicine
- Working to get your medicine to you