Rare Daily Staff
Roche said its phase 3 PEMPHIX study demonstrated the superiority of MabThera/Rituxan compared to mycophenolate mofetil (MMF) as a treatment for adults with moderate to severe pemphigus vulgaris, a rare autoimmune condition.
Pemphigus vulgaris (PV) is a serious and potentially life-threatening condition characterized by progressive painful blistering of the skin and mucous membranes. It is the most common type of a group of autoimmune disorders collectively called pemphigus. It is estimated that around three in every 100,000 people are diagnosed with this disease globally.
Although MMF is not approved to treat PV, it is a commonly used treatment for the condition that is recommended in published treatment guidelines. MabThera/Rituxan became the first biologic therapy for PV when it was approved by the U.S. Food and Drug Administration in June 2018 and the European Commission in March 2019. The PEMPHIX study provides additional clinical evidence of the effectiveness of MabThera/Rituxan for PV.
The study met the primary endpoint at week 52 and demonstrated that MabThera/Rituxan is superior to MMF, with 40.3 percent of patients treated with MabThera/Rituxan achieving sustained complete remission without the use of steroids for 16 consecutive weeks or more, compared to 9.5 percent in the MMF arm.
All secondary endpoints were statistically significant in favor of MabThera/Rituxan: lower cumulative oral corticosteroid dose, fewer flares, a greater likelihood of sustained complete response, a lesser likelihood of flare, and a greater improvement in the Dermatology Life Quality Index (DLQI) at week 52 compared to the MMF arm.
Adverse events were generally consistent with those seen in previous MabThera/Rituxan clinical studies in PV and other approved autoimmune indications. Results were presented as a late-breaking oral presentation at the 28th Congress of the European Academy of Dermatology and Venereology in Madrid, Spain on 12 October.
“The approval of MabThera/Rituxan for the treatment of pemphigus vulgaris was the first major advancement in the treatment of this rare, serious disease in more than 60 years,” said Levi Garraway, Roche’s chief medical officer and head of global product development. “The PEMPHIX study showed that 40 percent of people in the study could achieve complete remission from painful blistering without the need for corticosteroids for 16 weeks or more and that MabThera/Rituxan may be a superior treatment option to mycophenolate mofetil.”
The study is ongoing, with patients participating in a 48-week safety follow-up period after treatment completion or discontinuation.
Photo: Levi Garraway, Roche’s chief medical officer and head of global product development