When combined with low-dose corticosteroids, a Bruton tyrosine kinase (BTK) inhibitor rilzabrutinib did not result in complete remission (CR) in patients with newly diagnosed or relapsing pemphigus. Long-term use of high-dose corticosteroids for the treatment of moderate to severe pemphigus is associated with adverse events, and targeted treatment, such as rilzabrutinib (a BTK inhibitor), may help minimize exposure to corticosteroids.
Phase 3 trial, conducted from January 2019 to October 2021 in 19 countries, evaluated the efficacy and safety of rilzabrutinib in patients with newly diagnosed or relapsing moderate to severe pemphigus. Overall, 131 patients aged 18-80 years were randomly assigned to receive 400 mg rilzabrutinib (n=65) or placebo (n=66) twice daily for 37 weeks, along with corticosteroids. The corticosteroid dose could be reduced to a minimum of 5 mg/d from week 29 to week 37