Rilzabrutinib Pemphigus Vulgaris. It was well-tolerated by patients and improved patients’
It was well-tolerated by patients and improved patients’ quality of life. The study focused on the use of rilzabrutinib which is an oral, reversible, covalent Bruton tyrosine kinase (BTK) inhibitor to manage pemphigus. Despite the well-defined pathophysiology and unmet need for safe and effective therapies for pemphigus, Aug 1, 2024 · An open-label study of oral rilzabrutinib monotherapy in 4 dogs with pemphigus foliaceus demonstrated 77–100% improvement in disease activity (Goodale et al, 2020b), providing strong rationale to pursue clinical trials of rilzabrutinib in human pemphigus. The phase 2 pemphigus study of oral, reversible, covalent Bruton tyrosine kinase inhibitor rilzabrutinib demonstrated rapid and sustained efficacy with well-tolerated safety. Pemphigus vulgaris (PV) is a B-cell-mediated autoimmune blistering disease characterized by autoantibodies targeting skin cell adhesion proteins. A recent phase 2 clinical trial found positive outcomes in patients with pemphigus a rare and potentially life-threatening autoimmune disease. Oct 4, 2021 · We found that treatment with rilzabrutinib (which can be taken alone or together with low doses of corticosteroids) showed rapid and sustained improvement in pemphigus disease activity measures. Aug 1, 2024 · The phase 2 pemphigus study of oral, reversible, covalent Bruton tyrosine kinase inhibitor rilzabrutinib demonstrated rapid and sustained efficacy with well-tolerated safety. Short abstract What is already known about this topic? Oct 1, 2021 · In conclusion, rilzabrutinib alone, or with much lower corticosteroid doses than usual, was safe and effective. Rilzabrutinib alone, or with much lower CS doses than usual, was safe, with rapid clinical activity in pemphigus vulgaris. These data suggest that BTK inhibition may be a promising treatment strategy and support further investigation of rilzabrutinib for the treatment of pemphigus. This content is only available as a PDF. These findings suggest that BTK inhibition is a promising treatment option and therefore, further investigation of rilzabrutinib for the treatment of pemphigus is needed. Sep 9, 2005 · Rilzabrutinib was granted FDA Fast Track Designation for ITP in November 2020 and for pemphigus vulgaris in May 2021. Conclusions Rilzabrutinib alone, or with much lower CS doses than usual, was safe, with rapid clinical activity in pemphigus vulgaris. Rilzabrutinib is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority. Jul 17, 2025 · This is a parallel-group, Phase 3, double-blind, 2-arm study to investigate the efficacy, safety, PK and PD of oral rilzabrutinib in achieving durable Hb response (DHR) compared with placebo in approximately 90 male and female participants ≥ 18 years of age with a confirmed diagnosis of primary wAIH We would like to show you a description here but the site won’t allow us. We would like to show you a description here but the site won’t allow us. . Apr 3, 2024 · Rilzabrutinib did not meet primary or secondary endpoints in the phase 3 PEGASUS trial of patients with pemphigus vulgaris or foliaceus.