Rilzabrutinib — Investigational Agent
BTK Inhibitor · Phase 3 · Sanofi
Mechanism of Action
Rilzabrutinib is an oral, highly selective, reversible (non-covalent) inhibitor of Bruton tyrosine kinase (BTK). Unlike first-generation BTK inhibitors that bind covalently to the Cys481 residue, rilzabrutinib utilizes a non-covalent binding mechanism that maintains efficacy even in the presence of C481S mutations. It inhibits BTK signaling in both B-cells and mast cells, thereby reducing the production of autoantibodies and pro-inflammatory mediators. This dual action on the adaptive and innate immune systems makes it particularly effective in autoimmune and hematologic conditions without…
Investigational Indications
- Immune Thrombocytopenia (ITP)
- Pemphigus Vulgaris
- Warm Autoimmune Hemolytic Anemia