Immune thrombocytopenia (ITP) involves impaired platelet production with complex underlying immune dysregulation. Current therapies offer inconsistent efficacy and unresolved quality of life concerns. Rilzabrutinib, an oral BTK inhibitor recently approved by the FDA, has shown rapid, durable platelet increases in chronic ITP patients. Phase 2 (LUNA2) and phase 3 (LUNA3) trials demonstrated significant platelet response, fatigue reduction, and bleeding improvement, with a well-tolerated safety profile. Early use of rilzabrutinib also enhanced its efficacy. Longer-term studies, and investigations in pediatric patients and other immune-mediated diseases are underway, which position rilzabrutinib as a promising step forward in redefining ITP management and patient care.

References

Kuter DJ, Ghanima W. Immunotherapy. 2025 Aug;17(11):767-782.