The U.S. Food and Drug Administration approved ibrutinib, a Bruton tyrosine kinase inhibitor, for secondline treatment of adults with chronic graft-versus-host disease (cGVHD), making it the first FDA-approved therapy for cGVHD.
The approval was based on results from a single-arm, open-label, multicenter trial of 42 patients with cGVHD whose symptoms (such as mouth ulcers and skin rashes) persisted despite standard treatment with corticosteroids. A majority of patients (88%) had two or more organs affected by cGVHD. After treatment with ibrutinib 420 mg once-daily, 67 percent of patients (n=28; 95% CI 51-80) experienced an improvement in their cGVHD symptoms. The median time to response was 12.3 weeks (range = 4.1-42.1 weeks), and responses were seen in all involved organs. Almost half of patients (48%; n=20) had responses that lasted five months or longer.
The most common AEs associated with ibrutinib included fatigue, bruising, diarrhea, thrombocytopenia, muscle spasms, stomatitis, nausea, severe bleeding, anemia, and pneumonia. Serious AEs included hemorrhage, infections, cytopenias, atrial fibrillation, hypertension, second primary malignancies, and tumor lysis syndrome.
The FDA previously granted ibrutinib priority review, breakthrough-therapy designation, and orphan-drug designation. Ibrutinib is already approved for the treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma with or without 17p deletion, Waldenström macroglobulinemia, marginal zone lymphoma, and mantle cell lymphoma.
Source: U.S. Food and Drug Administration news release, August 2, 2017.