FDA Approves Ibrutinib as First Therapy for Chronic-Graft-Versus Host Disease

The U.S. Food and Drug Administration (FDA) approved ibrutinib – a Bruton tyrosine kinase inhibitor that treats mantle cell lymphoma, chronic lymphocytic leukemia, and other hematologic conditions – for secondline treatment of adult patients with chronic graft-versus-host disease (cGVHD). This is the first FDA-approved therapy for the treatment of cGVHD.

Ibrutinib was approved for this indication based on results from a single-arm trial of 42 patients with cGVHD whose symptoms (such as mouth ulcers and skin rashes) persisted despite standard treatment with corticosteroids. More than half of patients had two or more organs affected by cGVHD. In the trial, 67 percent of patients experienced an improvement in their cGVHD symptoms after treatment with ibrutinib. The improvements lasted for at least five months in 48 percent of patients.

The most common adverse events were fatigue, bruising, diarrhea, thrombocytopenia, muscle spasms, stomatitis, nausea, severe bleeding, anemia, and pneumonia. Serious AEs were hemorrhage, infections, cytopenias, atrial fibrillation, hypertension, second primary malignancies, and tumor lysis syndrome.

Ibrutinib previously received priority-review, breakthrough-therapy, and orphan-drug designations from the FDA.

Source: FDA press release, August 2, 2017.