The US Food and Drug Administration (FDA) will review a supplemental New Drug Application (sNDA) for ibrutinib as a treatment of chronic graft-versus-host-disease (cGVHD) after the failure of steroid therapy, according to a press release.1

cGHVD is a potentially fatal condition that can arise after allogeneic stem cell transplant. It occurs in about 30% to 70% of patients, and is usually treated with a corticosteroid. There has, however, been a lack of FDA-approved alternatives if steroid-therapy fails.

Ibrutinib, a Bruton’s kinase inhibitor, was evaluated as a second-line therapy for patients with cGVHD in a phase 1b/2 trial ( Identifier: NCT02195869), from which data were presented at the 2016 American Society of Hematology Annual Meeting.2

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The overall response rate in this study was 67%, with a complete response rate of 21%.

In an ongoing, placebo-controlled, phase 3 trial ( Identifier: NCT02959944), researchers are evaluating the efficacy and safety of ibrutinib and prednisone as a combination first-line therapy.

RELATED: Ibrutinib for the Relief of GVHD-related Symptoms

Common adverse events among patients with B cell malignancies treated with ibrutinib include “neutropenia, thrombocytopenia, diarrhea, anemia, musculoskeletal pain, rash, nausea, bruising, fatigue, hemorrhage, and pyrexia.”


  1. U.S. FDA accepts for review the application of ibrutinib (IMBRUVICA®) for chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy [news release]. Raritan, NJ: Janssen; April 4, 2017. Accessed April 4, 2017.
  2. Miklos D, Cutler CS, Arora M, et al. Multicenter open-label phase 2 study of ibrutinib in chronic graft versus host disease (cGVHD) after failure of corticosteroids. Paper presented at: American Society of Hematology 58th Annual Meeting and Exposition; December 3-6, 2016; San Diego, CA.