Ibrutinib Granted Expanded Indication for Chronic Graft Versus Host Disease

The FDA today approved expanded indications for the cancer drug ibrutinib (Imbruvica) to treat adult patients with chronic graft versus host disease (cGVHD) after failure of 1 or more lines of systemic therapy.

The approval was based on the open-label, single-arm, PCYC-1129-CA clinical trial of 42 patients with cGVHD after failure of first-line corticosteroid therapy and in need of additional therapy. Ibrutinib is the first approved treatment for cGVHD, according to a press release.

At baseline, 88% of patients had at least 2 organs involved, with the most common being mouth, skin, and gastrointestinal tract.

During the study, patients received 420 mg of ibrutinib orally, once daily. The overall response rate was 67%, and the median time-to-response coinciding with the first scheduled response assessment was 12.3 weeks.

Investigators observed responses in all organs involved with cGVHD, according to the release. Responses that lasted 5 months or longer were observed in 48% of patients.

The most common adverse events (AEs) were fatigue, bruising, diarrhea, thrombocytopenia, stomatitis, muscle spasms, nausea, hemorrhage, anemia, and pneumonia. Only 1 patient reported having atrial fibrillation.

Twenty-four percent of patients discontinue treatment due to AEs, with the most common fatigue and pneumonia. Dose reduction due to AEs occurred in 26% of patients.

Ibrutinib was previously approved by the FDA to treat chronic lymphocytic leukemia/small lymphocytic lymphoma, chronic lymphocytic leukemia/small lymphocytic lymphoma with 17p deletion, Waldenström’s macroglobulinemia, marginal zone lymphoma, and mantle cell lymphoma.