FDA Expands Approval for Ibrutinib To Include Chronic Graft vs Host Disease

The US Food and Drug Administration (FDA) expanded the indications of ibrutinib to include the treatment of adult patients with chronic graft vs host disease (cGVHD) who have failed at least one prior therapy.¹

Ibrutinib, a kinase inhibitor, is under accelerated approval status for mantle cell lymphoma, and is already indicated for chronic lymphocytic leukemia, Waldenström’s macroglobulinemia, and marginal zone lymphoma.

It is the first FDA-approved agent for cGVHD.

The FDA based its approval on evidence from the PCYC-1129 phase 1b/2 trial (ClinicalTrials.gov Identifier: NCT02195869). Forty-two patients with cGVHD who had failed prior therapies received ibrutinib 420mg once daily. Patients continued any ongoing therapies, which included corticosteroids and other immunosuppressants.

The overall response rate (ORR) was 67%, and 48% of patients had a sustained response of 5 months or longer.

Serious adverse effects (AEs) for ibrutinib include hemorrhage, infections, atrial fibrillation, cytopenias, second primary malignancies, hypertension, and tumor lysis syndrome.

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

Reference

1. FDA approves treatment for chronic graft versus host disease [news release]. Silver Spring, MD: US Food and Drug Administration; August 2, 2017. https://www.fda.gov/NewsEvents/Newsroom/ PressAnnouncements/ucm569710.htm? source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed August 2, 2017.