The U.S. Food and Drug Administration approved several formulations of ibrutinib, a Bruton tyrosine kinase inhibitor, for chronic graft-versus-host disease (cGVHD) in pediatric patients 1 year of age and older who have relapsed or were refractory to at least one prior line of therapy.
Approval was granted based on the results of an open-label, multicenter, single-arm trial dubbed iMAGINE. Observed patients were between the ages of 1 and 19 and had moderate or severe cGVHD. The overall response rate by week 25 of the trial was 60%, and median response duration was 5.3 months.
Ibrutinib has several common adverse reactions including pyrexia, diarrhea, abdominal pain, headache, musculoskeletal pain, pneumonia, anemia, thrombocytopenia, and stomatitis. Discontinuation related to adverse events occurred in 24% of adult patients and 23% of pediatric patients.
In addition, ibrutinib is approved to treat mantle cell lymphoma, Waldenström macroglobulinemia, marginal zone lymphoma, chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), and CLL/SLL with 17p deletion.
“The iMAGINE trial showed encouraging safety results and sustained response rates in children, and the new [ibrutinib] oral suspension formulation helps address challenges children may have with swallowing capsules or tablets,” said Paul A. Carpenter, MD, attending physician at Seattle Children’s Hospital and a principal investigator.
Sources: FDA, August 24, 2022; Johnson & Johnson, August 24, 2022.