FDA Approves Ibrutinib for Pediatric Chronic Graft-Versus-Host-Disease

The US Food and Drug Administration has approved ibrutinib (IMBRUVICA) for the treatment of pediatric patients 1 year and older with chronic graft-versus-host-disease (cGVHD) following failure of 1 or more lines of systemic therapy.

The treatment, developed by the Janssen Pharmaceutical Companies of Johnson & Johnson, represents the first pediatric indication of the treatment and the first treatment for younger patients with cGVHD. Ibrutinib also is a new oral suspension formulation for patients aged 1-11 years.

The treatment has a kinase profile, meaning it inhibits both BTK and interleukin-2-inducible T-cell kinase.

The approval is based on a phase 1/2 study called iMAGINE, where the treatment resulted in an overall response rate of 60% through week 25 (95% CI, 44-74) in 47 patients with a median age of 13 years with relapsed/refractory moderate to severe cGVHD.

The safety profile was consistent with established data for ibrutinib. The observed adverse events in this population were similar to what is found in adult populations with moderate to severe cGVHD.

"Imagine going through a transplant and then being told you have a moderate to severe chronic disease that can sometimes also be life-threatening," said Paul A. Carpenter, MD, attending physician at Seattle Children's Hospital and a study principal investigator, in a statement. "If these children were between one and 12 and didn't respond to steroid treatment, we didn't have any rigorously studied treatment options — until now. The iMAGINE trial showed encouraging safety results and sustained response rates in children, and the new IMBRUVICA oral suspension formulation helps address challenges children may have with swallowing capsules or tablets."

The FDA originally approved ibrutinib in 2017 for the treatment of adults with cGVHD after the failure of 1 or more lines of systemic therapy.