FDA Approves Ibrutinib for Pediatric Chronic GVHD

The FDA has approved ibrutinib for use in pediatric patients aged one year and older who have chronic graft-versus-host disease (GVHD) after failure of at least one line of systemic therapy.

This is the first pediatric indication for the drug, and the manufacturer has introduced a new oral suspension formulation for patients aged one to 11 years old, officials said. The drug was approved in 2017 for adults with chronic GVHD after failure of at least one line of systemic therapy. It was first approved by the FDA in 2013.

Ibrutinib blocks the Bruton’s tyrosine kinase protein, which is used by normal and abnormal cancerous B cells to spread and multiply. The drug’s mechanism may help inhibit the proliferation of abnormal B cells and remove them from their environment.

The pediatric indication is based on the results from the open-label, multicenter, single-arm, phase I/II iMAGINE study, which resulted in an ORR of 60% in 47 pediatric patients with relapsed/refractory moderate-to-severe chronic GVHD. The safety profile in pediatric patients was consistent with the established profile in adults.

Source: PR Newswire, August 2022