FDA approves Imbruvica for children with chronic graft-versus-host disease
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The FDA approved ibrutinib for children with chronic graft-versus-host disease.
The designation applies to use of the agent by children aged 1 year or older who failed at least one prior line of systemic therapy.
Formulations include tablets, capsules or a new oral suspension.
Ibrutinib (Imbruvica; Pharmacyclics, Janssen) — a Bruton tyrosine kinase inhibitor — already had been approved in the United States for adults with chronic GVHD after failure of prior systemic therapy.
The open-label, single-arm iMAGINE study assessed the agent for 47 children or young adults (median age, 13 years; range, 1-19; 70% male; 36% white, 9% Black or African American; 55% other or unreported) with moderate or severe chronic GVHD who required additional therapy after failure of at least one line of systemic therapy.
Overall response rate through week 25 served as the primary efficacy outcome.
Researchers reported an ORR of 60% (95% CI, 44-74) at week 25, with median response duration of 5.3 months (95% CI, 2.8-8.8) and median time from first response to death or new systemic therapy for chronic GVHD of 14.8 months (95% CI, 4.6-not evaluable).
“Imagine going through a transplant and then being told you have a moderate to severe chronic disease that can sometimes also be life-threatening," Paul A. Carpenter, MD, physician at Seattle Children's Hospital and an iMAGINE study investigator, said in a press release. "If these children were between 1and 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.”
Adverse events that occurred among at least 20% of iMAGINE study participants included anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia and headache.
The FDA previously granted priority review to ibrutinib for this indication.
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