Imbruvica Now Approved for Treatment of Pediatric Patients with Chronic GVHD, Including New Oral Suspension Formulation

October 2022, Vol 13, No 5


On August 24, 2022, the FDA accelerated the approval of ibrutinib (Imbruvica; Pharmacyclics), a Bruton tyrosine kinase inhibitor, for the treatment of chronic graft-versus-host disease (GVHD) in pediatric patients aged ≥1 years who have not responded to ≥1 lines of systemic treatment.

Ibrutinib was previously approved for the treatment of marginal zone lymphoma, Waldenström’s macroglobulinemia, chronic lymphocytic leukemia or small lymphocytic lymphoma with and without 17p deletion, and mantle-cell lymphoma.

On the same day, the FDA also accelerated the approval of a new oral suspension formulation of ibrutinib for this new indication. Ibrutinib was previously available in oral capsule and tablet formulations.

“If these children were between 1 and 12 and didn’t respond to steroid treatment, we didn’t have any rigorously studied treatment options—until now,” said Paul A. Carpenter, MD, MBBS, an iMAGINE study investigator from Fred Hutchinson Cancer Research Center, Seattle Children’s Hospital, WA, in a press release. “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.”

These new indications were approved based on the results of the open-label, multicenter, single-arm iMAGINE trial that enrolled pediatric patients and young adults with moderate or severe chronic GVHD. A total of 47 patients aged 1 to <22 years who required additional treatment after nonresponse to ≥1 systemic therapies were included in the study.

The main efficacy outcome of the study was overall response rate (ORR) through week 25. By week 25, the ORR was 60% (95% confidence interval [CI], 44-74). The median duration of response was 5.3 months (95% CI, 2.8-8.8), and the median time from first response to new systemic therapies for chronic GVHD or death was 14.8 months (95% CI, 4.6-not evaluable).

The most common (≥20%) adverse events were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.

The recommended doses of ibrutinib for chronic GVHD are 240 mg/m2 orally once daily, up to 420 mg, in patients aged 1 to <12 years, and 420 mg orally once daily in patients aged ≥12 years.

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