The Food and Drug Administration (FDA) has expanded the approval of Imbruvica (ibrutinib) to include pediatric patients 1 year of age and older with chronic graft-vs-host disease (cGVHD) after failure of 1 or more lines of systemic therapy.

A new oral suspension formulation of Imbruvica has also been approved for pediatric patients. The oral suspension is supplied as 108 mL in a 150 mL amber glass bottle; each mL contains 70 mg of ibrutinib. The recommended dose of Imbruvica is based on body surface area for patients 1 to less than 12 years of age using either Imbruvica capsules/tablets or oral suspension.

The expanded approval was based on data from the open-label, multicenter, single-arm phase 1/2 iMAGINE trial ( Identifier: NCT03790332), which included 47 patients 1 year to less than 22 years of age with moderate to severe cGVHD after failure of 1 or more lines of systemic therapy. Patients were excluded if single organ genitourinary involvement was the only manifestation of cGVHD.

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Study participants 1 to less than 12 years of age received Imbruvica at 240 mg/m2 orally once daily, while those 12 years and older were treated with 420 mg orally once daily. Supportive care therapies were permitted during the study, though initiation of new systemic cGVHD therapy was not.

Results showed an overall response rate (main efficacy outcome measure) of 60% (n=28; 95% CI, 44-74) through week 25. The complete response rate was 4% (n=2), and the partial response rate was 55% (n=26). The median duration of response was 5.3 months (95% CI, 2.8-8.8). The median time to first response was 0.9 month (range, 0.9-6.1 months), and the median time from first response to death or new systemic therapies for cGVHD was 14.8 months (95% CI, 4.6-not evaluable).

Additionally, 50% of patients 12 years of age and older showed at least a 7-point decrease in patient-reported symptom bother through week 25, as assessed by the Lee Symptom Scale overall summary score.

The most common adverse reactions reported were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.

“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,” said Dr Paul A. Carpenter, attending physician at Seattle Children’s Hospital and a study principal investigator.

Imbruvica, a Bruton tyrosine kinase inhibitor, is also indicated for mantle cell lymphoma, chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), CLL/SLL with 17p deletion, Waldenström’s macroglobulinemia, and marginal zone lymphoma.


  1. FDA approves ibrutinib for pediatric patients with chronic graft versus host disease, including a new oral suspension. News release. US Food and Drug Administration. August 24, 2022.
  2. US FDA approves Imbruvica® (ibrutinib) as first and only BTKi treatment for pediatric patients with chronic graft-versus-host disease. News release. August 24, 2022.
  3. Imbruvica. Package insert. Pharmacyclics; 2022. Accessed August 24, 2022.

This article originally appeared on MPR