On August 24, 2022, the Food and Drug Administration approved ibrutinib for paediatric patients ≥ 1 year of age with chronic graft versus host disease (cGVHD) after failure of 1 or more lines of systemic therapy. Formulations include capsules, tablets, and oral suspension.

Efficacy was evaluated in iMAGINE (NCT03790332), an open-label, multi-centre, single-arm trial of ibrutinib for paediatric and young adult patients 1 year to less than 22 years old with moderate or severe cGVHD. The trial included 47 patients who required additional therapy after failure of 1 or more lines of systemic therapy. Patients were excluded if single organ genitourinary involvement was the only manifestation of cGVHD.

The median age of patients was 13 years (range, 1 to 19). Selected demographics of the 47 patients were as follows: 70% male, 36% White, 9% Black or African American, 55% other or unreported.

The main efficacy outcome measure was overall response rate (ORR) through Week 25. ORR included complete response or partial responses according to the 2014 NIH Consensus Development Project Response Criteria. ORR by Week 25 was 60% (95% CI: 44, 74). The median duration of response was 5.3 months (95% CI: 2.8, 8.8). The median time from first response to death or new systemic therapies for cGVHD was 14.8 months (95% CI: 4.6, not evaluable).

The most common (≥20%) adverse reactions, including laboratory abnormalities, were anaemia, musculoskeletal pain, pyrexia, diarrhoea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.

The recommended dosage of IMBRUVICA for patients 12 years of age and older with cGVHD is 420 mg orally once daily, and for patients 1 to less than 12 years of age with cGVHD is 240 mg/m2 orally once daily (up to a dose of 420 mg), until cGVHD progression, recurrence of an underlying malignancy, or unacceptable toxicity.

View full prescribing information for ibrutinib here.

Source: FDA