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FDA Approves Ibrutinib for Pediatric Patients With Chronic GVHD
On August 24, 2022, the Food and Drug Administration (FDA) granted expedited approval to ibrutinib for pediatric patients 1 year of age or older with chronic graft-versus-host disease (GVHD) after failure of 1 or more lines of systemic therapy. This approval includes 3 different formulations of ibrutinib: capsules, tablets, and a new oral suspension.
The approval was based on efficacy demonstrated by the iMAGINE trial, an open-label, multi-center, single-arm trial of ibrutinib that included 47 pediatric and young adult patients aged 1 year to less than 22 years old with moderate or severe chronic GVHD. Patients were excluded if single organ genitourinary involvement was the only manifestation of chronic GVHD.
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% confidence interval [CI], 44 to 74). The median duration of response was 5.3 months (95% CI, 2.8 to 8.8). The median time from first response to death or new systemic therapies for chronic GVHD was 14.8 months (95% CI, 4.6 to not evaluable).
The trial found that the most common (≥20%) adverse reactions, including laboratory abnormalities, were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.
Source:
FDA approves ibrutinib for pediatric patients with chronic graft versus host disease, including a new oral suspension. Press Release. The US Food and Drug Administration. August 24, 2022. Accessed August 24, 2022. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-ibrutinib-pediatric-patients-chronic-graft-versus-host-disease-including-new-oral
