On August 24, 2022, the FDA approved Imbruvica (ibrutinib; from Pharmacyclics), a Bruton tyrosine kinase inhibitor, for the treatment of chronic graft-versus-host disease (GVHD) in pediatric patients aged 1 year or older whose disease did not respond to 1 or more systemic therapies.
On the same day, the FDA approved a new oral suspension formulation of Imbruvica for use in young patients. Imbruvica was previously approved for adults with chronic GVHD, and for several types of blood cancer.
This new approval was based on results of the multicenter clinical trial that included 47 pediatric patients and young adults aged 1 year to under 22 years with moderate or severe chronic GVHD who required additional treatment after systemic therapy.
In the study, after 25 weeks of Imbruvica therapy, more than half (60%) of the patients responded to the treatment. The average duration of response was 5.3 months, and the average time from the first response to starting a different therapy for chronic GVHD, or to death, was 14.8 months.
“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, of Seattle Children’s Hospital, and a study investigator. “The new Imbruvica oral suspension formulation helps address challenges children may have with swallowing capsules or tablets,” Dr. Carpenter added.
The most common side effects reported in the patients receiving Imbruvica in this study included anemia, musculoskeletal pain, fever, diarrhea, pneumonia, abdominal pain, oral ulcers, and headache.
