FDA approves Imbruvica for kids with chronic graft-versus-host disease

The U.S. FDA has approved Janssen's Imbruvica for the treatment of patients aged one and older with chronic graft-versus-host disease (cGVHD) — making it the first and only Bruton's tyrosine kinase (BTK) available for pediatric patients.

Chronic graft-versus-host disease can occur after stem cell or bone marrow transplants when the immune system mistakenly attacks the transplant’s body. According to Janssen, 52-65% of children undergoing allogenic transplants develop cGVHD. 

The new indication approval hinged on results from the iMAGINE phase 1/2 study, which showed promising results and an overall response rate by week 25 of 60%. The study included a total of 47 patients aged 22 and younger, who required additional therapy after failure of one or more prior lines of systemic therapy. 

"cGVHD has life-threatening implications for children, and we are deeply proud of the opportunity to make an impact for these young patients with Imbruvica and their families,” said Craig Tendler, global head of Late Development, Diagnostics & Medical Affairs, Hematology & Oncology Janssen Research & Development, about the recent approval. 

First approved by the FDA in 2013, Imbruvica is a once-daily oral medication jointly developed and commercialized by Janssen and AbbVie. In addition to the new pediatric indication, the drug is approved for adult patients in six disease areas, including five hematologic cancers.