FDA approves 'ibrutinib' for chronic graft-versus-host disease in children 1 year and older

On August 24, Janssen, a Johnson & Johnson company, announced that the FDA approved the BTK inhibitor ibrutinib (ibrutinib) for the treatment of patients with relapsed/refractory chronic graft-versus-host disease (cGVHD) in children aged 1 year and older. Ibrutinib was approved by the FDA in August 2017 for adult patients with relapsed/refractory cGVHD and has now been expanded to include pediatric cGVHD patients aged 1 year and older, making it the first and only BTK inhibitor for the treatment of pediatric cGVHD.

Chronic graft-versus-host disease (cGVHD) is a serious complication after stem cell or bone marrow transplantation with symptoms including rash, mouth ulcers, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and lung damage. Between 52% and 65% of children who receive allogeneic transplants develop cGVHD.

BTK is a major regulatory target of B-cell activity, and inhibition of its activity can ameliorate a variety of B-cell-mediated tumors, self-replete diseases, etc. Ibrutinib is the world's first marketed BTK inhibitor that blocks BTK, thereby removing or inhibiting abnormal B-cell proliferation.