The Janssen Pharmaceutical Companies of Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has approved IMBRUVICA® (ibrutinib) for the treatment of pediatric patients one year and older with chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy. This marks the first pediatric indication for IMBRUVICA® and the introduction of a new oral suspension formulation for patients ages one to less than 12. IMBRUVICA® is now the first FDA-approved therapy for these younger patients who previously had no approved treatment options for this life-threatening disease.
Chronic graft-versus-host disease is a life-threatening complication that can occur after a stem cell or bone marrow transplant when newly transplanted donor cells attack the transplant recipient's body. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. Among children who undergo allogeneic transplants, 52-65 percent will develop cGVHD.
The new indication is based on results from the Phase 1/2 iMAGINE study, which showed an overall response rate (ORR) through week 25 of 60 percent (Confidence Interval [CI] 95 percent; 44-74) in patients median age 13 years (range, one to 19 years) (n=47) with relapsed/refractory (R/R) moderate to severe cGVHD. Safety was consistent with the established profile for IMBRUVICA®, with observed adverse events (AEs) in pediatric patients being consistent with those observed in adult patients with moderate to severe cGVHD. IMBRUVICA® was approved to treat adults with cGVHD after failure of one or more lines of systemic therapy in 2017. Because of its unique kinase profile (e.g., inhibiting both BTK and interleukin-2-inducible T-cell kinase [ITK]), IMBRUVICA® has the potential to provide a clinical benefit for cGVHD.