A treatment for chronic graft versus host disease in children aged one year and older has been approved by the U.S. Food and Drug Administration (FDA).
Graft versus host disease is a common complication after receiving a donor stem-cell or bone marrow transplantation. Sometimes, the graft doesn’t recognize the host as being friendly and sees the body as a threat, the chronic type typically occurs later after transplant.
The Janssen Pharmaceutical Companies of Johnson & Johnson made the announcement today (August 25) that IMBRUVICA (ibrutinib) for the treatment of pediatric patients after failure of one or more lines of systemic therapy had been given the go-ahead.
This milestone marks the first pediatric indication for IMBRUVICA and the introduction of a new oral suspension formulation for patients ages one to under-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.
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% will develop cGVHD.
The iMAGINE study is an open-label, multi-center, single-arm trial of IMBRUVICA for the treatment of pediatric and young adult patients aged one year to less than 22 years with moderate or severe cGVHD.
The study included 47 patients who required additional therapy after failure of one or more prior lines of systemic therapy. Patients aged 12 years and older were treated with IMBRUVICA 420 mg orally once daily, and patients aged one year to less than 12 years were treated with IMBRUVICA 240 mg/m2 orally once daily.
Paul A. Carpenter is an attending physician at Seattle Children’s Hospital and a study principal investigator.
He said: “Imagine going through a transplant and then being told you have a moderate to severe chronic disease that can sometimes also be life-threatening.
“If these children were between one and 12 and didn’t respond to steroid treatment, we didn’t have any rigorously studied treatment options — until now. The iMAGINE trial showed encouraging safety results and sustained response rates in children, and the new IMBRUVICA oral suspension formulation helps address challenges children may have with swallowing capsules or tablets.”
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 such as inhibiting both BTK and interleukin-2-inducible T-cell kinase [ITK]), IMBRUVICA has the potential to provide a clinical benefit for cGVHD.
Craig Tendler, of Janssen research and development, said: “The pediatric cGVHD community is a prime example of an underserved patient population with high unmet medical needs for whom Janssen is committed to developing life-saving therapies.
“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.”
IMBRUVICA is approved in more than 100 countries and has been used to treat more than 250,000 patients worldwide.