Janssen and AbbVie announced that the US Food and Drug Administration (FDA) has approved Imbruvica (ibrutinib) to treat chronic graft-versus-host disease (cGVHD) in children.

The drug is indicated for treating cGVHD in paediatric patients, aged one year and above, after failure of one or more lines of systemic therapy.

Imbruvica is a once-daily oral medication that works by blocking the Bruton’s tyrosine kinase (BTK) protein, which is crucial for B cells, including specific cancer cells to multiply.

The BTK inhibitor was jointly developed by Janssen Biotech, a biotechnology company of Johnson & Johnson (J&J) and AbbVie’s biotech subsidiary Pharmacyclics.

It was previously was approved by the US FDA, to treat adults with cGVHD after failure of one or more lines of systemic therapy, in 2017.

The expanded approval is the first paediatric indication for Imbruvica and offers a new oral suspension formulation for children aged one to 12 years.

Also, Imbruvica becomes the first approved therapy for the younger population, who previously had no approved treatment options for this disease, said Janssen.

Janssen Research & Development haematology and oncology late development, diagnostics and medical affairs global head Craig Tendler 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.”

The FDA approval is based on results from the Phase 1/2 iMAGINE study, an open-label, multi-centre, single-arm study of Imbruvica.

In the trial, Imbruvica showed an overall response rate (ORR) of 60% in patients with median age 13 years with relapsed/refractory (R/R) moderate to severe cGVHD, through week 25.

The drug showed a consistent safety profile with the established profile for Imbruvica.

Also, the observed adverse events (AEs) in paediatric patients are consistent with those observed in adult patients with moderate to severe cGVHD.

Study principal investigator Paul A Carpenter 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.”

Chronic GVHD is a severe complication that occurs after a stem cell or bone marrow transplant, after newly transplanted donor cells attack the recipient’s body.

Its symptoms include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs.