FDA approves ibrutinib for pediatric patients with chronic graft-versus-host disease

Ibrutinib (Imbruvica) is the first treatment to gain FDA approval for younger patients who had no previous treatment options for chronic graft-versus-host disease.

The FDA has approved ibrutinib (Imbruvica) for the treatment of pediatric patients 1 year of age and older with chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy. The approval is the first pediatric indication for ibrutinib and the new oral suspension formulation for patients ages 1 to less than 12 years makes ibrutinib the first treatment to gain FDA approval for older patients. young people who had no prior treatment options for cGVHD.

“Imagine having a transplant and being told that you have a moderate to severe chronic disease that can sometimes also be life-threatening,” said study lead researcher Paul A. Carpenter, MD, attending physician at Seattle Children’s Hospital, in a press release. . “If these children were between 1 and 12 years old and did not respond to steroid treatment, we had no rigorously studied treatment options until now. The iMAGINE trial has shown encouraging safety results and sustained response rates in children, and imbruvica’s new oral suspension formulation helps address the challenges children may experience swallowing capsules or tablets.

Ibrutinib is a once-daily oral medication that blocks Bruton’s protein tyrosine kinase (BTK), which is needed for normal and abnormal B cells to multiply and spread. By blocking BTK, ibrutinib can help move abnormal B cells out of their nurturing environment in the lymph nodes, bone marrow, and other organs. Ibrutinib was first approved by the FDA in 2013 and is currently indicated for adult patients in 6 disease areas, including 5 blood cancers. It was approved in 2017 to treat adult patients with cGVHD after failure of one or more lines of systemic treatment.

cGVHD is a life-threatening complication that affects approximately 14,000 patients each year after receiving a stem cell or donor bone marrow transplant. This occurs when donated peripheral blood or bone marrow stem cells perceive the recipient’s body as foreign and the donated cells launch an immune attack on the body. It affects major organs and most commonly the skin, eyes, mouth and liver.

Symptoms of cGVHD include skin rashes, mouth sores, dry eyes, inflammation of the liver, development of scar tissue in the skin and joints, and lung damage. Among children who received an allogeneic transplant, 52% to 65% develop cGVHD. Almost half of these patients develop GVHDc, but there were previously no FDA-approved treatment options for children under 12.

“It is heartbreaking for parents to watch their child struggle with the debilitating effects of cGVHD, especially since there are so few treatment options,” said Susan Stewart, Executive Director of BMT InfoNet, in the press release. “The FDA’s approval of Imbruvica puts another weapon in their arsenal and has the potential to really make a difference for those facing this difficult disease.”

iMAGINE (PCYC-1146-IM) was an open-label, multicenter, single-arm trial of ibrutinib in pediatric and young adult patients aged 1 to

The study recruited 47 patients who needed additional treatment after failing 1 or more prior lines of systemic therapy. Patients 12 years of age and older received ibrutinib 420 mg orally once daily, while patients 1 to less than 12 years of age received ibrutinib 240 mg/m2 orally once daily. day.

Patients with a median age of 13 years (range: 1 to 19 years) (n=47) with moderate to severe relapsed/refractory cGVHD who received ibrutinib had an ORR through week 25 of 60% (95% CI; 44-74). The safety profile of ibrutinib was consistent with the established profile for the drug, with adverse events (AEs) observed in pediatric patients consistent with AEs observed in adults with moderate to severe cGVHD.

“The pediatric GVHDc community is a prime example of an underserved patient population with high unmet medical needs for which Janssen is committed to developing lifesaving therapies,” said Craig Tendler, MD, global head of Late Development, Diagnostics and Medical Affairs, Hematology & Oncology Janssen Research & Development, LLC, in a press release. “cGVHD has life-threatening implications for children, and we are deeply proud of the opportunity to make an impact for these young Imbruvica patients and their families.”


US FDA approves IMBRUVICA® (ibrutinib) as the first and only BTKi treatment for pediatric patients with chronic graft-versus-host disease. Johnson & Johnson. Press release. August 24, 2022. https://www.jnj.com/us-fda-approves-imbruvica-ibrutinib-as-first-and-only-btki-treatment-for-pediatric-patients-with-chronic-graft-versus – host disease