FDA Approves Ibrutinib for Pediatric Patients With Chronic Graft-Versus-Host Disease


Ibrutinib (Imbruvica) is the first therapy to gain FDA approval for younger patients who had no prior 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 1 or more lines of systemic therapy. The approval is the first pediatric indication for ibrutinib and the new oral suspension formulation for patients aged 1 to less than 12 years makes ibrutinib the first therapy to gain FDA approval for younger patients who had no prior treatment options for cGVHD.

“Imagine going through a transplant and then being told you have a moderate to severe chronic disease that can sometimes also be life-threatening,” said study principal investigator Paul A. Carpenter, MD, attending physician at Seattle Children's Hospital, in a press release. “If these children were between 1 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.”

Ibrutinib is a once-daily oral medication that blocks the Bruton’s tyrosine kinase (BTK) protein, which is necessary for normal and abnormal B cells to multiply and spread. By blocking BTK, ibrutinib can help move abnormal B cells out of their nourishing environments 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 hematologic cancers. It was approved in 2017 to treat adult patients with cGVHD after failure of 1 or more lines of systemic therapy.

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

Symptoms of cGVHD 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 administered allogeneic transplants, 52%-65% develop cGVHD. Nearly half of these patients develop cGVHD, but there were previously no FDA-approved treatment options for children under 12 years of age.

“It’s 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 approval of Imbruvica puts another weapon in their arsenal and has the potential to truly make a difference for those who are faced with this challenging disease.”

iMAGINE (PCYC-1146-IM) was an open-label, multi-center, single-arm trial of ibrutinib in pediatric and young adult patients aged 1 to less than 22 years with moderate or severe cGVHD as defined by NIH Consensus Criteria. The trial’s primary endpoints included pharmacokinetics and safety, with secondary endpoints that included overall response rate (ORR), overall survival, and duration of response.

The study enrolled 47 patients needing additional therapy after failing 1 or more prior lines of systemic therapy. Patients 12 years of age and older were administered 420 mg of ibrutinib orally once daily, while patients aged 1 to less than 12 years of age were administered ibrutinib 240 mg/m2 orally once daily.

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

“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,” 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 patients with Imbruvica and their families.”


U.S. FDA Approves IMBRUVICA® (ibrutinib) as First and Only BTKi Treatment for Pediatric Patients with Chronic Graft-Versus-Host Disease. Johnson & Johnson. News release. August 24, 2022. https://www.jnj.com/u-s-fda-approves-imbruvica-ibrutinib-as-first-and-only-btki-treatment-for-pediatric-patients-with-chronic-graft-versus-host-disease

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