AbbVie Applies to FDA for New Ibrutinib Indication in Pediatric Patients With Chronic Graft Versus Host Disease


A supplemental application was also submitted for an oral suspension formulation to provide an alternative administration option for children.

AbbVie has submitted a supplemental New Drug Application (sNDA) to the FDA for a new indication for ibrutinib (Imbruvica; AbbVie) in the treatment of pediatric and adolescent patients 1 year of age and older with chronic graft versus host disease (cGVHD) after failure of 1 or more lines of systemic therapy. An sNDA was also submitted for an oral suspension formulation to provide an alternative administration option for children.

“For young children, the availability of a liquid oral suspension versus an oral capsule or tablet can be significant to enable them to take the recommended dose and address challenges swallowing capsules or tablets,” said James Dean, MD, PhD, Imbruvica global development lead and executive medical director at AbbVie, in a press release.

cGVHD is a life-threatening complication that affects approximately 14,000 patients each year after receiving a donor stem cell or bone marrow transplantation, according to the press release. Nearly half of these transplant patients develop cGVHD, but there are currently no FDA-approved treatment options for children under 12 years of age. To address this gap, the new supplemental applications seek to update ibrutinib prescribing information based on an analysis of 3 years of data from the phase 1/2 iMAGINE clinical trial.

According to the press release, cGVHD is caused 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.

The trial enrolled 59 patients between 1 and 19 years of age with relapsed/refractory (R/R) or new-onset moderate to severe cGVHD. Pharmacokinetics and safety were both primary endpoints, and secondary endpoints included overall response rate.

According to the study results, participants had an overall response rate of 78% with ibrutinib and pharmacokinetics data were consistent with adult dosing. After 20 weeks, sustained response rates were observed in 70% and 58% of treatment-naïve and R/R responders, respectively.

Furthermore, safety was consistent with the established profile for ibrutinib and observed adverse events (AEs) were consistent with those observed in adults with moderate to severe cGVHD. The most common grade 3 or higher AEs overall were pyrexia (8.5%), neutropenia (6.8%), stomatitis (6.8%), hypoxia (6.8%), osteonecrosis (6.8%), increased alanine aminotransferase (5.1%), hypokalemia (5.1%), and pneumothorax (5.1%).

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. It was first approved by the FDA in 2013 and is currently indicated for adult patients in 6 disease areas, including 5 hematologic cancers, according to the press release.

“It is important to empower patients and their families with evidence-based knowledge and I am encouraged by the results from the iMAGINE clinical trial of Imbruvica,” said Paul A. Carpenter, MD, study principal investigator, in the press release.


AbbVie Seeks New Indication for IMBRUVICA (ibrutinib) in Pediatric Patients with Chronic Graft Versus Host Disease (cGVHD). News release. AbbVie; February 28, 2022. Accessed February 28, 2022.

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