AbbVie Submits sNDA for IMBRUVICA® (ibrutinib) to Treat Pediatric and Adolescent Patients with Chronic Graft-versus-Host Disease

AbbVie Submits sNDA for Imbruvica (Ibrutinib) to FDA for Pediatric and Adolescent Chronic GVHD PatientsAbbVie today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. FDA for Imbruvica (ibrutinib), a Bruton's tyrosine kinase (BTK) inhibitor, for the treatment of chronic graft-versus-host disease (cGVHD) in children and adolescents aged one year and older who have received one or more lines of systemic therapy. The company also submitted a New Drug Application (NDA) for an oral suspension formulation of Imbruvica, providing an alternative dosing option for pediatric patients. The press release noted that, if approved, this would represent the first pediatric indication for Imbruvica.

cGVHD is a life-threatening complication that occurs after stem cell or bone marrow transplantation. It happens when the donated peripheral blood or bone marrow stem cells recognize the recipient's tissues as foreign and launch an immune attack against them. Nearly half of these transplant patients develop cGVHD, and currently, there are no FDA-approved treatment options in the United States for children under 12. Imbruvica can block the signaling of BTK, which is required for the proliferation and spread of both normal and abnormal B cells. It has been approved in more than 100 countries and regions and has been used to treat over 250,000 patients worldwide.

This application is primarily based on the results of a Phase 1/2 clinical trial that enrolled 59 patients aged 1-19 with relapsed/refractory (R/R) or newly diagnosed moderate to severe cGVHD. The primary endpoints of the study were pharmacokinetics (PK) and safety, with secondary endpoints including overall response rate. The results showed an overall response rate of 78% for Imbruvica, with PK data consistent with adult dosing of Imbruvica. After 20 weeks, sustained response rates of 70% and 58% were observed in treatment-naïve and R/R responders, respectively. The safety profile was consistent with the known characteristics of Imbruvica, and the adverse events (AEs) observed were consistent with those seen in adult patients with moderate to severe cGVHD.

References:

[1] AbbVie Seeks New Indication for IMBRUVICA® (ibrutinib) in Pediatric Patients with Chronic Graft Versus Host Disease (cGVHD). Retrieved February 28, 2022, from https://www.prnewswire.com/news-releases/abbvie-seeks-new-indication-for-imbruvica-ibrutinib-in-pediatric-patients-with-chronic-graft-versus-host-disease-cgvhd-301491494.html

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