- cGVHD is a life-threatening and rare condition that currently does not have an approved treatment for children under 12 and can occur in patients after receiving peripheral blood or bone marrow stem cell transplantation

- If approved, IMBRUVICA would offer pediatric and adolescent patients the first BTKi treatment option with an oral suspension formulation to help manage their cGVHD after failure of one or more lines of systemic therapy

- The sNDA and NDA submissions were primarily based on results of three years of data from the Phase1/2 iMAGINE clinical trial

- IMBRUVICA was the first treatment approved in the U.S. in 2017 for adult patients with cGVHD after failure of one or more lines of systemic therapy

NORTH CHICAGO, Ill., Feb. 28, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV), today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for IMBRUVICA® (ibrutinib) for the treatment of pediatric and adolescent patients one year and older with chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy. A New Drug Application (NDA) was also submitted for an oral suspension formulation of IMBRUVICA to provide an alternative administration option for pediatric patients. If approved, this represents AbbVie's first pediatric indication for IMBRUVICA.

cGVHD is a life-threatening complication for about 14,000 patients each year after receiving a donor stem cell or bone marrow transplantation.1,2 Nearly half of these transplant patients develop cGVHD, and there are currently no FDA-approved treatment options for children under 12.3 The applications seek to update the IMBRUVICA U.S. Prescribing Information primarily based on the analysis of three years of data from the Phase 1/2 iMAGINE clinical trial, including use of a new oral suspension formulation of the treatment.

"We are committed to this work with IMBRUVICA in the hopes of providing the first FDA-approved BTKi treatment option for younger patients with cGVHD, including a new oral suspension formulation," said James Dean, M.D., Ph.D., IMBRUVICA Global Development Lead and Executive Medical Director at AbbVie. "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."

The iMAGINE clinical trial enrolled 59 patients 1-19 years of age with relapsed/refractory (R/R) or new-onset moderate/severe cGVHD. The primary endpoints of the study were pharmacokinetics (PK) and safety; secondary endpoints included overall response rate. Results showed an overall response rate of 78 percent with IMBRUVICA and that PK data was consistent with adult dosing of IMBRUVICA. After 20 weeks, sustained response rates were observed in 70 percent and 58 percent of treatment-naive and R/R responders, respectively. Safety was consistent with the established profile for IMBRUVICA, with observed adverse events (AEs) consistent with those observed in adult patients with moderate to severe cGVHD. The most common Grade ≥3 AEs (≥ 5% of subjects) overall were pyrexia (8.5%), neutropenia (6.8%), stomatitis (6.8%), hypoxia (6.8%), osteonecrosis (6.8%), alanine aminotransferase increased (5.1%), hypokalemia (5.1%), and pneumothorax (5.1%).

"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 Dr. Paul A. Carpenter, attending physician at Seattle Children's Hospital and study principal investigator. "Results show that PK and safety were consistent with the known profile of IMBRUVICA and that of cGVHD. Efficacy results, including sustained response rates, were also encouraging."

In 2017, IMBRUVICA was first approved as a single-agent therapy for adult patients with cGVHD who have experienced failure of prior systemic therapy, becoming the first FDA-approved treatment for adults with cGVHD. IMBRUVICA could be the first FDA-approved BTKi treatment option for pediatric and adolescent patients with cGVHD.

About cGVHD

cGVHD 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.4 cGVHD impacts major organs, with the skin, eyes, mouth and liver being most common.5 About 35 percent of the estimated 8,000 patients who undergo life-saving allogeneic Hematopoietic Stem Cell Transplant (HSCT) per year develop cGVHD that requires systemic treatment.4 Additionally, cGVHD is the most common cause of morbidity after allogeneic transplant.6 Steroids are the current standard treatment for pediatric cGVHD.7

About the iMAGINE Study

iMAGINE (PCYC-1146-IM) is an interventional single group treatment Phase 1/2 study, which enrolled 59 pediatric patients with chronic graft versus host disease (cGVHD). In the two-part study, Part A patients ages one to