NORTH CHICAGO, Ill. - AbbVie (NYSE: ABBV) announced that the U.S. Food and Drug Administration (FDA) approved the use of IMBRUVICA (ibrutinib) for the treatment of pediatric patients one year and older with chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy.

The approval is AbbVie's first pediatric indication for IMBRUVICA, marking the 12th FDA approval for IMBRUVICA and the first Bruton's tyrosine kinase inhibitor (BTKi) treatment approved for a pediatric patient population. This approval also marks the first approved treatment option for children under 12 years of age suffering from cGVHD. The approval is primarily based on positive results from the iMAGINE Phase 1/2 clinical trial.

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.1 cGVHD impacts major organs, including the skin, eyes, mouth and liver as the most commonly affected.2 About 35% of the estimated 8,000 patients who undergo life-saving allogeneic hematopoietic stem cell transplant (HSCT) per year develop cGVHD that requires systemic treatment.1 Additionally, cGVHD is the most common cause of morbidity after an allogeneic transplant.3 Steroids are the current standard treatment for pediatric cGVHD.4

'For a substantial fraction of children who develop moderate or severe chronic graft versus host disease after blood or marrow transplantation, treatment options supported by methodical, formal study in children are limited,' said Dr. Paul A. Carpenter, attending physician at Seattle Children's Hospital and a study principal investigator. 'Now, healthcare professionals have another effective treatment option for both pediatric and adult patients living with this disease after failure of one or more lines of systemic therapy. Additionally, having an oral suspension formulation designed for children is a helpful alternative.'

'This approval is incredibly meaningful as AbbVie's first pediatric approval for IMBRUVICA as well as within our company's oncology portfolio. As a Pediatric Oncologist, when my patients describe the physical pain they experience from simply hugging their parents due to their cGVHD, the importance of researching alternative treatment options in this patient population is further validated,' said Gauri Sunkersett, D.O., associate medical director at AbbVie. 'At AbbVie, we are committed to driving oncology research and developing therapies that have the potential to help patients of all ages, including underserved populations like children diagnosed with cGVHD.'

The iMAGINE study demonstrated an Overall Response Rate (ORR) through week 25 of 60% (Confidence Interval [CI] 95%; 44-74) in patients median age 13 years (range, 1-19 years) (n=47) with relapsed/refractory (R/R) moderate to severe cGVHD.5 The median duration of response was 5.3 months (95% CI: 2.8, 8.8).5 Safety was consistent with the established profile for IMBRUVICA, with observed adverse reactions (ARs) consistent with those observed in adult patients with moderate to severe cGVHD.5 The most common ARs (occurring in 20% or more of patients), including laboratory abnormalities, were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.5

Since 2017, IMBRUVICA has been 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 cGVHD.

'In some ways, children struggling with cGVHD have been an 'orphan population' in relation to having treatment options available. Although there has been progress in approved treatment options for adults with cGVHD, the safety and efficacy of these have not been well studied in children,' said Susan Stewart, executive director of BMT InfoNet, a non-profit advocacy organization for bone marrow, stem cell and cord blood transplant patients. 'This FDA approval of IMBRUVICA gives children and their families struggling with this very difficult disease newfound hope.'

The recommended dose of IMBRUVICA for cGVHD patients 12 years and older is 420 mg taken orally once daily until disease progression or unacceptable toxicity.6 In cGVHD patients one year old to less than 12 years of age, the recommended dose of IMBRUVICA is 240 mg/m2 taken orally once daily (up to a dose of 420 mg) until disease progression or unacceptable toxicity.6 This FDA approval also includes an oral suspension formulation of IMBRUVICA to provide an alternative and child-friendly administration option.

About the iMAGINE Study5

iMAGINE (PCYC-1146-IM) is an open-label, multi-center, single-arm trial of IMBRUVICA for the treatment of pediatric and young adult patients aged one year to less than 22 years with moderate or severe cGVHD as defined by NIH Consensus Criteria. The study included 47 patients who required additional therapy after failure of one or more prior lines of systemic therapy. Patients aged 12 years and older were treated with IMBRUVICA 420 mg orally once daily, and patients aged one year to less than 12 years were treated with IMBRUVICA 240 mg/m2 orally once daily. Primary endpoints included pharmacokinetics (PK) and safety, and secondary endpoints included overall response rate (ORR; CR/PR) per 2014 NIH criteria, overall survival, and duration of response (DOR).

About IMBRUVICA

IMBRUVICA (ibrutinib) is a once-daily oral medication that is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company. IMBRUVICA blocks the Bruton's tyrosine kinase (BTK) protein, which is needed by normal and abnormal B cells, including specific cancer cells, to multiply and spread. By blocking BTK, IMBRUVICA may help move abnormal B cells out of their nourishing environments and inhibits their proliferation.7,8,9

IMBRUVICA is approved in more than 100 countries and has been used to treat more than 250,000 patients worldwide. There are more than 50 company-sponsored clinical trials, including 18 Phase 3 studies, over 11 years evaluating the efficacy and safety of IMBRUVICA.

IMBRUVICA was first approved by the U.S. Food and Drug Administration (FDA) in November 2013, and today is indicated for adult patients in six disease areas, including five hematologic cancers. These include indications to treat adults with CLL/SLL with or without 17p deletion (del17p), adults with Waldenstrom's macroglobulinemia (WM), adults with previously treated mantle cell lymphoma (MCL)*, adult patients with previously treated marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy*, as well as adult and pediatric patients with previously treated chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy.6

Accelerated approval was granted for MCL and MZL based on overall response rate. Continued approval for MCL and MZL may be contingent upon verification and description of clinical benefit in confirmatory trials.

Since 2019, the National Comprehensive Cancer Network (NCCN), recommends ibrutinib (IMBRUVICA) as a preferred regimen for the initial treatment of CLL/SLL and has Category 1 treatment status for treatment-naive patients without deletion 17p/TP53 mutation and as a preferred treatment for treatment-naive patients with deletion 17p/TP53 mutation. The NCCN Guidelines also recommend IMBRUVICA, with or without rituximab, as a preferred regimen for the treatment of relapsed/refractory MCL, as a Category 1 preferred regimen for both untreated and previously treated WM patients, and as a preferred regimen for relapsed/refractory MZL.10

About AbbVie in Oncology

At AbbVie, we are committed to transforming standards of care for multiple blood cancers while advancing a dynamic pipeline of investigational therapies across a range of cancer types. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potentially breakthrough medicines. We are evaluating more than 20 investigational medicines in over 300 clinical trials across some of the world's most widespread and debilitating cancers. As we work to have a remarkable impact on people's lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit http://www.abbvie.com/oncology.

About AbbVie

AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.

Forward-Looking Statements

Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words 'believe,' 'expect,' 'anticipate,' 'project' and similar expressions, among others, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements. Such risks and uncertainties include, but are not limited to, failure to realize the expected benefits from AbbVie's acquisition of Allergan plc ('Allergan'), failure to promptly and effectively integrate Allergan's businesses, competition from other products, challenges to intellectual property, difficulties inherent in the research and development process, adverse litigation or government action, changes to laws and regulations applicable to our industry and the impact of public health outbreaks, epidemics or pandemics, such as COVID-19. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, 'Risk Factors,' of AbbVie's 2021 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no obligation to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law.

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