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.
'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
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
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
About IMBRUVICA
IMBRUVICA (ibrutinib) is a once-daily oral medication that is jointly developed and commercialized by
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
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
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