The Janssen Pharmaceutical Companies – US FDA Approves IMBRUVICA (ibrutinib) as First and Only BTKi Treatment for Pediatric Patients With Chronic Graft-vs-Host Disease

HORSHAM, Pennsylvania – The Janssen Pharmaceutical Companies of Johnson & Johnson announced that the US Food and Drug Administration (FDA) has approved IMBRUVICA (ibrutinib) for the treatment of pediatric patients 1 year of age and older with chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy. This milestone marks the first pediatric indication for IMBRUVICA and the introduction of a new oral suspension formulation for patients aged one to less than 12 years. IMBRUVICA is now the first FDA-approved treatment for these younger patients who previously had no approved treatment options for this lifetime. – threatening disease.

Chronic graft versus host disease is a life-threatening complication that can occur after a stem cell or bone marrow transplant when newly transplanted donor cells attack the recipient’s body.[1] Symptoms may include skin rashes, mouth sores, dry eyes, liver inflammation, development of scar tissue on the skin and joints, and lung damage.[1] Of children who undergo allogeneic transplantation, 52-65% will develop cGVHD.[2]

“Imagine having a transplant and being told that you have a moderate to severe chronic condition that can sometimes also be life threatening,” said Dr. Paul A. Charpentierattending physician at Seattle Children’s Hospital and a principal investigator of the study.(+) “If these children were between one and 12 years old and were not responding to steroid treatment, we had no rigorously studied treatment options – until now. The iMAGINE trial has shown encouraging safety results and sustained response rates in children, and the new IMBRUVICA oral suspension formulation helps address the challenges children may face swallowing capsules or tablets.

“It is heartbreaking for parents to watch their child struggle with the debilitating effects of cGVHD, especially since there are so few treatment options,” said Susan Stewart, executive director of BMT InfoNet^, a nonprofit organization dedicated to providing patients and their loved ones with emotional support and high-quality, easy-to-understand information about blood stem cell transplants. “The FDA’s approval of IMBRUVICA puts another weapon in their arsenal and has the potential to truly make a difference for those facing this difficult disease.”

The new indication is based on results from the Phase 1/2 iMAGINE study, which showed an overall response rate (ORR) through week 25 of 60% (confidence interval [CI] 95%; 44-74) in patients with a median age of 13 years (range: one to 19 years) (n=47) with moderate to severe relapsed/refractory (R/R) cGVHD. Safety was consistent with the established profile for IMBRUVICA, with adverse events (AEs) seen in pediatric patients being consistent with those seen in adult patients with moderate to severe cGVHD. IMBRUVICA was approved for the treatment of adults with cGVHD after failure of one or more lines of systemic therapy in 2017. Due to its unique kinase profile (eg, inhibiting both BTK and interleukin-2 inducible T cells [ITK]), IMBRUVICA has the potential to provide clinical benefit for cGVHD.[3]

“The pediatric GVHDc community is a prime example of an underserved patient population with high unmet medical needs for which Janssen is committed to developing lifesaving therapies,” said Craig TenderMD, Global Head of Late developmentDiagnostics & Medical Affairs, Hematology & Oncology Janssen Research & Development, LLC. “GVHDc has life-threatening implications for children, and we are deeply proud of the opportunity to make an impact for these young IMBRUVICA patients and their families.”

About the iMAGINE Study

iMAGINE (PCYC-1146-IM) is an open-label, multicenter, single-arm trial of IMBRUVICA for the treatment of pediatric and young adult patients 1 year of age to less than 22 years of age with moderate or severe cGVHD as defined by the NIH Consensus Criteria. Primary endpoints included pharmacokinetics (PK) and safety, and secondary endpoints included ORR (complete response [CR]/partial answer [PR]) for 2014 NIH criteria, overall survival and duration of response (DOR). The study included 47 patients who needed additional treatment after failure of one or more prior lines of systemic treatment. Patients 12 years of age and older were treated with IMBRUVICA 420 mg orally once daily, and patients 1 year to less than 12 years of age were treated with IMBRUVICA 240 mg/m2 orally once daily. per day. Efficacy of IMBRUVICA was established based on ORR through week 25.

About IMBRUVICA

IMBRUVICA (ibrutinib) is a once-daily oral drug jointly developed and marketed by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company. IMBRUVICA blocks Bruton’s protein tyrosine kinase (BTK), which normal and abnormal B cells, including specific cancer cells, need to multiply and spread. By blocking BTK, IMBRUVICA can help move abnormal B cells out of their nurturing environment and inhibit their proliferation.[4],[5],[6]

IMBRUVICA is approved in over 100 countries and has been used to treat over 250,000 patients worldwide. There are over 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 US Food and Drug Administration (FDA) in November 2013, and is now indicated for adult patients in six therapeutic areas, including five hematologic cancers. These include indications for treating adults with chronic lymphocytic leukemia (CLL)/lymphocytic lymphoma (LLS) with or without deletion 17p (del17p), and adults with Waldenstrom’s macroglobulinemia (WM) and adult patients with previously treated mantle cell lymphoma (MCL)*, as well as to treat adult patients with previously treated marginal zone lymphoma (MZL) who require systemic treatment and who have received at least one treatment prior therapy with anti-CD20*, and adult and pediatric patients aged 1 year and older with prior therapy for chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy.[7]

Accelerated approval was granted for MCL and MZL based on overall response rate. Continued approval of MCL and MZL may depend on verification and description of clinical benefits in confirmatory trials.

As of 2019, the National Comprehensive Cancer Network (NCCN) recommends ibrutinib (IMBRUVICA) as the preferred treatment regimen for the initial treatment of CLL/LLS and has Category 1 treatment status for treatment-naïve patients without a 17p deletion. TP53 mutation and as a preferred treatment treatment naïve patients with 17p deletion/TP53 mutation. The NCCN Guidelines also recommend IMBRUVICA, with or without rituximab, as the preferred treatment regimen for the treatment of relapsed/refractory MCL, as the preferred Category 1 treatment regimen for untreated and previously treated WM patients, and as the preferred treatment regimen for Relapsed/refractory MZL .[8]

About the Janssen Pharmaceutical Companies of Johnson & Johnson

At Janssen, we are creating a future where illness is a thing of the past. We are the pharmaceutical companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting disease with science, improving access with ingenuity, and healing despair with heart. We focus on the areas of medicine where we can make the biggest difference: cardiovascular, metabolism and retina; Immunology; Infectious diseases and vaccines; neuroscience; Oncology; and pulmonary hypertension.

Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal and www.twitter.com/JanssenUS. Janssen Research and Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

Cautions Regarding Forward-Looking Statements

This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and impact of IMBRUVICA (ibrutinib) treatment. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could differ materially from the expectations and projections of Janssen Research and Development, LLC or any of the other Janssen Pharmaceutical Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents obtained by competitors; patent challenges; product efficacy or safety issues resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of healthcare products and services; changes in applicable laws and regulations, including global healthcare reforms; and trends towards health care cost containment. A list and additional descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s Annual Report on Form 10-K for the fiscal year ended January 2, 2022, including in the sections entitled “Caution Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in the Company’s most recent Quarterly Report on Form 10-Q, as well as in the Company’s subsequent filings with the Security and Exchange Commission. Copies of these documents are available online at www.sec.gov, www.jnj.com or upon request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies or Johnson & Johnson undertakes to update any forward-looking statements as a result of new information or future events or developments.

Contact:

Jessica Castles Smith

Such. : +1 732-501-8181

Christie Corbett

Such. : +1 857-636-0211