Janssen bags USFDA nod for IMBRUVICA for chronic graft-versus-host disease treatment

New Delhi: The Janssen Pharmaceutical Companies of Johnson & Johnson has announced that the U.S. Food and Drug Administration (USFDA) has approved IMBRUVICA (ibrutinib) for the treatment of pediatric patients one year and older with chronic graft-versus-host disease (cGVHD) after the 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 ages one to less than 12. IMBRUVICA is now the first FDA-approved therapy for these younger patients who previously had no approved treatment options for this life-threatening disease," claims the company.

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 transplant recipient's body. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. Among children who undergo allogeneic transplants, 52-65 percent will develop cGVHD.

"Imagine going through a transplant and then being told you have a moderate to severe chronic disease that can sometimes also be life-threatening," said Dr. Paul A. Carpenter, attending physician at Seattle Children's Hospital and a study principal investigator. "If these children were between one and 12 and didn't respond to steroid treatment, we didn't have any rigorously studied treatment options — until now. The iMAGINE trial showed encouraging safety results and sustained response rates in children, and the new IMBRUVICA oral suspension formulation helps address challenges children may have with swallowing capsules or tablets."

"It's 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 non-profit 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 approval of IMBRUVICA puts another weapon in their arsenal and has the potential to truly make a difference for those who are faced with this challenging 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 percent (Confidence Interval [CI] 95 percent; 44-74) in patients median age 13 years (range, one to 19 years) (n=47) with relapsed/refractory (R/R) moderate to severe cGVHD. Safety was consistent with the established profile for IMBRUVICA, with observed adverse events (AEs) in pediatric patients being consistent with those observed in adult patients with moderate to severe cGVHD.

IMBRUVICA was approved to treat adults with cGVHD after failure of one or more lines of systemic therapy in 2017. Because of its unique kinase profile (e.g., inhibiting both BTK and interleukin-2-inducible T-cell kinase [ITK]), IMBRUVICA has the potential to provide a clinical benefit for cGVHD.

"The pediatric cGVHD community is a prime example of an underserved patient population with high unmet medical needs for whom Janssen is committed to developing life-saving therapies," said Craig Tendler, M.D., Global Head of Late Development, Diagnostics & Medical Affairs, Hematology & Oncology Janssen Research & Development, LLC. "cGVHD has life-threatening implications for children, and we are deeply proud of the opportunity to make an impact for these young patients with IMBRUVICA and their families."

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