FDA Expands Imbruvica for Young Children with GVHD

Imbruvica is the first approved treatment option for children under 12 with chronic graft versus host disease and the first BTKi therapy approved for pediatric patients.

The FDA has approved Imbruvica (ibrutinib) to treat children one year and older with chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy. This is the first pediatric indication for Imbruvica and the 12th approval for the therapy.

The recommended dose for patients 12 years and older is 420 mg taken orally once daily. In patients one year old to less than 12 years of age, the recommended dose is 240 mg/m2 taken orally once daily (up to a dose of 420 mg). This FDA approval also includes an oral suspension formulation of Imbruvica.

Imbruvica is jointly developed and commercialized by Janssen Biotech and Pharmacyclics, an AbbVie company.

“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,” Paul A. Carpenter, M.D., attending physician at Seattle Children's Hospital and a study principal investigator, said in a press release. “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.”

The approval is primarily based on positive results from the iMAGINE phase 1/2 clinical trial. The study demonstrated an overall response rate (ORR) through week 25 of 60% in patients median age 13 years (range, 1 to 19 years) with relapsed/refractory (R/R) moderate-to-severe cGVHD. The median duration of response was 5.3 months. Adverse reactions consistent with those observed in adult patients with moderate-to-severe cGVHD. The most common adverse events were anemia, musculoskeletal pain, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.

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 this indication. It was first approved by the FDA in 2013 and is indicated for adult patients in six disease areas, including five hematologic cancers. These include indications to treat adults with chronic lymphocytic leukemia/small lymphocytic leukemia, adults with Waldenström’s macroglobulinemia, adults with previously treated mantle cell lymphoma, adult patients with previously treated marginal zone lymphoma.