Bosutinib approved by EMA for children ≥ 6 years with CML based on the results of the ITCC-054/COG AAML1921 trial.

On 2 June 2025, Bosutinib has been approved by the European Medicines Agency (EMA) for the treatment of children ≥ 6 years with Philadelphia chromosome-positive chronic myeloid leukemia (CML) in chronic phase.

The registrational study has been carried out as an academic sponsored trial within the ITCC-consortium. The ITCC-054/COG AAML1921 was sponsored by the Erasmus MC and carried out also by the Princess Máxima Center (coordinating investigator: Michel Zwaan), in collaboration with the Children's Oncology Group (COG) (Coordinating investigator: Nobuko Hijiya). The study was implemented through an innovative partnership in the field of pediatric oncology, a ‘clinical research collaboration’ between Pharma (Pfizer Inc) and Academy to deliver a fit for filing study.

The results of the study have already led to the approval of Bosutinib by the Food and Drug Administration (FDA) in the U.S. for pediatric patients 1 year of age and older with CML on September 26, 2023.

In this single arm study, which involved 64 children and 67 participating hospitals in Europe and the U.S., bosutinib was found to have an equivalent efficacy compared to other tyrosine kinase inhibitors. Muscle and bone pain were generally less common than with other drugs when comparing across trials. However, nausea or diarrhea were more common. In addition, research in animals has shown that bosutinib hinders growth less than other TKIs, a side effect which strongly affects children with CML who could remain on treatment for decades. Although the enrolment is completed, the trial is still active to collect long-term safety data and the results on the effect of bosutinib on the growth- and bone metabolism are expected at the end of the trial in August 2028.