FDA Approves Bosutinib for Chronic Phase Ph-Positive CML in Pediatric Patients

The US Food and Drug Administration (FDA) has approved bosutinib (Bosulif) for pediatric patients one year of age and older who have chronic phase Philadelphia chromosome (Ph)-positive chronic myeloid leukemia (CML) that is newly diagnosed, or resistant or intolerant to prior therapy, according to the FDA.

The FDA also announced it approved a new capsule dosage form of bosutinib that is available in strengths of 50 mg and 100 mg.

The BCHILD trial evaluated the efficacy of bosutinib in pediatric patients with newly diagnosed, resistant, or intolerant chronic phase Ph-positive CML. The major efficacy outcome measures of the BCHILD trial included major cytogenetic response, complete cytogenetic response, and major molecular response (MMR).

In pediatric patients with newly diagnosed chronic phase Ph-positive CML, the major cytogenetic response rate was 76.2% and the complete cytogenetic response rate was 71.4%. The MMR rate was 28.6%.

In pediatric patients with resistant or intolerant chronic phase Ph-positive CML, the major cytogenetic response rate was 82.1%, and the complete cytogenetic response rate was 78.6%. The MMR rate was 50%.

The recommended dose for pediatric patients with newly diagnosed chronic phase Ph-positive CML is bosutinib 300 mg/m² orally once daily with food. The recommended dosage for pediatric patients with resistant or intolerant chronic phase Ph-positive CML is bosutinib 400 mg/m² orally once daily with food. For patients who are unable to swallow capsules, the contents of the capsules can be mixed with applesauce or yogurt.

This application was granted priority review and orphan drug designation, according to the FDA.

Source: US FDA, September 2023