FDA approves bosutinib for pediatric patients with chronic myelogenous leukemia
Regulatory approval: On September 26, 2023, the FDA approved bosutinib (Bosulif, Pfizer) for pediatric patients aged ≥1 year with chronic-phase (CP) Philadelphia chromosome–positive (Ph+) chronic myelogenous leukemia (CML) that is newly diagnosed or resistant/intolerant to prior therapy. The agency also approved a new capsule dosage form (50 mg and 100 mg). Approval was supported by results from the multicenter, nonrandomized, open-label BCHILD trial (NCT04258943).
FDA approves bosutinib for pediatric CP Ph+ CML
Type: Regulatory approval
Date: 2023-09-26
Agency: US Food and Drug Administration (FDA)
On September 26, 2023, the FDA approved bosutinib (Bosulif; Pfizer) for pediatric patients aged 1 year and older with chronic-phase (CP) Philadelphia chromosome–positive (Ph+) chronic myelogenous leukemia (CML) that is newly diagnosed (ND) or resistant/intolerant (R/I) to prior therapy. The FDA also approved a new capsule dosage form (50 mg and 100 mg).
Supporting evidence
Efficacy was evaluated in BCHILD (NCT04258943), a multicenter, nonrandomized, open-label trial enrolling 28 R/I pediatric patients treated at 300–400 mg/m² once daily and 21 ND patients treated at 300 mg/m² once daily.
ND CP Ph+ CML: MCyR 76.2% (95% CI 52.8–91.8); CCyR 71.4% (95% CI 47.8–88.7); MMR 28.6% (95% CI 11.3–52.3). Median follow-up 14.2 months (range 1.1–26.3).
R/I CP Ph+ CML: MCyR 82.1% (95% CI 63.1–93.9); CCyR 78.6% (95% CI 59.0–91.7); MMR 50% (95% CI 30.6–69.4). Two of 14 patients achieving MMR later lost MMR (after 13.6 and 24.7 months). Median follow-up 23.2 months (range 1.0–61.5).
Safety and dosing (per FDA label)
Common adverse reactions (≥20%) included diarrhea, abdominal pain, vomiting, nausea, rash, fatigue, hepatic dysfunction, headache, pyrexia, decreased appetite, and constipation. Recommended dosing: 300 mg/m² QD with food (ND) and 400 mg/m² QD with food (R/I). The application received priority review and orphan drug designation.