European regulators block pediatric use of Mylotarg for AML. The EMA's CHMP rejected Pfizer's application to extend gemtuzumab ozogamicin (Mylotarg) to children aged 1–17 with newly diagnosed AML, citing insufficient evidence of benefit. The key study lacked a comparator arm, and a supportive trial showed improved event-free survival but no overall survival benefit — and more deaths in patients who had achieved remission.
European regulators block pediatric use of Mylotarg for AML
Children in Europe with newly diagnosed acute myeloid leukemia (AML) won't be gaining a new targeted treatment option anytime soon. The EMA's Committee for Medicinal Products for Human Use (CHMP) rejected Pfizer's application to extend Mylotarg (gemtuzumab ozogamicin) — currently approved for adults 15+ with AML — to children aged 1 to under 18 years.
The decision came down to insufficient evidence. The main study of 515 children had no comparator arm (every child received the drug), making it impossible to determine whether Mylotarg actually improved outcomes. A supportive study of 1,063 patients did show improved event-free survival (EFS), but this didn't translate into better overall survival — and more deaths occurred among patients in the Mylotarg group who had achieved complete remission.
By the Numbers
Why it matters: This ruling leaves a significant gap in treatment options for pediatric AML patients in Europe. While the adult indication remains unchanged, the EMA will update Mylotarg's prescribing information to include the pediatric study data — giving clinicians at least some guidance when navigating treatment decisions.
