On 16 June 2020, the US Food and Drug Administration (FDA) extended the indication of gemtuzumab ozogamicin (MYLOTARG, Wyeth Pharmaceuticals LLC) for newly diagnosed CD33-positive acute myeloid leukaemia (AML) to include paediatric patients 1 month and older.<\/p>\n
Efficacy and safety in the paediatric population were supported by data from AAML0531 (NCT00372593), a multicentre randomised study of 1,063 patients with newly diagnosed AML ages 0 to 29 years. Patients were randomised to 5-cycle chemotherapy alone or with gemtuzumab ozogamicin (3 mg\/m2<\/sup>) administered once on day 6 in Induction 1 and once on day 7 in Intensification 2.<\/p>\n The main efficacy outcome measure was event-free survival (EFS) measured from the date of trial entry until induction failure, relapse, or death by any cause. The EFS hazard ratio was 0.84 (95% confidence interval [CI] 0.71-0.99). The estimated percentage of patients free of induction failure, relapse, or death at five years was 48% (95% CI 43%-52%) in the gemtuzumab ozogamicin plus chemotherapy arm versus 40% (95% CI 36%\u201145%) in the chemotherapy alone arm. No difference between treatment arms in overall survival was demonstrated.<\/p>\n The most common grade 3 and higher adverse reactions that occurred during Induction 1 and Intensification 2 in \u2265 5% of patients who received gemtuzumab ozogamicin were infection, febrile neutropenia, decreased appetite, hyperglycaemia, mucositis, hypoxia, haemorrhage, increased transaminase, diarrhoea, nausea, and hypotension.<\/p>\n Full prescribing information for MYLOTARG is available here<\/a>.<\/p>\n This application was granted priority review.<\/p>\n Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA\u2019s MedWatch Reporting System.<\/p>\n For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact FDA\u2019s Oncology Center of Excellence<\/a> Project Facilitate<\/a>.<\/p>\n