Oops, you're using an old version of your browser so some of the features on this page may not be displaying properly.

MINIMAL Requirements: Google Chrome 24+Mozilla Firefox 20+Internet Explorer 11Opera 15–18Apple Safari 7SeaMonkey 2.15-2.23

FDA Approves Gemtuzumab Ozogamicin for CD33-positive AML in Paediatric Patients

Efficacy and safety were supported by data from the AAML0531 study
23 Jun 2020
Anticancer agents & Biologic therapy;  Cancer in Special Situations / Population;  Haematologic malignancies

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.

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) administered once on day 6 in Induction 1 and once on day 7 in Intensification 2.

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%‑45%) in the chemotherapy alone arm. No difference between treatment arms in overall survival was demonstrated.

The most common grade 3 and higher adverse reactions that occurred during Induction 1 and Intensification 2 in ≥ 5% of patients who received gemtuzumab ozogamicin were infection, febrile neutropenia, decreased appetite, hyperglycaemia, mucositis, hypoxia, haemorrhage, increased transaminase, diarrhoea, nausea, and hypotension.

Full prescribing information for MYLOTARG is available here.

This application was granted priority review.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact FDA’s Oncology Center of Excellence Project Facilitate.

This site uses cookies. Some of these cookies are essential, while others help us improve your experience by providing insights into how the site is being used.

For more detailed information on the cookies we use, please check our Privacy Policy.

Customise settings