All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit Know AML.

The AML Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy


Now you can personalise
your AML Hub experience!

Bookmark content to read later

Select your specific areas of interest

View content recommended for you

Find out more

The AML Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the AML Hub cannot guarantee the accuracy of translated content. The AML Hub and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.

Steering CommitteeAbout UsNewsletterContact
You're logged in! Click here any time to manage your account or log out.
You're logged in! Click here any time to manage your account or log out.

FDA extends approval for gemtuzumab ozogamicin in newly diagnosed CD33-positive AML

Jun 19, 2020

Bookmark this article

On June 16, 2020, the U.S. Food and Drug Administration (FDA) extended the indication of gemtuzumab ozogamicin (GO) for newly diagnosed CD33-positive acute myeloid leukemia (AML) to include pediatric patients ≥ 1 month of age. This approval was based on data from the AAML0531 trial (NCT00372593).1

Study design1,2

  • Phase III, multicenter, randomized study of 1,063 patients with newly diagnosed AML who were between the ages of 0 and 29 years
  • Aimed to compare the efficacy of GO in combination with chemotherapy with that of chemotherapy alone
  • Patients were randomized to 5-cycle chemotherapy alone or with GO administered at 3 mg/m2 once on Day 6 in the induction 1 stage and once on Day 7 of the intensification 2 stage
  • Primary endpoint: Event-free survival (EFS), measured from the date of trial entry until induction failure, relapse, or death by any cause
  • The study started in August 2006 and completed in August 2013


  • EFS hazard ratio was 0.84 (95% CI, 0.71–0.99)
  • Estimated percentage of patients free of induction failure, relapse, or death at 5 years was 48% (95% CI, 43–52) in the chemotherapy plus GO arm vs 40% (95% CI, 36–45) in the chemotherapy alone arm
  • No demonstrable difference in overall survival between treatment arms
  • The most common ≥ Grade 3 adverse reactions occurring in at least 5% of patients treated with GO included infection, febrile neutropenia, decreased appetite, hyperglycemia, mucositis, hypoxia, hemorrhage, increased transaminase, diarrhea, nausea, and hypotension

GO was previously approved by the FDA for the treatment of adult patients with newly diagnosed CD33-positive AML. Ongoing trials AML18 and AML19 are investigating how GO should be applied in AML and whether a fractionated dosing schedule can provide benefit over a single dose.

  1. S. Food and Drug Administration. FDA approves gemtuzumab ozogamicin for CD33-positive AML in pediatric patients. Published June 16, 2020. Accessed June 17, 2020
  2. Combination chemotherapy with or without gemtuzumab in treating young patients with newly diagnosed acute myeloid leukemia. Updated April 28, 2017. Accessed June 17, 2020.


Subscribe to get the best content related to AML delivered to your inbox