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

Introducing

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
  TRANSLATE

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

The AML Hub is an independent medical education platform, sponsored by Daiichi Sankyo, Jazz Pharmaceuticals, Kura Oncology, Roche and Syndax and has been supported through a grant from Bristol Myers Squibb. The funders are allowed no direct influence on our content. The levels of sponsorship listed are reflective of the amount of funding given. View funders.

2017-09-04T09:43:55.000Z

FDA grants gemtuzumab ozogamicin approval for the treatment of CD33 positive AML

Sep 4, 2017
Share:

Bookmark this article

On 1st September 2017, the U.S. Food and Drug Administration (FDA) granted approval to gemtuzumab ozogamicin for the treatment of adult patients with newly diagnosed CD33-positive Acute Myeloid Leukemia (AML). Mylotarg® was also approved for adults and pediatric patients 2 years and older with Relapsed or Refractory (R/R) CD33-positive AML.1 According to the drug manufacturers, Pfizer, gemtuzumab ozogamicin “is the first therapy with an indication that includes pediatric AML. It is also the only AML therapy that targets CD33, an antigen expressed on AML cells in up to 90% of patients”.

Gemtuzumab ozogamicin, an Antibody Drug Conjugate (ADC), is an antibody-targeted chemotherapeutic agent consisting of a humanized murine CD33 antibody. Since it is a humanized anti-CD33 monoclonal antibody, it is highly specific for targeting leukemic blasts. Mylotarg® binds to and is internalized by tumor cells expressing CD33. As a result, this ADC can then dispense the anti-tumor antibiotic calicheamicin to CD33-expressing tumor cells.2

Gemtuzumab ozogamicin, was previously approved by the FDA via accelerated review in May 2000 for monotherapy of elderly individuals with relapsed AML, but it was voluntarily withdrawn by the company, Pfizer, from market on October 15th, 2010 for the treatment of AML patients. However, in February 2017, the AGP reported on the resubmission of a Biologics License Application (BLA), that sought approval of gemtuzumab ozogamicin in combination with daunorubicin and cytarabine for the treatment of adults with treatment-naive CD33–positive AML. The FDA Oncologic Drug Advisory Committee (ODAC) also unanimously voted 6 to 1 in favor of allowing Mylotarg® to be approved for the treatment of newly diagnosed adult patients with de novo CD33-positive AML in July 2017, which was reported here.

The approval by the FDA for gemtuzumab ozogamicin was based on results obtained from the phase III ALFA-701 study (NCT00927498), the phase III AML-19 study, and the phase II MyloFrance-1 study. Data from the ALFA-701 study showed that combination of gemtuzumab ozogamicin with standard chemotherapy regimen in adult newly diagnosed de novo AML patients significantly improved 3-year Event Free Survival (EFS) and Relapse Free Survival (RFS) compared to chemotherapy alone.3

Additionally, findings from the AML-19 study, which compared gemtuzumab ozogamicin  monotherapy to best supportive care in elderly AML patients, showed that Mylotarg® significantly improved the Overall Survival (OS) compared to best supportive care (4.9 vs 3.6 months, HR = 0.69, P = 0.005).4

The MyloFrance-1 study in adult patients (n = 57) in first relapse, showed that monotherapy with gemtuzumab ozogamicin resulted in 26% of patients achieving Complete Remission (CR) with a median RFS of 11.6 months.5

  1. Business Wire: Pfizer Receives FDA Approval for MYLOTARG™ (gemtuzumab ozogamicin). 2017 Sep 1. http://www.businesswire.com/news/home/20170901005518/en/Pfizer-Receives-FDA-Approval-MYLOTARG%E2%84%A2-gemtuzumab-ozogamicin [Accessed 2017 Sep 1].
  2. Ricart A D. Antibody-drug conjugates of calicheamicin derivative: gemtuzumab ozogamicin and inotuzumab ozogamicin. Clin Cancer Res. 2011 Oct 15; 17(20): 6417-27. DOI: 10.1158/1078-0432.CCR-11-0486.
  3. Castaigne S.et al. Final Analysis of the ALFA 0701 Study. Blood. 2014; 124: 376.
  4. Amadori S. et al. Randomized trial of two schedules of low-dose gemtuzumab ozogamicin as induction monotherapy for newly diagnosed acute myeloid leukemia in older patients not considered candidates for intensive chemotherapy. A phase II study of the EORTC and GIMEMA leukemia groups (AML-19). Br J Haematol. 2010 May; 149(3):376-82. DOI: 10.1111/j.1365-2141.2010.08095.x. Epub 2010 Mar 8.
  5. Taksin A.L. et al. High efficacy and safety profile of fractionated doses of Mylotarg as induction therapy in patients with relapsed acute myeloblastic leukemia: a prospective study of the alfa group. Leukemia. 2007 Jan; 21(1): 66–71. Epub 2006 Oct 19.

Your opinion matters

HCPs, what is your preferred format for educational content on the AML Hub?
21 votes - 68 days left ...

Related articles

Newsletter

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