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FDA grants Mylotarg® (gemtuzumab ozogamicin) approval for the treatment of CD33 positive AML

Sep 4, 2017

On 1st September 2017, the U.S. Food and Drug Administration (FDA) granted approval to Mylotarg® (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, Mylotarg® “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”.

Mylotarg® (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

Mylotarg® 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FDA via accelerated review in May 2000 for monotherapy of elderly individuals with relapsed AML, but it was voluntarily withdrawn by the company, Pfizer, from market on October 15th, 2010 for the treatment of AML patients.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 Mylotarg® 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 Mylotarg® 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 Mylotarg® 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 Mylotarg® 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. [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 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.