The US Food and Drug Administration (FDA) has granted approval to Pfizer’s antibody-drug conjugate (ADC) Mylotarg (gemtuzumab ozogamicin) for the treatment of patients with acute myeloid leukaemia (AML).

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With the approval, the Pfizer ADC can be used to treat adults with newly diagnosed CD33-positive AML, as well as adults and children aged two and older with relapsed or refractory CD33-positive AML.

Mylotarg comprises the cytotoxic agent calicheamicin attached to a monoclonal antibody (mAB) targeting CD33, which is an antigen expressed on the surface of myeloblasts in up to 90% of AML patients.

It is currently the first approved therapy with an indication that includes paediatric AML, and also only AML treatment that targets CD33.

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Pfizer oncology global president Liz Barrett said: “The FDA approval of Mylotarg fills a critical unmet need for many adults and children with AML, which can be fatal in a matter of months or even weeks if not treated and has a high relapse rate.

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“Based on clinical data, real-world experience and support from the AML community, we are grateful Mylotarg now has the potential to help a broad range of AML patients.”

"Based on clinical data, real-world experience and support from the AML community, we are grateful Mylotarg now has the potential to help a broad range of AML patients."

The current FDA approval is based on the results from several investigator-led clinical trials, including ALFA-0701, AML-19 and MyloFrance-1.1.

ALFA-0701 is the multicentre, randomised, open-label, Phase III trial that involved 271 patients with newly diagnosed de novo AML.

The AML-19 was also a multicentre, randomised, open-label Phase III trial that compared single agent Mylotarg to best supportive care for elderly patients who could not tolerate other AML therapies.

The single-arm, open-label Phase II MyloFrance-1 trial investigated 57 adult patients in first relapse, and the efficacy of the treatment was demonstrated on the basis of complete remission (CR) rate and duration of remission.

Image: Bone marrow aspirate showing acute myeloid leukaemia. Photo: courtesy of VashiDonsk at the English language Wikipedia.