Mylotarg (gemtuzumab ozogamicin) given ‘positive opinion’ by the EMA CHMP for use in untreated CD33-positive AML

On 23 February 2018, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) granted a positive opinion, recommending the approval of Mylotarg (gemtuzumab ozogamicin) in combination with daunorubicin and cytarabine for the treatment of patients aged 15 years and above with previously untreated  de novo, CD33-positive Acute Myeloid Leukemia (AML), except Acute Promyelocytic Leukemia (APL).¹ This positive opinion follows the recent U.S. Food and Drug Administration (FDA) approval of Mylotarg for newly diagnosed and relapsed/refractory CD33-positive AML on 4 September 2017, which was reported here.

Mylotarg, 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

This recommendation of approval for Mylotarg by the EMA CHMP was based on results from the phase III randomized ALFA-0701 study (NCT00927498), which showed that the combination of Mylotarg with standard chemotherapy regimen in newly diagnosed de novo adult AML patients significantly improved 3-year Event Free Survival (EFS) and Relapse Free Survival (RFS) compared to chemotherapy alone.³

If Mylotarg is approved by the European Commission (EC), the drug manufacturers, Pfizer, highlighted that “the addition of Mylotarg to standard chemotherapy will provide an important new treatment option for patients with AML who would typically be treated with chemotherapy alone”.