NICE recommends funding for gemtuzumab ozogamicin for patients with untreated CD33+ acute myeloid leukemia

On 14 November 2018, the National Institute for Health and Care Excellence (NICE) published an appraisal guidance recommending gemtuzumab ozogamicin (Mylotarg®), with daunorubicin and cytarabine, as an option for untreated de novo CD33-positive acute myeloid leukemia (AML), except acute promyelocytic leukemia, in people 15 years and over.¹

This recommendation would only be applicable if patients start induction therapy when either the cytogenetic test confirms that the disease has favorable, intermediate or unknown cytogenetics (that is because the test was unsuccessful) or when their cytogenetic test results are not yet available and they start consolidation therapy. In addition, the recommendation by NICE is also dependent on the drug manufacturers, providing gemtuzumab ozogamicin according to the commercial arrangement.¹

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. As a result, this ADC can then dispense the anti-tumor antibiotic calicheamicin to CD33-expressing tumor cells.²

In April 2018, the European Commission (EC) granted approval to 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 AML. The approval by the EC for Mylotarg was based on results obtained from the phase III ALFA-701 study (NCT00927498). Data from the ALFA-701 study showed that combination of Mylotarg with standard chemotherapy regimen in newly diagnosed de novo AML patients significantly improved 3-year event-free survival and relapse-free survival (RFS) compared to chemotherapy alone.³