Issue: July 10, 2020

Read more

June 16, 2020
1 min read
Save

FDA expands Mylotarg approval for pediatric AML subset

Issue: July 10, 2020
You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

The FDA today expanded the approval of gemtuzumab ozogamicin to include children aged 1 month and older newly diagnosed with CD33-positive acute myeloid leukemia.

Gemtuzumab ozogamicin (Mylotarg; Pfizer, Wyeth Pharmaceuticals) is a recombinant, humanized anti-CD33 antibody-drug conjugate.

Photomicrograph of bone marrow aspirate showing myeloblasts of acute myeloid leukemia (AML), a cancer of white blood cells.
FDA expanded the approval of gemtuzumab ozogamicin to include children aged 1 month and older newly diagnosed with CD33-positive acute myeloid leukemia.

FDA assessed the efficacy and safety of the agent for children using data from the multicenter randomized AAML0531 trial, which included 1,063 patients aged 0 to 29 years with newly diagnosed AML.

Researchers randomly assigned patients to five cycles of chemotherapy alone or with 3 mg/m2 gemtuzumab ozogamicin on day 6 of the induction 1 cycle and on day 7 of the intensification 2 cycle.

EFS, which served as the study’s main efficacy outcome measure, appeared significantly longer among patients assigned gemtuzumab ozogamicin (HR = 0.84; 95% CI, 0.71-0.99).

Forty-eight percent (95% CI, 43-52) of patients assigned gemtuzumab ozogamicin plus chemotherapy remained free of induction failure, relapse or death at 5 years compared with 40% (95% CI, 36-45) of patients assigned chemotherapy alone.

Researchers reported no difference in OS between the groups.

The most common grade 3 or worse adverse events associated with gemtuzumab ozogamicin that occurred during the induction 1 and intensification 2 cycles included infection, febrile neutropenia, decreased appetite, hyperglycemia, mucositis, hypoxia, hemorrhage, increased transaminase, diarrhea, nausea and hypotension.

The FDA previously granted gemtuzumab ozogamicin priority review for this indication.