Overview of 4 FDA Approvals in Acute Myeloid Leukemia

Daniel J. DeAngelo, MD, PhD, associate professor at the Harvard School of Medicine and a member of the Adult Leukemia Program at Dana-Farber Cancer Institute, highlighted the 4 FDA approvals in the last year for the treatment of patients with acute myeloid leukemia (AML).

Midostaurin (Rydapt) was the first FLT3 inhibitor approved for treatment in combination with chemotherapy. This has become the standard of care for patients with AML. CPX-351 (Vyzeos) was also approved as a treatment option for patients with newly diagnosed therapy-related AML or AML with myelodysplasia-relatedchanges, says DeAngelo. 

Gemtuzumab ozogamicin (Mylotarg) was FDA approved more than a decade ago, but has made a reappearance again with a new FDA approval for patients with CD33-positive AML. Dana-Farber is mirroring the ALFA-0701 trial, on which this approval was based. DeAngelo says data showed an improvement in event-free and overall survival with the addition of this drug to standard treatment.

Enasidenib was also approved in this landscape for patients with IDH2 mutations in the relapsed/refractory setting. This is still being investigated in ongoing trials.