Posted: Wednesday, May 6, 2020
On May 1, the U.S. Food and Drug Administration (FDA) granted Priority Review status for the use of oral azacitidine as a maintenance treatment of adult patients with acute myeloid leukemia (AML). Patients eligible for this investigational therapy should have complete remission or complete remission with incomplete blood cell count recovery, following induction therapy with or without consolidation treatment. In addition, patients will need to be ineligible or choose not to have hematopoietic stem cell transplantation. Azacitidine is an investigational hypomethylating agent that incorporates into DNA and RNA.
This new drug application submission was based on the results of the international, double-blind, placebo-controlled phase III QUAZAR AML-001 study. The trial enrolled 472 patients who were randomly assigned to receive a once-daily dose of 300 mg of oral azacitidine or an oral placebo for 14 days of a 28-day cycle, plus best supportive care. Patients remained on treatment until unacceptable toxicity or disease progression. The trial met its primary endpoint of overall survival for patients receiving oral azacitidine versus placebo.