Azacitidine Tablets Approved for AML Following Chemotherapy

The FDA has approved azacitidine tablets, formerly known as CC-486, for the continued treatment of adults with acute myeloid leukemia (AML) who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction chemotherapy and who are ineligible for intensive curative therapy.

Approval was supported by the multicenter, randomized, double-blind, placebo-controlled phase III QUAZAR trial. The study enrolled 472 adults over the age of 55 with AML who had achieved first CR or CRi following intensive induction chemotherapy with or without consolidation treatment and who were ineligible for hematopoietic cell transplantation. Patients were randomized 1:1 to receive either once-daily oral azacitidine 300 mg or placebo on days 1 to 14 of each 28-day cycle.

Patients in the azacitidine cohort had a median overall survival of 24.7 months, compared with 14.8 months in the placebo group. A subgroup analysis showed a consistent survival benefit in the azacitidine-treated patients, regardless of CR or CRi status.

Common AEs (occurring in ≥10% of patients) included nausea, vomiting, diarrhea, fatigue/asthenia, constipation, pneumonia, abdominal pain, arthralgia, decreased appetite, febrile neutropenia, dizziness, and pain in extremity. Due to differences in pharmacokinetics, the drug is not a substitute for injectable azacitidine as frontline therapy for AML or myelodysplastic syndromes.

Sources: FDA press release, September 1, 2020; BMS press release, September 1, 2020.