The FDA granted priority review status to CC-486 for maintenance treatment of adult patients with acute myeloid leukemia (AML) who achieved complete remission (CR) or CR with incomplete blood count recovery (CRi) after induction therapy with or without consolidation treatment and who are ineligible for hematopoietic cell transplantation (HCT).
The decision was based on results from the QUAZAR AML-001 trial, a phase III study of 472 adults with AML who achieved CR or CRi following induction chemotherapy (with or without consolidation) and had no plans to go to HCT or were ineligible for transplant. Patients were randomized 1:1 to either CC-486 dosed at 300 mg or placebo once daily for 14 days of a 28-day cycle, plus best supportive care. Treatment continued until disease progression or unacceptable toxicity. The primary endpoint was overall survival (OS), and key secondary endpoints included relapse-free survival (RFS) and safety and tolerability.
Patients who received CC-486 achieved a median OS of 24.7 months, significantly longer than the median OS of 14.8 months for those who received the placebo (95% CI 0.55-0.86). The CC-486 group also achieved a median RFS of 10.2 months, compared with the placebo group’s median RFS of 4.8 months (95% CI 0.52-0.81). The 1-year relapse rate was 53% in the CC-486 group and 71% in the placebo group. The most common adverse events, occurring in more than 20% of patients, were nausea, vomiting, and diarrhea.
The FDA set a Prescription Drug User Fee Act decision goal date of September 3, 2020.