Azacitidine Tablets Approved by FDA for Patients With AML in First Remission
Posted: Thursday, September 3, 2020
On September 1, the U.S. Food and Drug Administration (FDA) approved the use of 300-mg azacitidine (Onureg) tablets for oral therapy in adults with acute myeloid leukemia (AML) who achieved first complete remission. Patients with AML in complete remission but with incomplete blood cell count recovery following intensive induction chemotherapy and who are not able to complete intensive curative therapy are also eligible for treatment.
The FDA approval was based on the results of the international, double-blind, phase III QUAZAR AML-001 clinical trial. Eligibility included patients 55 years or older with AML and within 4 months of achieving first complete remission or complete remission with incomplete blood cell count recovery following intensive induction chemotherapy with or without consolidation treatment and who were not candidates for hematopoietic stem cell transplant at the time of screening (n = 472). Patients were randomly assigned to receive 300 mg of azacitidine orally (n = 238) or an oral placebo (n = 234) once daily plus best supportive care. Oral azacitidine was continued until disease progression or unacceptable toxicity.
There was a statistically significant and clinically meaningful improvement in overall survival of nearly 10 months with azacitidine versus placebo. The median overall survival was 24.7 months with azacitidine compared with 14.8 months (P = .0009) with placebo.
Serious adverse reactions occurred in 15% of patients given azacitidine. Sepsis occurred in one patient taking azacitidine. The most common adverse reactions with azacitidine versus placebo were nausea (65% vs. 24%), vomiting (60% vs. 10%), diarrhea (50% vs. 21%), fatigue/asthenia (44% vs. 25%), constipation (39% vs. 24%), pneumonia (27% vs. 17%), abdominal pain (22% vs. 13%) arthralgia (14% vs. 10%), decreased appetite (13% vs. 6%), febrile neutropenia (12% vs. 8%), dizziness (11% vs. 9%), and extremity pain (11% vs. 5%).