On September 1, 2020, the U.S. Food and Drug Administration (FDA) approved azacitidine tablets (Onureg®) for continued treatment of patients with acute myeloid leukemia who achieved first complete remission (CR) or CR with incomplete blood count recovery (CRi) following intensive induction chemotherapy and who are unable to complete intensive curative therapy.

FDA Approves Azacitidine Tablets for Acute Myeloid Leukemia

Efficacy was investigated in a multicenter, randomized (1:1), double-blind, placebo-controlled trial (QUAZAR; NCT01757535). Patients who achieved CR or CRi with intensive induction chemotherapy with or without receiving subsequent consolidation therapy (N = 472) received either 300 mg of azacitidine (n = 238) or placebo (n = 234) orally on days 1–14 of each 28-day cycle.

The main efficacy outcome measure was overall survival (OS). Median OS was 24.7 months (95% CI = 18.7, 30.5) in the azacitidine arm and 14.8 months (95% CI = 11.7, 17.6) in the placebo arm (HR = 0.69; 95% CI = 0.55, 0.86; p = 0.0009). A subgroup analysis showed consistency in OS benefit for patients in either CR or CRi.

The most common adverse reactions (≥ 10%) were nausea, vomiting, diarrhea, fatigue or asthenia, constipation, pneumonia, abdominal pain, arthralgia, decreased appetite, febrile neutropenia, dizziness, and pain in extremities.

The recommended azacitidine dose is 300 mg orally once daily with or without food on days 1–14 of each 28-day cycle until disease progression or unacceptable toxicity. 

View full prescribing information for azacitidine. 

FDA granted the application priority review and azacitidine was granted orphan drug designation. A description of FDA expedited programs is in the Guidance for Industry—Expedited Programs for Serious Conditions—Drugs and Biologics

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 800-FDA-1088.   

For assistance with single-patient oncology investigational new drug applications, contact the Oncology Center of Excellence’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov