Oral Onureg First Drug Approved for Adults with AML in Remission

Web Exclusives - FDA Approvals, Leukemia, Select Drug Profiles

On September 1, 2020, the FDA approved azacitidine (Onureg; Celgene), an oral nucleoside metabolic inhibitor, for maintenance treatment of adults with acute myeloid leukemia (AML) who had first complete remission (CR) or CR with incomplete blood count recovery after intensive induction chemotherapy and who are not candidates for intensive curative therapy. The FDA granted azacitidine an orphan drug designation and used its priority review for this indication.

The FDA approved azacitidine based on the results of the QUAZAR study, a multicenter, randomized, double-blind, placebo-controlled clinical trial of 472 patients with AML who achieved CR or CR with incomplete blood count recovery. The patients were randomized in a 1:1 ratio to azacitidine 300 mg (N = 238) or to placebo (N = 234) on days 1 to 14 of each 28-day cycle. The primary end point was overall survival (OS).

The median OS was 24.7 months (18.7-30.5) in the azacitidine arm versus 14.8 months in the placebo arm (hazard ratio, 0.69; 95% confidence interval, 0.55-0.86; P = .0009). A subgroup analysis showed consistent OS benefits with azacitidine in patients with CR or with CR and incomplete blood count recovery.

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

The recommended dose of azacitidine is 300 mg orally once daily, on days 1 to 14 of each 28-day cycle.

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Last modified: August 30, 2021
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