Azacitidine Tablets for Acute Myeloid Leukemia


The FDA approved azacitidine tablets (Onureg; Celgene Corporation) on September 1, 2020


The FDA approved azacitidine tablets (Onureg; Celgene Corporation) on September 1, 2020.1 Azacitidine tablets are indicated for the continued treatment of patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy. Azacitidine is a pyrimidine nucleoside analog of cytidine that inhibits DNA/RNA methyltransferases.2 It is incorporated into DNA and RNA, inhibiting DNA and RNA methyltransferases and reducing DNA and RNA methylation; alters DNA gene expression; and decreases protein synthesis and RNA stability.


Investigators evaluated the efficacy of azacitidine tablets in the QUAZAR AML-001 trial (NCT01757535), a multicenter, randomized, double-blind, placebo-controlled study.1 Patients with AML (n = 472) who achieved CR or CRi while undergoing intensive induction chemotherapy with or without subsequent consolidation therapy were randomized (1:1) to receive azacitidine 300 mg (n = 238) or placebo (n = 234) orally on days 1 to 14 of each 28-day cycle. The primary efficacy outcome measure was overall survival (OS), which was 24.7 months (95% CI, 18.7-30.5) with azacitidine and 14.8 months (95% CI, 11.7-17.6) with placebo (HR, 0.69; 95% CI, 0.55-0.86; P = .0009). The OS benefit was consistent in patients in both CR and CRi subgroups.


The most common adverse reactions (≥ 10%) were nausea, vomiting, diarrhea, fatigue/asthenia, constipation, pneumonia, abdominal pain, arthralgia, decreased appetite, febrile neutropenia, dizziness, and pain in extremities.2 Because of the potential for myelosuppression, monitor complete blood counts (CBCs) every other week for the first 2 cycles and prior to starting each cycle thereafter. Azacitidine may cause fetal harm. Women are advised not to breastfeed during treatment with azacitidine and for 1 week after the last dose. Pregnancy testing is recommended for women of reproductive potential before starting azacitidine, and they should use effective contraception during azacitidine treatment and for 6 months after the last dose. Men should use effective contraception during treatment with azacitidine and for 3 months after the last dose. Azacitidine is associated with an increase in early mortality in patients with myelodysplastic syndromes. Treatment of these patients with azacitidine is not recommended. Do not substitute azacitidine for intravenous or subcutaneous azacitidine (due to substantial differences in pharmacokinetic parameters and indications). No reported drug interactions exist at this time.

Dosing and Cost

Both 200 mg and 300 mg tablets are available.2 The recommended dose is 300 mg orally once daily with or without food on days 1 through 14 of each 28-day cycle. A dose reduction to 200 mg orally once daily is recommended for severe myelosuppression, or grade 3 or 4 nausea, vomiting, diarrhea, or other toxicity. Continue azacitidine until disease progression or unacceptable toxicity. Administer an antiemetic 30 minutes prior to each dose of azacitidine for the first 2 cycles. Antiemetic prophylaxis may be omitted after 2 cycles if there has been no nausea and vomiting. The cost per 200-mg and 300-mg tablet is $1813.55.3 The cost for one 28-day cycle of azacitidine therapy is $25,389.70.


1. FDA approves Onureg (azacitidine tablets) for acute myeloid leukemia. FDA. Updated September 1, 2020. Accessed October 6, 2020.

2. Onureg. Prescribing information. Celgene Corporation; 2020. Accessed October 6, 2020.

3. Wolters Kluwer Health, Inc. Lexicomp. Lexi-Drugs/Azacitidine. Accessed October 6, 2020.

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