FDA Approves Oral Azacitidine for Acute Myeloid Leukemia


Azacitidine showed an overall survival benefit among patients with acute myeloid leukemia compared with a placebo.

The FDA has approved 300 mg tablets of azacitidine (Onureg, Bristol Myers Squibb) for the continued treatment of adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or CR with incomplete blood count recovery (CRi) following intensive induction chemotherapy and who are not able to complete intensive curative therapy.

AML accounts for 1.1% of all cancers, which amounts to nearly 20,000 new cases a year. In 2017, there were an estimated 64,500 people living with AML in the United States, according to a Bristol Myers Squibb press release. It is characterized by the rapid growth of abnormal cells in the bone marrow, which then interferes with normal blood cell production and function.

Because of this, AML can present with signs of anemia, bleeding, and infection. AML can rapidly progress if not treated promptly and is associated with diverse genetic mutations.

The azacitidine approval is based on the phase 3 Quazar AML-001 study, which showed the drug significantly improved overall survival (OS) at the study’s primary endpoint of almost 10 months. Median OS time was 24.7 months from the time of randomization for those receiving azacitidine.

For those receiving the placebo, the median OS time was 14.8 months. New or worsening grade 3 of 4 neutropenia and thrombocytopenia occurred in 49% and 22% of patients administered azacytidine, respectively.

“Continued treatment with Onureg demonstrated an overall survival benefit in adults with AML who had achieved first complete remission in the QUAZAR® AML-001 study and, notably, it has the potential to do this in a convenient manner, given its once daily oral formulation…This approval should help establish continued treatment with Onureg as a standard component of AML therapy for adults who achieved first complete remission following chemotherapy and who cannot proceed to intensive curative therapy, like hematopoietic stem cell transplant,” QUAZAR® AML-001 lead investigator Andrew Wei, MBBS, PhD, said in the press release.

Serious adverse effects (AEs) occurred in 15% of the participants receiving azacitidine, including pneumonia and febrile neutropenia. The most common AEs among patients administered azacytidine compared with placebo were nausea, vomiting, and diarrhea.


U.S. Food and Drug Administration Approves Onureg® (azacitidine tablets), a New Oral Therapy, as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia (Press release) New York, NY September 1, 2020, Bristol Myers Squibb, Accessed September 1, 2020

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