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

The approval was based on data from the multicenter, randomized, double-blind, placebo-controlled, phase 3 QUAZAR study that evaluated the efficacy and safety of Onureg, a nucleoside metabolic inhibitor, in 472 patients aged ≥55 years with AML who achieved first complete remission or complete remission with incomplete blood count recovery following intensive induction chemotherapy. Patients were randomized 1:1 to receive Onureg 300mg or placebo orally once daily on days 1 through 14 of each 28-day cycle, in addition to best supportive care. The primary end point was overall survival.

Results showed a statistically significant improvement in overall survival (OS) for patients treated with Onureg; median OS was 24.7 months (95% CI, 18.7-30.5) in the Onureg arm and 14.8 months (95% CI, 11.7-17.6) in the placebo arm (hazard ratio 0.69; 95% CI, 0.55-0.86; P =.0009). Additionally, in a subgroup analysis, the OS benefit was found to be consistent for patients in either complete remission or complete remission with incomplete blood count recovery.

With regard to safety, the most common adverse reactions reported with treatment were nausea, vomiting, diarrhea, fatigue/asthenia, constipation, pneumonia, abdominal pain, arthralgia, decreased appetite, febrile neutropenia, dizziness, and pain in extremity. 

“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,” said Andrew Wei, MBBS, PhD, QUAZAR AML-001 lead investigator, Alfred Hospital and Monash University, Melbourne, Australia.

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Onureg is supplied in 200mg and 300mg tablets and is expected to be available soon. Due to differences in pharmacokinetic parameters, the recommended dose and schedule for Onureg are different from those for intravenous or subcutaneous azacitidine products; Onureg should not be substituted for these other formulations.

For more information visit onuregpro.com.

References

  1. US Food and Drug Administration approves Onureg® (azacitidine tablets), a new oral therapy, as continued treatment for adults in first remission with acute myeloid leukemia. https://news.bms.com/press-release/corporatefinancial-news/us-food-and-drug-administration-approves-onureg-azacitidine-ta. Accessed September 1, 2020. 
  2. Onureg [package insert]. Summit, NJ: Bristol-Myers Squibb; 2020.