Professor Andrew Wei

Australian patients with AML can now access azacitidine 200 mg/300 mg tablets (Onureg, Bristol Myers Squibb) on the PBS.

From 1 September, the anti-metabolite chemtherapy drug is reimbursed for the continued treatment of adult patients with 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.

The approval is based on results from the international phase 3 QUAZAR AML-001 study [link to NEJM here] in which treatment with the oral hypomethylating agent led to significantly longer overall and relapse-free survival than placebo.

Eligible patients with AML were aged ≥55 years, within four months of achieving first CR or CRi following intensive induction chemotherapy with or without consolidation treatment, and were not candidates for HSCT at the time of screening.

The study, led by Melbourne haematologist Professor Andrew Wei, found the median overall survival from the time of randomisation was 24.7 months with azacitidine versus 14.8 months with placebo (P<0.001).

The proportion of patients surviving at 1 year was 72.8% with azacitidine and 55.8% with placebo and 50.6% and 37.1% respectively at 2 years.

Similarly, relapse-free survival was significantly longer with azacitidine than placebo at 10.2 months v 4.8 months (P<0.001).

The findings were consistent across most subgroups including age, sex, cytogenetic risk, initial response to induction chemotherapy, receipt of consolidation therapy, or measurable residual disease status at trial entry.

Professor Wei said in a statement from BMS that as well as the survival benefit, the once daily oral formulation was convenient for patients.

“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 complete intensive curative therapy, including haematopoietic stem cell transplant.”

The trial found the most common serious adverse reactions in patients who received azacitidine were febrile neutropenia (6.8%) and pneumonia (5.1%).