BMS’s oral AML therapy Onureg receives EU approval

The European Commission has granted Bristol Myers Squibb’s (BMS) Onureg a full marketing authorisation as maintenance therapy for adult patients with acute myeloid leukaemia (AML). 

Onureg (azacitidine tablets) has been cleared for the treatment of AML patients who have achieved complete remission (CR) or CR with incomplete blood count recovery (CRi), following first-line chemotherapy with or without consolidation treatment who are not candidates for stem cell transplant.

The approval is based on results from the phase 3 QUAZAR AML-001 study, which evaluated Onureg maintenance therapy versus placebo in this patient population. Dosing was once a day for 14 days of a 28-day cycle, with treatment continuing until disease relapse.

The primary endpoint of median overall survival (OS) from time of randomisation was 24.7 months in the Onureg arm, compared to 14.8 months for placebo.

The median duration of treatment was 12 cycles for Onureg and six cycles with placebo, with median relapse-free survival also significantly longer with Onureg compared to placebo – 10.2 months and 4.8 months, respectively.

However, patients in the Onureg arm experienced higher rates of certain adverse reactions compared to placebo in the clinical trial.

This includes grade 3 or 4 adverse events of neutropenia, with 41% of Onureg-treated patients experiencing this side effect compared to 24% of patients in the placebo arm.

“Today’s approval of Onureg represents a significant advance for patients in the European Union living with acute myeloid leukaemia, who have remained in urgent need of maintenance therapies for this aggressive blood cancer,” said Noah Berkowitz, senior vice president, haematology development, Bristol Myers Squibb.

“We are committed to helping to improve long-term outcomes and greatly extending survival for patients with hard-to-treat diseases, as we work collaboratively with European Union member states to make Onureg available to eligible patients as quickly as possible,” he added.

Onureg is the oral version of Vidaza, two of the drugs BMS gained as part of its acquisition of Celgene for $74bn in 2019.

In the US, the Food and Drug administration (FDA) approved Onureg in September 2020 for the continued treatment of AML patients, based on the same QUAZAR AML-001 study results.