Expanded PBS Listing for Venetoclax

From 1 December 2021, venetoclax is available on the Pharmaceutical Benefits Scheme (PBS) for the treatment of acute myeloid leukaemia (AML). This expands on the previous listing for chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL).

Venetoclax is an oral inhibitor of the anti-apoptotic protein, B-cell lymphoma-2 (BCL-2). This protein is overexpressed in many malignancies, including CLL and AML, leading to dysregulation of apoptosis.

The PBS listing for AML requires patients to receive venetoclax in combination with azacitidine. The VIAL-A trial studied the safety and efficacy of this combination. Patients were randomly assigned to receive venetoclax plus azacitidine or control (azacitidine plus placebo). The primary endpoint of mean overall survival was 14.7 months in the venetoclax group and 9.6 months in the control group. Composite complete remission (complete remission or complete remission with incomplete haematological recovery) was also higher in the venetoclax group (66.4% versus 28.3%).

Haematological adverse effects were more common in the venetoclax group. The haematological adverse events of grade 3 or higher most frequently reported in the venetoclax group included thrombocytopenia (45%), neutropenia (42%), and febrile neutropenia (42%). In contrast, these events occurred in 38%, 28%, and 19% of the control group, respectively.