FDA grants venetoclax approval for the treatment of newly diagnosed acute myeloid leukemia

On 21 November 2018, the US Food and Drug Administration (FDA) granted accelerated approval to venetoclax, an oral inhibitor of BCL-2, in combination with azacitidine, decitabine or low-dose cytarabine (LDAC) for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.¹

The approval for venetoclax was based on data obtained from two phase Ib/II open-label non-randomized trials (M14-358 and M14-387 study) in patients with newly-diagnosed AML who were age 75 years or older or had comorbidities that precluded the use of intensive induction chemotherapy. In the M14-358, the combination of venetoclax with azacitidine (n = 67) or decitabine (n = 13) led to a complete remission (CR) rate of 37% and a CR with partial hematological recovery (CRh) rate of 24%.² More results of this study are reported here.

In the M14-387 study, the combination of venetoclax with LDAC demonstrated a clinically durable activity in elderly patients with newly diagnosed AML who are eligible for intensive chemotherapy. Venetoclax and LDAC combination led to a CR rate of 21% and a CRh rate of 21% in elderly patients in newly diagnosed AML patients who are ineligible for intensive chemotherapy.³ More results from this study are reported here.

The FDA approved this indication for venetoclax under accelerated approval based on response rates. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. About the approval for venetoclax, Daniel A. Pollyea, from the University of Colorado, Denver, US, said “many of my patients are ineligible for the intensive treatment for AML, which typically involves intensive chemotherapy. Only a minority of AML patients older than 60 are able to tolerate the standard chemotherapy required to achieve optimal results. Having a new medicine to treat AML is encouraging for my patients and their families. Venetoclax approval is a true breakthrough for AML patients ineligible for intensive chemotherapy."¹