The Food and Drug Administration (FDA) has granted accelerated approval to Venclexta (venetoclax; AbbVie and Genentech) for the treatment newly-diagnosed acute myeloid leukemia (AML) in patients aged ≥75 years, or for those ineligible for intensive induction chemotherapy due to comorbidities. It is intended for use in combination with a hypomethylating agent (azacitidine or decitabine) or low-dose cytarabine (LDAC).

The FDA approval was supported by data from the Phase 1b M14-358 dose escalation and expansion study and the Phase 1/2 M14-387 dose escalation and expansion study that included patients with newly-diagnosed AML, including those who were ineligible for intensive induction chemotherapy. In the M14-358 study, the rate of complete remission (CR) was 37% (N=25/67) in the Venclexta + azacitidine group and 54% (N=7/13) in the Venclexta + decitabine group; the rate of complete remission with partial blood count recovery (CRh) was 24% (N=16/67) and 8% (N=1/13) in the Venclexta + azacitidine and Venclexta + decitabine groups, respectively. In the M14-387 study, the data showed the rates of CR and CRh were both 21% (N=13/61) for patients who received Venclexta + LDAC. 

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“Many people with acute myeloid leukemia are unable to tolerate standard intensive chemotherapy, and the Venclexta combination regimens represent important new options for these patients,” said Sandra Horning, MD, chief medical officer and head of Global Product Development.

The conditional approval is based on surrogate endpoints that are reasonably likely to predict a clinical benefit, including CR and CRh. Continued FDA approval for this indication may be contingent upon verification and description of clinical benefit observed in confirmatory trials. 

Venclexta, a BCL-2 inhibitor, is also approved to treat chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, in patients who have received at least one prior therapy, alone or in combination with rituximab. It is available as 10mg, 50mg, and 100mg strength tablets.

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