The US Food and Drug Administration (FDA) has approved Venclexta (venetoclax) to treat elderly patients with acute myeloid leukemia (AML).
More specifically, the drug is approved in combination with azacitidine, or decitabine, or low-dose cytarabine (LDAC) for the treatment of newly diagnosed AML in adults who are age 75 years or older, or who have comorbidities that preclude the use of intensive induction chemotherapy. The FDA granted accelerated approval for this indication in 2018 based on preliminary data but has now granted full approval based on data from two phase 3 studies (VIALE-A and VIALE-C) as well as updated data from earlier studies.
In the VIALE-A study, patients taking venetoclax plus azacitidine (n=286) had a median overall survival rate of 14.7 months versus 9.6 months in patients (n=145) taking azacitidine plus placebo group (hazard ratio for death, 0.66; 95% confidence interval, 0.52 to 0.85; P < .001). In the VIALE-C study, the median overall survival for the group receiving venetoclax plus LDAC (n=143) was 7.2 months versus 4.1 months for LDAC plus placebo (n=68). This difference was not statistically significant (P = .114).
AML is a rare and aggressive blood cancer with a low survival rate without aggressive chemotherapy. It generally afflicts the elderly and due to their age and comorbidities, intensive chemotherapy is not always an option.
The most frequent adverse events for patients taking venetoclax plus azacitidine were nausea (44%), diarrhea (43%), febrile neutropenia (42%), musculoskeletal pain (36%), fatigue (31%), and vomiting (30%). Serious adverse reactions were reported in 83% of patients in the venetoclax group and were febrile neutropenia (30%), pneumonia (22%), sepsis (excluding fungal; 19%), and hemorrhage (6%).
Venetoclax is a BCL-2 inhibitor that is also approved to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
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