In the EU, venetoclax monotherapy is indicated for the treatment of chronic lymphocytic leukemia (CLL) in the presence of 17p deletion or TP53 mutation in adults who are unsuitable for or have failed a B cell receptor pathway inhibitor and for the treatment of CLL in the absence of 17p deletion or TP53 mutation in adults who have failed both chemoimmunotherapy and a B cell receptor pathway inhibitor.

[7] For this purpose it is used with azacitidine, decitabine, or low-dose cytarabine for newly diagnosed adults who are age 75 years or older, or those with other health problems where intensive chemotherapy cannot be used.

[7] Steady state maximum concentration with low-fat meal conditions at the 400 mg once daily dose was found to be 2.1 ± 1.1 μg/mL.

[7] Additionally, while using venetoclax it is not recommended to use other drugs which contain CYP3A inhibitors (i.e.: erythromycin, ciprofloxacin, diltiazem, dronedarone, fluconazole, verapamil).

[7] In 2015, the United States Food and Drug Administration (FDA) granted the breakthrough therapy designation to venetoclax for people with CLL or SLL who have relapsed, become intolerant to, or refractory to previous treatment.

[7] The efficacy of venetoclax was tested in a single-arm clinical trial of 106 participants with CLL who have a 17p deletion and who had received at least one prior therapy.

[8] In June 2018, the FDA granted regular approval to venetoclax for people with CLL or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy.

[17] In November 2018, in the United States, venetoclax was approved in combination with azacitidine or decitabine or low-dose cytarabine 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.

[18] Accelerated approval was based on two open-label non-randomized trials in participants with newly diagnosed AML who were >= 75 years of age or had comorbidities that precluded the use of intensive induction chemotherapy.

[18] Study M14-358 (NCT02203773) was a non-randomized, open-label clinical trial of venetoclax in combination with azacitidine (n=67) or decitabine (n=13) in newly diagnosed participants with AML.

[18] In a phase 3 study of azacitidine and venetoclax in untreated acute myeloid leukemia not eligible for standard induction chemotherapy, the addition of venetoclax to azacitidine resulted in an improvement in median overall survival (14.7 months versus 9.6 months) and improved complete remission rates.

[9] The trial also demonstrated statistically significant improvements in rates of minimal residual disease negativity (less than one CLL cell per 104 leukocytes) in bone marrow and peripheral blood.

[21]: 7–8 On 13 June 2020 at the European Hematology Association (EHA) annual congress, AbbVie and Roche announced the results of a Phase III trial that showed a 34 percent reduction in the risk of death in AML patients who were ineligible for intensive chemotherapy treated with venetoclax plus azacitidine compared to azacitidine plus placebo.