FDA Approves Frontline Venetoclax for AML

The BCL2 inhibitor venetoclax, in combination with azacitidine or decitabine or low-dose cytarabine, was approved by the FDA for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) who are aged 75 years or older or who have comorbidities that preclude use of intensive induction chemotherapy.

The approval is based on results from two phase II trials: the M14-358 and M14-397 studies.

The M14-358 study evaluated venetoclax in combination with azacitidine (n=67) or decitabine (n=13) in patients with newly diagnosed AML. Twenty-five patients who received venetoclax in combination with azacitidine achieved a CR, with a median duration of remission of 5.5 months (range = 0.4-30 months). Seven of the patients who received venetoclax in combination with decitabine achieved a CR, with a median duration of remission of 4.7 months (range = 1.0-18 months).

The M14-387 study evaluated venetoclax in combination with low-dose cytarabine in patients with newly diagnosed AML (n=61), including patients with previous exposure to a hypomethylating agent for an antecedent hematologic disorder. When combined with low-dose cytarabine, treatment with venetoclax induced CR in 13 patients, with a median observed remission duration of six months (range = 0.03-25 months).

The most common AEs (occurring in ≥30% of patients) associated with venetoclax were nausea, diarrhea, thrombocytopenia, constipation, neutropenia, febrile neutropenia, fatigue, vomiting, peripheral edema, pneumonia, dyspnea, hemorrhage, anemia, rash, abdominal pain, sepsis, back pain, myalgia, dizziness, cough, oropharyngeal pain, pyrexia, and hypotension.

Source: FDA news release, November 23, 2018.

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