Last reviewed 2026-04-23 · How we verify

Venetoclax 400

Venetoclax is a small molecule inhibitor of the BCL-2 protein, which is involved in apoptosis regulation.

Venetoclax is a small molecule inhibitor of the BCL-2 protein, which is involved in apoptosis regulation. Used for Chronic lymphocytic leukemia (CLL), Acute myeloid leukemia (AML).

At a glance

Mechanism of action

By inhibiting BCL-2, venetoclax promotes apoptosis in cancer cells, leading to cell death. This mechanism is particularly effective in treating certain types of leukemia, such as chronic lymphocytic leukemia (CLL).

Approved indications

• Chronic lymphocytic leukemia (CLL)
• Acute myeloid leukemia (AML)

Common side effects

• Neutropenia
• Thrombocytopenia
• Anemia
• Infections
• Fatigue

Key clinical trials

• VAG Versus Standard Chemotherapy With FLT3 Inhibitor in Adult Patients With FLT3-Mutated AML (PHASE3)
• Venetoclax, Azacitidine and Liposomal Mitoxantrone for Newly Diagnosed AML (NA)
• Duvelisib and Venetoclax in Relapsed or Refractory CLL or SLL or RS (PHASE1, PHASE2)
• Venetoclax, Azacitidine, and Mitoxantrone Hydrochloride Liposome Versus Idarubicin and Cytarabine in Newly Diagnosed AML (PHASE3)
• Aclarubicin Plus With Azacitidine and Venetoclax in the Treatment of Acute Myeloid Leukemia (PHASE1, PHASE2)
• A Multicenter RCT of "3+7" vs Venetoclax + CACAG in Newly Diagnosed Mid/High-Risk AML Patients (PHASE2)
• Dose Escalation/ Expansion Study of CA-4948 as Monotherapy in Patients With Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS) (PHASE1, PHASE2)
• Study of Venetoclax Combined With Azacitidine Regimen in Newly Diagnosed T-ALL Patients (PHASE2)

Primary sources

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