Last reviewed · How we verify
VEN
Venetoclax is a selective inhibitor of the anti-apoptotic protein BCL-2 that promotes cancer cell death by restoring apoptosis.
Venetoclax is a selective inhibitor of the anti-apoptotic protein BCL-2 that promotes cancer cell death by restoring apoptosis. Used for Chronic lymphocytic leukemia (CLL), treatment-naïve and relapsed/refractory, Acute myeloid leukemia (AML), newly diagnosed in patients ≥75 years or unfit for intensive chemotherapy, Small lymphocytic lymphoma (SLL), relapsed/refractory.
At a glance
| Generic name | VEN |
|---|---|
| Sponsor | Janssen Research & Development, LLC |
| Drug class | BCL-2 inhibitor |
| Target | BCL-2 |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Venetoclax binds directly to BCL-2 and displaces pro-apoptotic proteins (BAX and BAK), allowing them to permeabilize the mitochondrial outer membrane and trigger the intrinsic apoptotic pathway. This mechanism is particularly effective in hematologic malignancies where BCL-2 is overexpressed or where cells are dependent on BCL-2 for survival.
Approved indications
- Chronic lymphocytic leukemia (CLL), treatment-naïve and relapsed/refractory
- Acute myeloid leukemia (AML), newly diagnosed in patients ≥75 years or unfit for intensive chemotherapy
- Small lymphocytic lymphoma (SLL), relapsed/refractory
Common side effects
- Neutropenia
- Thrombocytopenia
- Anemia
- Diarrhea
- Nausea
- Tumor lysis syndrome
- Infection
Key clinical trials
- BLAST MRD AML-2: BLockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia 2- A Randomized Phase 2 Study of Anti-PD-1 Pembrolizumab in Combination With Azacitidine and Venetoclax as Frontline Therapy in Unfit Patients With Acute Myeloid Leukemia (PHASE2)
- A Multicenter RCT of "3+7" vs Venetoclax + CACAG in Newly Diagnosed Mid/High-Risk AML Patients (PHASE2)
- A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) (PHASE3)
- Front-line VenObi Combination Followed by Ven or VenZan Combination in Patients With Residual Disease: a MRD Tailored Treatment for Young Patients With High-risk CLL (PHASE2)
- Safety and Efficacy of Venetoclax With Escalating Doses of Omacetaxine in Patients With Acute Myeloid Leukemia Safety and Efficacy of Venetoclax With Escalating Doses of Omacetaxine in Patients With Acute Myeloid Leukemia (AML) (PHASE1)
- De-Escalation Study Evaluating Venetoclax and Azacitidine Discontinuation in AML Responding Patients (PHASE2)
- Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML (PHASE3)
- A Study of Bleximenib in Combination With Acute Myeloid Leukemia (AML) Directed Therapies (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- VEN CI brief — competitive landscape report
- VEN updates RSS · CI watch RSS
- Janssen Research & Development, LLC portfolio CI