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drugsProductApproved2016🇪🇺🇬🇧🇯🇵🇨🇳🇦🇺

Venetoclax

A pill that removes the survival shield from leukaemia cells, enabling chemotherapy-free, time-limited treatment for CLL.

CLL (fixed-duration with obinutuzumab, CLL14; with ibrutinib), AML with azacitidine in unfit patients (VIALE-A). AbbVie/Genentech. Tumour lysis syndrome managed by ramp-up dosing.

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Venetoclax · 2D coordinates (no PubChem conformer)
PubChem
Mechanism, step by step
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1.Venetoclax occupies the BH3-binding groove of BCL-2

Modality
Small-molecule BCL-2 inhibitor
Mechanism
BH3-mimetic selective BCL-2 inhibitor.
Brand / code
Venclexta
Dosing & schedule
Route
Oral
Schedule
CLL: 5-week ramp-up 20 → 50 → 100 → 200 → 400 mg daily, then 400 mg daily (fixed 12 months with obinutuzumab); AML: 3-day ramp to 400 mg daily with azacitidine (100 mg with posaconazole)
Dose modifications
TLS prophylaxis (hydration, allopurinol; hospitalisation for high tumour burden); reduce with CYP3A inhibitors
Monitoring
Chemistry and uric acid at each ramp step; blood counts

Source: www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications. Doses are for orientation; the current label governs.

Medicare
Part D (self-administered)

Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026).

Commercial insurance
covered with prior authorisation

Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy.

Assistance programmes

Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.

Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.

NICE recommendedNICE TA663 · 2020SMC: accepted
Appraised for
With obinutuzumab for untreated CLL
Notes
Monotherapy for 17p/TP53 CLL: TA487 (2017, CDF). With rituximab for relapsed CLL: TA561 (2019). With azacitidine or low-dose cytarabine for AML unsuitable for intensive chemotherapy: TA765 (2022). Fixed-duration venetoclax-ibrutinib appraised 2024-25.
NHS England
Routinely funded for the appraised indication (or via managed access)

Sources: NICE TA663 · SMC advice: venetoclax. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

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  1. 11 Apr 2016ApprovalUS

    CLL with 17p deletion after ≥1 therapy: first BCL-2 inhibitor source

  2. 8 Jun 2018ApprovalUS

    CLL/SLL with rituximab (MURANO) source

  3. 21 Nov 2018ApprovalUS

    Newly diagnosed AML in unfit patients with azacitidine/decitabine/LDAC (accelerated) source

  4. 15 May 2019ApprovalUS

    First-line CLL with obinutuzumab (CLL14) source

  5. 16 Oct 2020ApprovalUS

    Full approval in AML (VIALE-A) source

Approvals

RegionYearIndication
US2016CLL with 17p deletion
US2018AML with azacitidine/decitabine/LDAC in unfit patients

Safety

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Toxicity profile
Adverse event
Neutropenia
CLL14: ~53% grade 3-4 with obinutuzumab
Diarrhoea
Nausea
Anaemia
Upper respiratory infection
Tumour lysis syndrome
Prevented by ramp-up; rare with prophylaxis

Events listed without rates were not read from a primary source; see the label. Blank cells mean the figure was not sourced, not that it is zero.

Cost & access

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Cost & access
CountryReimbursement
United StatesMedicare Part D (oral); commercial plans per formulary, often with prior authorisation
United KingdomNICE: recommended in CLL (TA487, TA561, TA663) and AML with azacitidine (TA765)

List prices are manufacturer or Medicare figures where publicly disclosed; net prices after rebates are usually lower. Reimbursement changes; check the payer.

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-06
562 studies42 recruiting82 Phase 222 Phase 3
Search “Venetoclax” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Venetoclax
intervention: Venetoclax
Open on ClinicalTrials.gov →

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Key papers

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rctNew England Journal of Medicine 2025changed practice
AMPLIFY: fixed-duration acalabrutinib plus venetoclax, with or without obinutuzumab, versus chemo-immunotherapy in fit CLL patients

AMPLIFY delivered the first all-oral, fixed-duration doublet for front-line CLL and supported its approval, giving fit patients a way to avoid both chemotherapy and years of continuous BTK inhibitor. It does not settle whether a doublet or triplet is best, or how AV compares with venetoclax-obinutuzumab. Patients with TP53 aberration were excluded and still need different strategies.

rctNew England Journal of Medicine 2022changed practice
AGILE: ivosidenib plus azacitidine for newly diagnosed IDH1-mutated AML in patients unfit for intensive chemotherapy

AGILE showed that for the roughly 6-10% of AML patients with an IDH1 mutation, a targeted doublet produces survival in the range of two years, an outcome previously unimaginable in unfit patients. Ivosidenib-azacitidine is approved and is one option alongside venetoclax-azacitidine for these patients. Which regimen, or triplet, is best for IDH1-mutated disease has not been settled by a randomised trial.

rctNew England Journal of Medicine 2020changed practice
VIALE-A: venetoclax plus azacitidine for older adults with acute myeloid leukaemia who cannot have intensive chemotherapy

VIALE-A turned a palliative regimen into one that produces remission in two-thirds of older AML patients and is now the reference treatment for anyone not fit for intensive chemotherapy. It shifted the field towards lower-intensity targeted combinations and opened the door to adding FLT3, IDH and menin inhibitors to the backbone. Cure remains uncommon and most patients relapse within two years.

rctNew England Journal of Medicine 2019changed practice
CLL14: one year of venetoclax plus obinutuzumab instead of chemo-immunotherapy in older, less fit CLL patients

CLL14 established the first chemotherapy-free, fixed-duration regimen for front-line CLL and made MRD-guided thinking mainstream in the disease. Patients get a year of treatment and then a treatment-free period rather than indefinite therapy. The choice today is between fixed-duration venetoclax combinations and continuous BTK inhibitors, with no proven survival difference.

rctNew England Journal of Medicine 2018changed practice
MURANO: two years of venetoclax plus rituximab versus chemo-immunotherapy in relapsed CLL

MURANO made fixed-duration venetoclax the standard for relapsed CLL and showed that stopping therapy after a deep response is safe for most patients. It also established MRD at end of treatment as a practical guide to who is likely to stay in remission. Retreatment with venetoclax at relapse appears feasible.

Latest papers

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Literature trend986 papers in the last 12 months+23% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Venetoclax" OR ABSTRACT:"Venetoclax" OR TITLE:"Venclexta" OR ABSTRACT:"Venclexta") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Venetoclax, not a curated reading list.

Connected

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cancers

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technologies

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targets

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companies

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institutions

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pathways

5

terms

9

trials

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pairings

5

ideas

3

people

3

bottlenecks

2

key papers

5