Drug Database
NI

nilotinib (Danziten)

✓ Approved

Azurity Pharmaceuticals, Inc. · ABL1 · Small Molecule

What is nilotinib?

nilotinib is a small molecule developed by Azurity Pharmaceuticals, Inc.. It is approved for therapeutic indications via oral (po).

Drug Profile

Brand NamesDanziten
CompanyAzurity Pharmaceuticals, Inc.
Drug ClassSmall Molecule
Molecular TargetABL1, BCR, KIT, PDGFRA
RouteOral (PO)
StatusApproved
Sources: ClinicalTrials.gov, Azurity Pharmaceuticals, Inc.

Mechanism of Action

Molecular Targets

nilotinib acts on 4 molecular targets:

ABL1ABL proto-oncogene 1, non-receptor tyrosine kinase (c-ABL, bcr/abl)
BCRBCR activator of RhoGEF and GTPase (CML, ALL)
KITKIT proto-oncogene, receptor tyrosine kinase (MASTC, CD117)
PDGFRAplatelet derived growth factor receptor alpha (PDGFR-2, CD140A)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

nilotinib is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Chronic myeloid leukaemia✓ Approved

Related Research Articles

PubMedBiochemical and biophysical research communications2026-07-25

Exploring the therapeutic potential of NCX1 in hematological cancers; integration of molecular docking, bioinformatics approaches, and experimental validation.

Misir Sema S, Yaman Serap Ozer SO, Petrović Nina N, Cakmak Isik I et al.

Hematological malignancies are highly heterogeneous diseases characterized by dysregulated signaling pathways and limited durable therapeutic responses. Calcium homeostasis has emerged as a critical regulator of cancer cell fate, yet the role of the sodium/calcium exchanger 1 (NCX1/SLC8A1) in leukemogenesis remains poorly defined. In this study, we comprehensively investigated the biological significance and therapeutic potential of NCX1 across major hematological malignancies by integrating transcriptomic analyses, protein-protein interaction networks, experimental validation, and in silico drug repurposing strategies. NCX1 was highly expressed in HL-60, K-562, and Jurkat cells compared to HaCaT controls. Network analyses revealed that NCX1 interacts with key regulators of calcium signaling, immune response, and signal transduction. In AML and CML patient datasets, a strong positive correlation was observed between NCX1 expression and immune-related pathways, while a negative correlation was observed with translation-related processes. Molecular docking analyses demonstrated that several clinically approved compounds, particularly imatinib and nilotinib, interact with NCX1. Molecular dynamics simulation was performed to evaluate the binding stability and safety of imatinib. Remarkably, the comprehensive analysis showed that imatinib exhibited a stable molecular dynamics profile. All these findings have demonstrated NCX1 as a biologically informative marker of myeloid differentiation and a promising therapeutic weak point within calcium signaling networks in hematological malignancies, providing a rationale for future functional and single-cell validation studies.

PMID 42497834
Read full article →
PubMedCell reports. Medicine2026-07-23

Nilotinib induces immunogenic cuproptosis to potentiate cancer immunotherapy.

Yao Lei L, Zhao Deze D, Meng Yu Y, Liu Yihuang Y et al.

Immunotherapy efficacy is limited by poor tumor immunogenicity and insufficient immune infiltration, highlighting the critical role of inducing immunogenic cell death for improved outcomes. Targeting cuproptosis holds promising therapeutic potential, but its immunogenic and capacity to stimulate anti-tumor immunity to potentiate immunotherapy remains unknown. Through screening of 240 Food and Drug Administration (FDA)-approved anti-tumor drugs, we identify nilotinib as a potent cuproptosis inducer in cancer cells, especially at physiologically relevant copper concentrations. Mechanistically, nilotinib-copper elicits cuproptosis in vitro and in vivo by repressing the copper exporter ATP7A via ETV1 to drive mitochondrial copper accumulation and activating mitogen-activated protein kinase (MAPK) pathway through ERK2 phosphorylation. Preclinically, nilotinib-copper induces immunogenic cuproptosis and enhances anti-PD-1 efficacy across multiple melanoma models, including B16F10 tumor-bearing mice, Braf/Pten-driven spontaneous melanoma mice, and PBMC-humanized mice. Clinically, the ATP7ALow/MAPKHigh signature correlates with improved immunotherapy response and prolonged overall survival. This work establishes nilotinib as a clinically tractable, non-ionophore immunogenic cuproptosis inducer capable of activating anti-tumor immunity to potentiate immunotherapy.

PMID 42486098
Read full article →
PubMedEuropean journal of haematology2026-07-23

Diabetic Patients With Newly Diagnosed Chronic Myeloid Leukemia: TKI Choice and Early Adverse Events. A Real-Life Survey Within the Italian Chronic Myeloid Leukemia Network.

Russo S S, Capodanno I I, Tiribelli M M, Miggiano M C MC et al.

Limited data are available regarding patients with newly diagnosed chronic myeloid leukemia (CML) and concomitant diabetes mellitus (DM). Hence, we retrospectively analyzed 2149 patients with newly diagnosed chronic phase (CP) CML to compare clinical characteristics, choice of frontline therapy, early adverse events, and molecular responses between patients with or without DM. Among this cohort of 2149 patients, 241 patients (11.2%) had DM at CML diagnosis. Diabetic patients were older (median age 69.6 vs. 58.2 years, p < 0.001), showed a higher Sokal/ELTS risk and had more comorbidities, resulting in more frequent polypharmacy. Imatinib was frequently selected as first-line treatment in diabetic patients (71% vs. 53%, p < 0.001), whereas nilotinib was more common in nondiabetics (6.2% vs. 31.9%). Discontinuation by the 12th month occurred in 24.2% of diabetic patients versus 19.4% of nondiabetics (p = 0.031), mainly for hematologic toxicity. Diabetic patients showed a lower incidence of major molecular response (MMR) at the 12th month (55.9% vs. 68.1%, p = 0.002). Among diabetic patients, a trend toward better molecular responses was observed in patients receiving metformin (p = 0.068). In conclusion, DM represents a clinically meaningful comorbidity in newly diagnosed CML-CP, and seems to define a subgroup with distinct features, influencing both treatment decisions and early outcomes.

PMID 42486531
Read full article →
PubMedSao Paulo medical journal = Revista paulista de medicina2026-07-22

Treatment patterns and clinical outcomes in chronic myeloid leukemia treated with second-line nilotinib-association of molecular milestones and prognostic scores with deep molecular response: a real-world retrospective cohort study.

Ćojbašić Irena I, Tijanić Ivan I, Ćojbašić Žarko Ž

Real-world data on the treatment patterns and clinical outcomes of second-line nilotinib use in chronic myeloid leukemia (CML) remain limited, particularly regarding predictors of deep molecular response (DMR). To characterize treatment patterns and long-term clinical outcomes in patients with CML receiving second-line nilotinib and to evaluate whether early molecular milestones combined with baseline prognostic scores predict DMR. Retrospective, single-center real-world study conducted at a tertiary academic center between 2011 and 2025. The data of 45 patients with CML receiving second-line nilotinib because of imatinib resistance or intolerance were analyzed. Treatment patterns, including duration and reasons for switching therapy, were assessed. Baseline prognostic scores (Sokal, European Treatment and Outcome Study [EUTOS], EUTOS Long-Term Survival [ELTS], Hammersmith) were calculated, and molecular responses were assessed according to the European LeukemiaNet (ELN) criteria. Predictors of DMR were examined using univariate and multivariate logistic regression, and survival outcomes were estimated using Kaplan-Meier analysis. The median duration of nilotinib administration was 67.3 months, with 60% of patients achieving a best response of DMR and 46.7% maintaining a sustained DMR. Univariate analysis showed that a favorable Hammersmith score (p = 0.001), low ELTS risk (p = 0.028), and attainment of ELN-defined milestones at 6 (p = 0.020) and 12 months (p = 0.006) were significantly associated with DMR. Multivariate analysis demonstrated that the 12-month ELN milestone (p = 0.008) and favorable Hammersmith score (p = 0.023) were independently associated with a DMR. Patients achieving a DMR showed a nonsignificant trend toward improved overall survival. In this real-world cohort, a DMR and favorable survival outcomes were achieved when nilotinib was administered as second-line treatment. Integrating baseline prognostic scores with molecular milestones enables individualized monitoring and supports a sustained DMR as a marker of durable disease control.

PMID 42484162
Read full article →
PubMedThe oncologist2026-07-21

Renal Dysfunction in Patients with CML on Long-Term First-Line TKI Therapy: A Single-Center Analysis.

Wang Sanmei S, Lei Yutian Y, Liu Simeng S, Zhang Bo B et al.

with life expectancies of patients with chronic myeloid leukemia (CML) now approaching those of the general population under tyrosine kinase inhibitor (TKI) therapy, understanding long-term treatment-related toxicities becomes paramount. Although imatinib's nephrotoxic potential is established, the renal safety profiles of second-generation TKIs-dasatinib, nilotinib, and flumatinib-remain incompletely characterized. this single-center retrospective study analyzed renal function trajectories in 348 patients with CML receiving first-line imatinib, dasatinib, nilotinib, or flumatinib. Renal function was assessed using serum creatinine and estimated glomerular filtration rate (eGFR). Mixed-effects models incorporating treatment group, time, and their interaction as fixed effects were used to formally compare eGFR trajectories across treatment groups. Chronic renal adverse events (CRAE) and acute kidney injury (AKI) were also evaluated. formal comparison of eGFR trajectories across treatment groups revealed a significant time × treatment interaction, indicating differential renal function changes among the four TKIs. Significant serum creatinine elevation and eGFR decline were observed in imatinib and flumatinib groups, contrasting with stable creatinine and divergent eGFR patterns in dasatinib (slight increase) and nilotinib (decline) groups. The incidence of chronic renal adverse events varied substantially across cohorts-12.3%(imatinib), 2.6%(nilotinib), 4.3%(dasatinib), and 9.6%(flumatinib). Notably, among three documented acute kidney injury cases, all occurred during flumatinib treatment. Furthermore, treatment-free remission achievement did not confer significant renal function recovery, suggesting potential irreversibility of established renal damage. TKIs exhibit differential renal safety profiles, underscoring the critical importance of individualized TKI selection based on baseline renal status and implementation of rigorous renal monitoring protocols throughout treatment duration.

PMID 42478751
Read full article →
PubMedBMC neurology2026-07-21

Tyrosine kinase inhibitor-associated cerebral vasculopathy with a distinct non-enhancing vessel wall phenotype: a case report.

Yang Jeong Eun JE, Jeon Jae Ho JH, Do Young Rok YR, Yun Jaeseob J

Tyrosine kinase inhibitors (TKIs) are associated with adverse vascular events, including cerebrovascular stenosis. However, most reported cases rely on luminal imaging, and the arterial wall characteristics of TKI-associated vasculopathy remain insufficiently characterized. A 47-year-old man with chronic myeloid leukemia developed recurrent transient ischemic attacks during long-term exposure to multiple tyrosine kinase inhibitors, including sequential treatment with nilotinib and ponatinib. Neurovascular imaging revealed extensive multifocal steno-occlusive lesions involving both intracranial and extracranial arteries, accompanied by severe hemodynamic compromise. High-resolution vessel wall MRI demonstrated diffuse circumferential wall thickening with negative remodeling in both distal internal carotid arteries and eccentric thickening along the anterior walls of the bilateral M1 segments of the middle cerebral artery. Notably, no definite mural enhancement was observed. Following discontinuation of ponatinib and transition to alternative therapy, cerebral perfusion improved at two months, and the patient remained clinically stable without recurrent ischemic events. This case illustrates a potentially distinct vessel wall thickening pattern in TKI-associated vasculopathy, providing information beyond luminal stenosis. The absence of definite mural enhancement and clinical and hemodynamic improvement after treatment modification suggest a drug-related vascular component. Vessel wall imaging may aid in the evaluation of this condition and inform management strategies.

PMID 42477610
Read full article →

+2921 more articles available with a free account

Sign up free to view all articles →

Ask about nilotinib