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

PubMedCase reports in oncology2026-10-15

Synchronous Chronic Myeloid Leukemia and Metastatic Papillary Thyroid Carcinoma: A Case Report of a Diagnostic and Therapeutic Challenge.

Ishti Nada SalahAldin NSA, Alkhateeb Nagham N NN, Alhashlamon Khaled K, Hamadneh Shoroq S et al.

The synchronous presentation of chronic myeloid leukemia (CML) and papillary thyroid carcinoma (PTC) is rare and may create important diagnostic and therapeutic challenges. A 36-year-old woman presented with leukocytosis and was diagnosed with chronic-phase CML. During the initial workup, ^18F-FDG PET/CT showed diffuse marrow activity together with mild-to-moderate uptake in selected cervical and right paratracheal lymph nodes. Neck imaging demonstrated a nodular thyroid gland and cervical lymphadenopathy with suspicious features including cystic change and calcifications, raising concern for a synchronous second primary malignancy. Fine-needle aspiration confirmed PTC, and nodal sampling established metastatic PTC. Hematologic stabilization with nilotinib was initiated before surgery. The patient subsequently underwent total thyroidectomy with bilateral neck dissection. Histopathology revealed multifocal PTC with lymphatic invasion, extensive nodal metastases (59/67 lymph nodes positive), and extranodal extension. Final staging was pT2N1bM0, corresponding to AJCC 8th edition stage I. This case highlights the importance of careful evaluation of atypical imaging findings in patients with newly diagnosed CML and the critical role of tissue confirmation in distinguishing leukemic disease from a synchronous second primary malignancy. It also illustrates the complexity of treatment sequencing and the importance of multidisciplinary management in patients with dual primary cancers.

PMID 42877460
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PubMedInternational journal of clinical pharmacology and therapeutics2026-10-13

Tyrosine kinase inhibitor (second-generation)-related pancreatic toxicity in chronic myeloid leukemia patients analyzed using the FAERS database.

Wang Shiyu S, Luo Qingmao Q, He Ning N, Zou Yu Y et al.

To analyze pharmacovigilance data for second-generation tyrosine kinase inhibitor (TKI)-related acute pancreatic toxicity in chronic myeloid leukemia (CML) patients contained in the FAERS database. This observational pharmacovigilance study examined reports for the period Q1 2016 to Q3 2025. Disproportionality analysis was performed using four procedures: i) reporting odds ratio (ROR), ii) proportional reporting ratio (PRR), iii) Bayesian confidence propagation neural network (BCPNN), and iv) multi-item gamma Poisson shrinker (MGPS). Of the 16,057,190 adverse events (AEs) retrieved, 24,803, 31,487, and 11,404 were linked to nilotinib, dasatinib, and bosutinib, respectively. Nilotinib exhibited the strongest and broadest risk signals, including significant associations with pancreatitis (ROR = 10.84), acute pancreatitis, elevated amylase (ROR = 19.94) and lipase (ROR = 14.25), and increased bilirubin (ROR = 11.40), indicating clear pancreatic toxicity with potential hepatic involvement and abdominal symptoms. Bosutinib primarily signaled intense and upper abdominal pain (ROR = 5.04 - 5.44), with weaker signals for pancreatitis. Dasatinib showed the weakest signals, mainly elevated amylase (ROR = 4.79) and lipase (ROR = 8.57). Four algorithms yielded 12 positive signals (17.39% of total). Median age was 52 - 58 years; onset of AEs typically occurred within 30 days of commencing treatment. Pharmacovigilance data in the FAERS database showed strongest pancreatic/gastrointestinal toxicity signals for nilotinib, indicating the need for close monitoring, followed next by bosutinib, with dasatinib exhibiting the weakest signals of the 3 agents. These findings arising from patient therapy serve as a valuable guide for decisions on drug selection, risk management, and future research.

PMID 42867190
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PubMedAlzheimer's & dementia : the journal of the Alzheimer's Association2026-10-12

Tyrosine kinase inhibitor use and risk of neurodegenerative disease: A Swedish population-based cohort study.

Wittström Felix F, Chen Enoch Yi-Tung EY, Blotenberg Iris I, Dahlén Torsten T

Effective disease-modifying therapies remain unavailable for neurodegenerative diseases (NDDs). Tyrosine kinase inhibitors (TKIs) indicated for chronic myeloid leukemia (CML) have been hypothesized to modulate the pathological mechanisms underlying neurodegeneration. Therefore, we aimed to explore the association between initiation of six different TKIs and the subsequent risk of developing NDDs in Swedish nationwide registries (2005-2024). CML patients ≥50 years at diagnosis initiating TKI therapy were identified, alongside five comparators without CML matched on sex, age, and residence at diagnosis. CML cases and comparators were followed from the initiation date of the respective TKI and could contribute follow-up time to multiple TKI cohorts. A composite measure of NDD diagnosis or death due to NDD was the outcome. Hazard ratios (HRs) were estimated using Cox proportional hazards models, with adjustments for calendar year at follow-up initiation, and relevant comorbidities. A total of 1322 CML cases were included in the study. 1188 initiated imatinib (of which 41 experienced an NDD event), 541 dasatinib (15 events), 390 nilotinib (five events), 174 bosutinib, 71 ponatinib, and 41 asciminib. Adjusted hazard ratios comparing each TKI group to their matched comparator populations were 0.98 (95% confidence interval [CI], 0.71 to 1.36) for imatinib users, 1.18 (95% CI, 0.68 to 2.03) for dasatinib users, and 0.37 (95% CI, 0.15 to 0.90) for nilotinib users. Bosutinib, ponatinib, and asciminib users were too few to conduct comparative analyses. Initiation of nilotinib, but not imatinib or dasatinib, was associated with a significantly lower NDD incidence relative to general population comparators.

PMID 42860391
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PubMedFrontiers in oncology2026-10-11

Perioperative care of patients with chronic myeloid leukemia on tyrosine kinase inhibitors.

Binidris Mutasim M, Žáčková Daniela D, Kaddoura Rasha R, Ghasoub Rola R et al.

Patients with chronic myeloid leukemia (CML) are increasingly undergoing surgical procedures as long-term survival improves with tyrosine kinase inhibitor (TKI) therapy. This narrative review aims to synthesize current evidence regarding perioperative risk assessment and management in patients with CML receiving TKIs. A literature search was conducted using structured searches of PubMed, Scopus, Web of Science, and Google Scholar for studies addressing perioperative management, anesthesia, bleeding, thrombosis, wound healing, obesity, pregnancy, and drug interactions in CML. Original studies, retrospective cohorts, case series, case reports, and narrative reviews relevant to perioperative care were included. Available evidence demonstrated that perioperative risk in CML extends beyond routine hematologic parameters and is strongly influenced by TKI-specific toxicities and disease-related factors. Dasatinib showed the greatest association with platelet dysfunction and cytopenias, while nilotinib and ponatinib were linked to increased arterial and cardiovascular risk. Splenomegaly, obesity, bariatric surgery, and pregnancy further altered perioperative risk and TKI pharmacokinetics. Available evidence does not establish validated drug-specific preoperative TKI interruption intervals, and the decision regarding temporary interruption should therefore be individualized according to TKI-specific toxicity, disease status, and procedural risk. The review additionally identified clinically significant CYP3A4-mediated interactions with perioperative anesthetic, antimicrobial, and analgesic agents, suggesting preferential use of propofol-based induction and non-CYP-dependent opioids when feasible. Perioperative management in CML requires individualized multidisciplinary planning integrating hematologic, anesthetic, surgical, and pharmacologic considerations to optimize surgical safety while maintaining disease control.

PMID 42857533
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PubMedThe AAPS journal2026-10-08

Insights into Drug Absorption Impairment Mechanisms in Hepatic Impairment using Physiologically-based Pharmacokinetic Modelling: A Case Study with Nilotinib.

Al-Amiry Santos Laura G LG, Litou Chara C, Rowland Yeo Karen K

Nilotinib is reported to have a reduced maximal drug concentration (Cmax) in patients with hepatic impairment (HI). Given this compound is poorly soluble, highly lipophilic and has a positive food effect, reduced absorption in HI may be due to impaired micelle-mediated solubility resulting from reduced intestinal bile salts. PBPK modelling can be used to test this hypothesis as well as being a diagnostic tool to elucidate other potential mechanisms not captured by the base model. In this study, a mechanistic PBPK model for nilotinib was developed and its performance was evaluated in HI patients. A 'learn-and-confirm' approach was taken, using clinical food effect data to validate the model's mechanistic sensitivity to bile salt-mediated solubility. The final model recovered clinical studies well, with area-under-the-curve (AUC) and Cmax being within 0.8 - 1.25-fold for fasted studies and 1.5-fold for fed/fasted ratios. AUC and Cmax were well recovered in mild HI (within 0.8 - 1.25-fold) and reasonably well recovered in moderate HI (within twofold) but significantly overpredicted in severe HI (> twofold). Bile salt concentrations were reduced across gastrointestinal segments for moderate and severe HI in line with in vitro data. Following twofold and 2.5-fold reductions in bile salts in moderate and severe HI, respectively, sensitivity was minor. Given uncertainty around the fugut, this was reduced to better recover the Cmax (within two-fold). PBPK modelling can be used as a diagnostic tool to evaluate impaired mechanisms in HI.

PMID 42844485
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PubMedJournal of biomolecular structure & dynamics2026-10-06

Significance of BCR-ABL P-loop point mutations (L248R/V) on binding to FDA approved drugs: molecular dynamics simulations and DFT studies.

Indra Neela Y Y, Guruprasad Lalitha L

The BCR-ABL fusion protein with dysregulated tyrosine kinase activity is the cause of chronic myelogenous leukemia (CML). Mutations in the P-loop, gatekeeper, catalytic and activation loop, within its kinase domain leads to drug resistance while treating CML. The mutation driven drug resistance mechanisms at the atomic level in BCR-ABL needs to be unravelled. This work aims to study the structural, dynamical, and functional effects of the P-loop point mutations on BCR-ABL kinase. In this work, the five main FDA approved drugs Imatinib, Dasatinib, Nilotinib, Ponatinib, Bosutinib and one investigational drug Rebastinib were analysed using molecular dynamics (MD), post-MD analyses and quantum chemical (QM) approaches. The P-loop residue L248 and its point mutations (L248R/V) forming direct interactions with the drug were validated. The root-mean-square deviation analysis suggests that Imatinib, Dasatinib and Nilotinib are promising drugs for L248 and L248R/V point mutations, followed by Rebastinib, Ponatinib, and Bosutinib, despite minor variations. The R and C-spines of the wild and mutant systems differ significantly, indicating that the protein backbone is directly affected by mutation. For all the considered wild and mutated amino acid-drug complexes, the interaction energy for L248R complexes is high compared to L248 and L248V complexes. The frontier molecular orbitals, HOMO-LUMO gap, and electrostatic potential maps were analysed. The MD simulations and QM studies suggest that L248 residue and its point mutations (L248R/V) are responsible for high affinity binding of BCR-ABL kinase with the drugs considered for the study.

PMID 42834848
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