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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

PubMedNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics2026-08-28

Use of receptor and nonreceptor tyrosine kinase inhibitors and Parkinson disease risk.

Malaty Giovanni R GR, Killion Jordan A JA, Doddamreddy Sai Anmisha SA, Laurido-Soto Osvaldo J OJ et al.

There are currently no disease-modifying therapies for Parkinson disease (PD), although various targets for disease modification have been explored. Expression and binding of receptor tyrosine kinases (rTKs) and nonreceptor tyrosine kinases (nrTKs) have been implicated in PD pathogenesis, and their inhibition is a promisingneuroprotective strategy. This population-based, case-control study of US Medicare beneficiaries included 207,532 incident PD patients and 975,177 comparable, population-based control subjects from 2016 to 2018. This study investigated associations between Medicare Part D prescription fills prior to PD diagnosis for 7 rTK inhibitors (erlotinib, sorafenib, pazopanib, imatinib, sunitinib, nintedanib, and dasatinib) and 4 nrTK inhibitors (nilotinib, ibrutinib, ruxolitinib, and tofacitinib) and PD risk. Logistic regression estimated relative risks (RR) and 95% confidence intervals (CIs) adjusted for age, sex, race, smoking, and healthcare utilization. All medications were inversely associated with incident PD. The rTK inhibitors erlotinib (RR = 0.53, 95% CI: 0.38-0.74), sorafenib (RR = 0.43, 95% CI: 0.25-0.74), imatinib (RR = 0.60, 95% CI: 0.47-0.75), pazopanib (RR = 0.58, 95% CI: 0.39-0.86), nintedanib (RR = 0.66, 95% CI: 0.50-0.88), sunitinib (RR = 0.60, 95% CI: 0.39-0.93), and dasatinib (RR = 0.53, 95% CI: 0.35-0.82) had the strongest inverse associations. Among nrTK inhibitors, ibrutinib (RR = 0.62, 95% CI: 0.51-0.75) had a marked inverse association. Given the chemotherapeutic mechanisms of these medications, and the known cancer-PD inverse relationship, we performed a sensitivity analysis adjusting for relevant cancer subtypes, yielding a similar inverse association. Overall, the use of rTK and nrTK inhibitors was associated with a lower risk of developing PD, although there may be differential impact of this medication class depending on specific inhibition pathways.

PMID 42659831
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PubMedCureus2026-08-27

Effectiveness and Safety of Imatinib and Nilotinib in Chronic Myeloid Leukemia: A Single-Center Study From Southern Morocco.

El Mousadik Mossaab M, El Khayat Houda H, El Hizazi Layla L, Bounar Oumaima O et al.

The prognosis of chronic myeloid leukemia (CML) has been revolutionized following the advent of tyrosine kinase inhibitors (TKIs); however, access to molecular monitoring, resistance mutation screening, and second- and third-generation TKIs remains limited in resource-constrained countries. The aim of this study was to describe the efficacy and safety profile of imatinib and nilotinib in southern Morocco. This was a single-center, retrospective study conducted in the Clinical Hematology Department of the Mohammed VI University Hospital of Agadir over a ten-year period (2014-2024), including 80 patients with CML in the chronic or accelerated phase at diagnosis. All patients received first-line imatinib, with a switch to nilotinib in cases of resistance, relapse, or intolerance. Molecular response was assessed by quantitative reverse transcription polymerase chain reaction (RT-PCR) according to the International Scale (IS), with a favorable response defined as a BCR::ABL1 transcript of ≤ 1% IS at 12 months. Survival was estimated using the Kaplan-Meier method. Among the 80 patients, the mean age was 47.0 ± 17.3 years, with a slight female predominance (M/F sex ratio = 0.9). The Sokal score classified 42.5% of patients as high risk. Under imatinib, a favorable molecular response at 12 months was achieved in 50% of patients, with primary resistance in the other half. As second-line therapy, nilotinib achieved a favorable response in 32.3% of the 31 treated patients. Grade ≥ 3 toxicity was observed in 12.5% of patients on imatinib and 19.4% on nilotinib, with no grade ≥ 3 cardiovascular events. After a median follow-up of 48 months, overall survival (OS) remained at 89.9% beyond 60 months, whereas event-free survival (EFS) under imatinib was shorter (median 24 months). The Sokal score was significantly associated with EFS (p = 0.007). Our series is notable for the young age of patients, a female predominance, and a high proportion of high-risk Sokal scores; a profile consistent with other Moroccan cohorts. Despite a high rate of imatinib resistance, likely related to the unfavorable prognostic profile, diagnostic delay, and limitations in molecular monitoring, OS remained excellent. These findings highlight the value of expanded access to second-generation TKIs as first-line therapy and to resistance mutation screening in our setting.

PMID 42657079
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PubMedHematology reports2026-08-26

The CD70/CD27 Axis in Tyrosine Kinase Inhibitor-Treated Chronic Phase Chronic Myeloid Leukemia Cells Is Not an Achilles Heel.

Cassels Jennifer J, Drotar Mark E ME, MacNeil Alyson A, Moles Michael W MW et al.

Background/Objectives: Revolutionary small molecule, targeted, tyrosine kinase inhibitors (TKIs) in the clinic have engendered the perception that optimal treatment has already been established for diseases like chronic myeloid leukemia (CML). Addressing BCR::ABL1 oncokinase domain mutations in CML is unlikely to redress disease recrudescence owing to persistent leukemia stem cells (LSCs). Therefore, it is necessary to consider an alternative strategic approach: disrupting LSC interactions with the protective microenvironment and/or immune system. The interaction of the TNF-α superfamily member, CD27, with its upregulated ligand, CD70, initiates survival signaling specifically in CML cells when BCR::ABL1 is inhibited by TKIs and thus represents an attractive target. Previous studies modulating the expression of CD27 on LSCs in a mouse model of advanced phase CML were encouraging. In our study, we explored the CD70/CD27 axis as a therapeutic target in early-phase disease. Methods: Primitive CD34+ cells from treatment-naïve chronic phase (CP) CML patients were drug-exposed in an in vitro co-culture system; combination drug treatments of the therapeutic anti-CD70 antibody and TKIs, nilotinib, were assessed in our CP CML murine model. Results: The blockade of the CD70/CD27 axis in combination with TKIs did not result in greater LSC elimination than with nilotinib as a single agent in our CP CML models. Nonetheless, there was an observed reduction in CD70+ cells with combination treatment. Conclusion: Further preclinical study of the antibody as an adjunct in CD70-expressing hematological malignancy is perhaps warranted.

PMID 42645798
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PubMedCancer2026-08-26

Vimseltinib for patients with tenosynovial giant cell tumor: A multicenter, open-label, phase 2 trial.

Stacchiotti Silvia S, Blay Jean-Yves JY, Rutkowski Piotr P, Gelderblom Hans H et al.

Tenosynovial giant cell tumor (TGCT) is a locally aggressive neoplasm caused by dysregulation of the colony-stimulating factor 1 (CSF1) gene. Patients report substantial pain, stiffness, and declining physical function; those whose disease is not amenable to surgery require systemic therapy. Vimseltinib is an oral, switch-control kinase inhibitor of the CSF1 receptor (CSF1R). Here, the authors report safety and efficacy of vimseltinib in patients with TGCT based on prior treatment. Cohort A included patients who did not receive prior specific anti-CSF1/CSF1R agents (n = 46; prior imatinib/nilotinib allowed), and cohort B included patients who received prior specific agents (n = 20). The phase 2 (expansion) portion of this ongoing, multicenter, open-label, phase 1/2 study (NCT03069469) enrolled adults (≥18 years) with histologically-confirmed TGCT not amenable to surgery. Patients received vimseltinib 30 mg twice weekly (recommended phase 2 dose). The primary objectives were to assess safety and antitumor activity; secondary objectives included assessment of active range of motion (ROM) and patient-reported outcomes. Most treatment-emergent adverse events were grade 1/2, and there was no evidence of cholestatic hepatotoxicity or drug-induced liver injury. Best overall response rates were 64% (29 of 45) and 37% (7 of 19) for cohorts A and B after mean follow-up of 23 and 19 months, respectively. Most patients experienced meaningful improvements in active ROM and patient-reported physical function, stiffness, health status, and pain. Vimseltinib had a manageable safety profile, demonstrated durable antitumor activity, and provided functional and symptomatic improvements in patients with TGCT, offering an effective treatment option regardless of previous treatment with anti-CSF1/CSF1R agents.

PMID 42643031
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PubMedCritical reviews in analytical chemistry2026-08-23

A Review on Analytical Insights into Impurity Profiling of Selected Anticancer Agents: Tinibs and Taxanes.

Susmitha Aggarapu A, Rajitha Galla G, Eri Gireesh Kumar GK, Orupalli Kavya K et al.

Impurity profiling is a critical quality and safety requirement for structurally complex anticancer agents. This review critically analyses impurity-profiling literature (1997-2025) for three tinibs, imatinib mesylate (IMM), dasatinib (DST), and nilotinib, and three taxanes, paclitaxel (PTX), docetaxel (DTX), and cabazitaxel (CTX), encompassing 54 analytical studies. Across the compiled dataset, reversed-phase HPLC accounted for 63.6% of methods, UPLC/ ultra-high-performance LC (UHPLC) for 16.4%, LC-MS/High-Resolution Mass Spectrometry (HRMS) for 9.1%, and GC-MS for 5.5%; high-performance thin-layer chromatography (HPTLC)-MS, headspace GC, and SFC appeared in isolated reports. HPLC/UPLC methods demonstrated LODs of 0.005-2 µg mL-1, whereas LC-MS/MS achieved LODs as low as 0.003-0.005 ng mL-1 for genotoxic impurities in tinibs. GC-based methods were especially valuable for volatile impurities and residual sulfonates, with detection in the low-ppb to sub-µg mL-1 range. Tinib impurity profiles are dominated by process-related, oxidative, nitrosamine, and genotoxic species, while taxane profiles are characterized by epimerization products, deacetylated derivatives, side-chain cleavage products, and precursor-related impurities. Regulatory implications under ICH Q3A(R2), Q3B(R2), M7(R2), S9, and Q3C are discussed, including dose-normalised threshold of toxicological concern (TTC) calculations. An impurity-type versus analytical-technique matrix is proposed to guide method selection. Critical analytical gaps are identified, and future directions encompassing green analytical chemistry (GAC), process analytical technology (PAT), and AI-assisted impurity prediction are outlined.

PMID 42634006
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PubMedNeurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2026-08-20

Nilotinib-associated cerebral vasculopathy: temporal shifts in vascular involvement by treatment duration.

Park Jong-Ho JH

PMID 42618702
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