Drug · Small Molecule

Nilotinib

CI-DRUG-00000404Explore in graph →NCIt C48375 CHEMBL255863 CIViC Approved

Regulatory

Approvals (31)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

openfdaRegulatory19 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	Chronic Myeloid Leukemia, Philadelphia Chromosome Negative, BCR-ABL1 Positive	Adult patients with chronic phase (CP) and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib.	approved	May 29, 2026	openfda
US FDA	—	Adult patients with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase.	approved	May 29, 2026	openfda
US FDA	Chronic Myeloid Leukemia, Philadelphia Chromosome Negative, BCR-ABL1 Positive	Adult patients with chronic phase (CP) and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib.	approved	Feb 19, 2025	openfda
US FDA	—

Health Canada records are DIN-level: one row per marketed product (brand, strength, form). The Drug Product Database does not publish indications, so no cancer is stated for these rows, and a cancelled or dormant DIN is the status of that one product — not a withdrawal of the molecule.

Data updated 18 hours agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 4	16	7	27	123	31	CA, EU, US	Oct 29, 2007	Jul 13, 2001
Leukemia	Approved	Phase 4	9	3	16	75	7	EU, US	Oct 29, 2007	Jul 13, 2001
Malignant Breast Neoplasm	Phase2	Phase 2	1	1	0	2	0	—	—	Oct 13, 2022
Malignant Ovarian Neoplasm	Phase2	Phase 2	1	1	0	2	0	—

Curated evidence

Clinical evidence (66)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–50 of 66 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
ABL1 T315I + ABL1 Fusion6
Dasatinib + Imatinib

Clinical trials

Trials with this intervention (134)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–50 of 134 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT05564377	Targeted Therapy Directed by Genetic Testing in Treating Patients With Locally Advanced or Advanced Solid Tumors, The ComboMATCH Screening Trial	recruiting	Phase 2	2,900	National Cancer Institute (NCI)NIH	2	Sep 28, 2026	clinicaltrials
NCT03516279	Pembrolizumab and Dasatinib, Imatinib Mesylate, or Nilotinib in Treating Patients With Chronic Myeloid Leukemia and Persistently Detectable Minimal Residual Disease

Molecule

Record

Kind: Small Molecule
Mechanism: Tyrosine-protein kinase ABL inhibitor; Bcr/Abl fusion protein inhibitor
Targets: 2 genes
UNII: F41401512X
Aliases: 4-Methyl-3-((4-(3-pyridinyl)-2-pyrimidinyl)amino)-N-(5-(4-methyl-1H-imidazol-1-yl)-3-(trifluoromethyl)phenyl)benzamide, AMN 107, AMN107, AMN 107 Base Form, APO-NILOTINIB, CAVHANZA, DANZITEN, Nilotinib, Nilotinib Accord, Nilotinib Hydrochloride Dihydrate, Nilotinib Hydrochloride Monohydrate, NSC-747599, PMS-NILOTINIB, REDDY-NILOTINIB, TASIGNA

Data updated 21 hours agoSource updated unknown

Identifiers

Codes (23)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

System	Code	Label
ATC (WHO)	L01EA03	NILOTINIB
DIN (Health Canada)	02315874

FLT3 Mutation2
Nilotinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	3	submitted	EID5575 Of the 14 AML patients receiving nilotinib, 80% achieved complete remission or complete remission with incomplete platelet recovery. Genetic testing revealed that 27% of these individuals conferred a … (full text at CIViC) PMID 28720666 · Shen et al., 2017 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	submitted	EID5574 Compared to non-treated control cells, MV4-11 cells (FLT3 mutated cell line) that were exposed to nilotinib (30 uM) showed a 58% reduction in colony numbers.Further evidence to support cell death incl… (full text at CIViC) PMID 28720666 · Shen et al., 2017 · Open in CIViC	civic

ABL1 Fusion3
Nilotinib	B Lymphoblastic Leukemia/LymphomaCURATED_BROADER	Predictive	A	Supports Sensitivity Response	4	submitted	EID11288 In this phase 1 dose-escalation study, 119 patients with imatinib-resistant, Ph-positive CML or acute lymphoblastic leukemia (ALL) to receive nilotinib orally at doses of 50 mg, 100 mg, 200 mg, 400 mg… (full text at CIViC) PMID 16775235 · Kantarjian et al., 2006 · Open in CIViC	civic
Dasatinib + NilotinibSubstitutes	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	A	Supports Sensitivity Response	4	accepted	EID261 The use of nilotinib and dasatinib, second-generation BCR-ABL targeted therapies, has been effective in patients resistant to imatinib, leading to their FDA-approval. PMID 20537386 · An et al., 2010 · Open in CIViC	civic
Bafetinib + Dasatinib + NilotinibSubstitutes	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	D	Supports Sensitivity Response	3	accepted	EID7838 In an in vitro study, six CML cell lines expressing the BCR-ABL1 fusion protein showed sensitivity to nilotinib (IC50: 24.3-84.5 nM), dasatinib (IC50: 1.5-10.9 nM), and bafetinib (IC50: 11.8-87.7 nM).… (full text at CIViC) PMID 18191450 · Deguchi et al., 2008 · Open in CIViC	civic

ABL1 Fusion2
Dasatinib + Imatinib + Nilotinib + PonatinibSubstitutes	B Lymphoblastic Leukemia/LymphomaCURATED_BROADER	Predictive	D	Does Not Support Sensitivity Response	2	accepted	EID9150 A 22-year-old male with B-cell acute lymphoblastic leukemia was treated with induction therapy. FISH using a BCR-ABL1 probe demonstrated 50-80 extrachromosomal copies of ABL1; FISH using probes to the… (full text at CIViC) PMID 23175662 · Eyre et al., 2012 · Open in CIViC	civic
Dasatinib + NilotinibSequential	B Lymphoblastic Leukemia/Lymphoma, BCR-ABL1-Like	Predictive	C	Supports Sensitivity Response	3	submitted	EID13329 A 21-year-old patient with refractory B-cell acute lymphoblastic leukemia (B-ALL) had a NUP214::ABL1 fusion identified by clinical next-generation sequencing failed multiple prior treatment regimens, … (full text at CIViC) PMID 37933765 · Shumock et al., 2023 · Open in CIViC	civic

ABL1 E255K + ABL1 Fusion3
Nilotinib	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	D	Supports Resistance	2	accepted	EID2662 In an in vitro study, Ba/F3 cell line stably expressing the E255K mutation in the ABL1 portion of the BCR-ABL1 fusion protein was associated with resistance to nilotinib treatment (IC50: 6.69-fold), a… (full text at CIViC) PMID 19075254 · Redaelli et al., 2009 · Open in CIViC	civic
Dasatinib + Imatinib Mesylate + NilotinibSubstitutes	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	D	Supports Resistance	2	accepted	EID6194 In an in vitro study, stochastic mutations were introduced into the ABL1 region of a stably expressed p210 BCR-ABL fusion in Ba/F3 cells using N-ethyl-N-nitrosourea mutagenesis. Resulting clones were … (full text at CIViC) PMID 16772610 · Bradeen et al., 2006 · Open in CIViC	civic
Imatinib Mesylate + NilotinibSubstitutes	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	C	Supports Resistance	3	accepted	EID6232 In this open-label, phase II registration trial, patients that were imatinib resistant or intolerant underwent nilotinib monotherapy. Within 12 months of nilotinib therapy, there was no significant di… (full text at CIViC) PMID 19652056 · Hughes et al., 2009 · Open in CIViC	civic

ABL1 E255V + ABL1 Fusion3
Nilotinib	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	D	Supports Resistance	3	accepted	EID2850 In an in vitro study, a Ba/F3 cell line stably expressing the E255V mutation in the ABL1 portion of the BCR-ABL1 fusion protein was associated with resistance to nilotinib treatment (IC50: 10.31-fold… (full text at CIViC) PMID 19075254 · Redaelli et al., 2009 · Open in CIViC	civic
Imatinib Mesylate + NilotinibSubstitutes	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	C	Supports Resistance	3	accepted	EID6971 In this open-label, phase II registration trial, patients that were imatinib resistant or intolerant underwent nilotinib monotherapy. Within 12 months of nilotinib therapy, there was no significant di… (full text at CIViC) PMID 19652056 · Hughes et al., 2009 · Open in CIViC	civic
〃	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	D	Supports Resistance	2	accepted	EID2851 In an in vitro study, a Ba/F3-p210BCR-ABL1 cell line was ENU-mutated and exposed to graded concentrations of imatinib, nilotinib or dasatinib to identify resistance mutations. The E255V mutation in th… (full text at CIViC) PMID 16772610 · Bradeen et al., 2006 · Open in CIViC	civic

ABL1 F359C + ABL1 Fusion3
Imatinib Mesylate + NilotinibCombination	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	D	Supports Resistance	2	accepted	EID6239 In an in vitro study, stochastic mutations were introduced into the ABL1 region of a stably expressed p210 BCR-ABL fusion in Ba/F3 cells using N-ethyl-N-nitrosourea mutagenesis. Resulting clones were … (full text at CIViC) PMID 16772610 · Bradeen et al., 2006 · Open in CIViC	civic
Imatinib Mesylate + NilotinibSubstitutes	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	C	Supports Resistance	3	accepted	EID6233 In this open-label, phase II registration trial, patients that were imatinib resistant or intolerant underwent nilotinib monotherapy. Within 12 months of nilotinib therapy, there was no significant di… (full text at CIViC) PMID 19652056 · Hughes et al., 2009 · Open in CIViC	civic
〃	Chronic Myeloid Leukemia, BCR-ABL1 PositiveALIAS	Predictive	D	Supports Resistance	2	accepted	EID2854 In an in vitro study, a Ba/F3-p210BCR-ABL1 cell line was ENU-mutated and exposed to graded concentrations of imatinib, nilotinib or dasatinib to identify resistance mutations. The F359C mutation in th… (full text at CIViC) PMID 16772610 · Bradeen et al., 2006 · Open in CIViC	civic