Nilotinib
Small molecule targets ABL1, CSF1R, DDR1, KIT, PDGFRA via inhibition
Regulatory milestones approvals, filings & regulatory actions · 12 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed chronic myeloid leukemia 1 milestone | |||||
| Filed | US (FDA) | Tasigna | chronic myeloid leukemia | 2025-08-19 | “has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its product candidate XS003 (nilotinib)” xspraypharma.com ↗ “the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for XS003 (nilotinib) for review” xspraypharma.com ↗ “Tasigna supplemental New Drug Application (sNDA) was accepted by FDA and granted priority review.” novartis.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (9) | US (FDA) | TASIGNA | — | 2010-06-17 – 2021-09-23 | fda.gov ↗ |
| Approved | EU (EMA) | Tasigna | — | 2007-11-19 | europa.eu ↗ |
| Approved | US (FDA) | TASIGNA | — | 2007-10-29 | fda.gov ↗ |
Trials 145 · started per year
News releases announcing trial results or a regulatory action · 24
| Date | Issuer | Release |
|---|---|---|
| 2026-09-23 | Xspray Pharma AB | Results Xspray Pharma to present CML data supporting differentiation of Dasynoc and Nilopki xspraypharma.com ↗
The data indicate that Nilopki avoids the food-induced increase in plasma exposure seen with crystalline nilotinib. |
| 2026-06-04 | Xspray Pharma AB | Regulatory Xspray Pharma Receives CRL from U.S. FDA for Nilopki™ xspraypharma.com ↗
Xspray Pharma AB (publ) (Nasdaq Stockholm: XSPRAY), a pharmaceutical company leveraging its proprietary HyNap™ technology platform to develop improved versions of marketed protein kinase inhibitors (PKIs) for cancer treatment, has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) for Nilopki™, an optimized formulation of nilotinib. |
| 2026-06-01 | Novartis Pharmaceuticals | Results Scemblix® continued to show superior efficacy and favorable safety and tolerability profile at week 144 in newly diagnosed CML novartis.com ↗
The longer-term data showed a progressively larger difference in MMR rates favorable to Scemblix vs. SoC TKIs, vs. imatinib and vs. 2G TKIs 1 . |
| 2026-04-14 | Xspray Pharma AB | Regulatory FDA accepts trade name Nilopki® for Xspray’s drug candidate XS003 (nilotinib) xspraypharma.com ↗
The US Food and Drug Administration (FDA) has accepted Nilopki® as the proprietary name for Xspray Pharma's drug candidate XS003 (nilotinib). |
| 2025-10-21 | Xspray Pharma AB | Regulatory Xspray Pharma: FDA accepts New Drug Application for XS003 (nilotinib) for the treatment of CML – PDUFA date set for June 18, 2026 xspraypharma.com ↗
Xspray Pharma AB (publ) (Nasdaq Stockholm: XSPRAY) announces that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for XS003 (nilotinib) for review under the 505(b)(2) regulatory pathway. |
| 2025-08-19 | Xspray Pharma AB | Regulatory Xspray Pharma Submits XS003 to the FDA – The Company’s Second Product Candidate from the HyNap Platform xspraypharma.com ↗
Xspray Pharma (Nasdaq Stockholm: XSPRAY) has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its product candidate XS003 (nilotinib) for the treatment of chronic myeloid leukemia (CML). |
| 2025-07-11 | Xspray Pharma AB | Results XSpray Pharma achieves significant milestone – demonstrating bioequivalence with absorption advantages compared to Tasigna xspraypharma.com ↗
The results confirm formal bioequivalence between XS003 and Tasigna in terms of systemic exposure (AUC and Cmax) – despite the XS003 dose being less than half that of the reference drug. |
| 2025-01-20 | Xspray Pharma AB | Results Xspray Pharma reports strong interim data for product candidate XS003 – confirms plan to apply for market approval in H1 2025 xspraypharma.com ↗
Xspray Pharma AB (publ) today presents interim data from a food interaction study with the nilotinib product candidate (XS003). |
| 2024-07-09 | Xspray Pharma AB | Results Xspray Pharma’s XS003 Study shows Matching Bioavailability to Tasigna at more than a 50% Lower Dose xspraypharma.com ↗
In the announced study, XS003 demonstrates matching bioavailability to Tasigna, with a 50% reduced dose. |
| 2024-05-31 | Novartis Pharmaceuticals | Results Novartis Scemblix® Phase III data first to show superior efficacy with a favorable safety and tolerability profile vs. standard-of-care TKIs in adults with newly diagnosed CML novartis.com ↗ |
| 2024-05-15 | Novartis Pharmaceuticals | Results Novartis highlights pioneering innovation in CML with data from Scemblix® Phase III ASC4FIRST study in newly diagnosed patients at ASCO and EHA novartis.com ↗
The primary results from ASC4FIRST, a pivotal Phase III study of Scemblix ® (asciminib) versus standard of care tyrosine kinase inhibitors (imatinib, nilotinib, dasatinib, and bosutinib) in newly diagnosed patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) will be shared at the ASCO official Press Program and at the EHA Plenary Session. |
| 2024-01-08 | Novartis Pharmaceuticals | Results Novartis Scemblix® shows superior major molecular response (MMR) rates vs. standard‐of‐care TKIs in Phase III trial for newly diagnosed patients with chronic myeloid leukemia novartis.com ↗ |
| 2023-11-21 | Xspray Pharma AB | Results Xspray Pharma’s XS003 Achieves Superior Bioavailability Milestone, Matching TASIGNA® at Reduced Dosage xspraypharma.com ↗
today announced that XS003, its amorphous, non-crystalline formulation of nilotinib, has demonstrated bioavailability within the 80-125% range to TASIGNA® following oral administration with significantly lower dose. |
| 2020-12-29 | Xspray Pharma AB | Regulatory Xspray Pharma’s HyNap-Nilo receives FDA Orphan Drug Designation for the treatment of Chronic Myeloid Leukemia xspraypharma.com ↗
The FDA’s Orphan Drug Designation (ODD) has been granted to Xspray Pharma for nilotinib oral formulation intended for the treatment of chronic myeloid leukemia. |
| 2019-06-14 | Novartis Pharmaceuticals | Results Novartis continues to innovate in CML with long-term treatment-free remission results following Tasigna® use and promising combination data with investigational compound asciminib (ABL001) novartis.com ↗
Long-term follow-up data from the ongoing, pivotal open-label ENESTfreedom and ENESTop trials showed sustained treatment-free remission (TFR) after stopping frontline and second-line Tasigna (nilotinib) therapy in eligible adult patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the chronic phase (CP). |
| 2018-06-02 | Novartis Pharmaceuticals | Results New Novartis data presented at ASCO find nearly half of CML patients treated with Tasigna® remain in remission almost three years after stopping therapy novartis.com ↗
Results from the open-label Phase II trials, ENESTop and ENESTfreedom, show sustained TFR in patients treated with both front-line and second-line Tasigna ® (nilotinib) therapy. |
| 2018-03-22 | Novartis Pharmaceuticals | Regulatory Novartis drug Tasigna® approved by FDA to treat children with rare form of leukemia novartis.com ↗
In the United States, Tasigna is now indicated for the treatment of adult and pediatric patients one year of age or older with newly diagnosed Ph+ CML-CP. |
| 2017-12-22 | Novartis Pharmaceuticals | Regulatory Novartis drug Tasigna® is approved by FDA as first and only CML therapy with Treatment-free Remission data in its label novartis.com ↗
Novartis announced today that the US Food and Drug Administration (FDA) approved the inclusion of Treatment-free Remission (TFR) data in the Tasigna ® (nilotinib) US product label. |
| 2017-11-20 | Novartis Pharmaceuticals | Regulatory Novartis drug Tasigna® (nilotinib) secures EU approval for first and second-line treatment of Ph+ CML-CP in children novartis.com ↗
Tasigna is the only second-generation tyrosine kinase inhibitor (TKI) currently approved in the European Union (EU) for the treatment of Ph+ CML-CP in children. |
| 2017-06-23 | Novartis Pharmaceuticals | Results Novartis data shows half of eligible Ph+ CML-CP patients remain in Treatment-free Remission nearly two years after stopping Tasigna® novartis.com ↗
More than 90% of Ph+ CML-CP patients in ENESTfreedom and ENESTop who stopped Tasigna and were in TFR at 48 weeks remained in TFR at 96 weeks |
| 2017-06-06 | Novartis Pharmaceuticals | Regulatory Novartis drug Tasigna receives EU approval for inclusion of Treatment-free Remission (TFR) data in product label novartis.com ↗
Novartis announced today that the European Commission (EC) has approved the inclusion of Treatment-free Remission (TFR) data in the Tasigna ® (nilotinib) Summary of Product Characteristics (SmPC). |
| 2016-12-05 | Novartis Pharmaceuticals | Results Novartis data shows Treatment-free Remission rates are consistently above 50% regardless of reason for switch to Tasigna® from Glivec® novartis.com ↗ |
| 2016-06-04 | Novartis Pharmaceuticals | Results Novartis data show more than 50 percent of eligible Ph+ CML patients maintain Treatment-free Remission (TFR) after stopping Tasigna® novartis.com ↗
ENESTop met primary endpoint with 57.9% of eligible patients who had switched to Tasigna from Glivec ® maintaining TFR for 48 weeks after treatment cessation[2] |
| 2015-06-13 | Novartis Pharmaceuticals | Results Novartis presents new data from large European study reinforcing the benefit of first-line Tasigna® in newly-diagnosed patients with CML novartis.com ↗
Novartis today announced results from the Phase 3b ENEST1st study in over 1,000 patients with newly-diagnosed, BCR-ABL positive chronic myeloid leukemia (CML), confirming the benefits of first-line Tasigna treatment seen in earlier trials. |
All press releases naming this drug 26 releases
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 180 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | nilotinib | “Nilotinib (formerly AMN107), a highly selective BCR-ABL tyrosine kinase inhibitor, is effective in patients with Philadelphia chromosome-positive chronic myelogenous leukemia...” PMID 17715389 ↗ Aug 2007171“Doses are halved/reduced per protocol: Flumatinib 400/200 mg QD, Imatinib 200 mg QD, Nilotinib 300 mg QD, Dasatinib 50 mg QD, or Olverembatinib 20 mg QD, based on pre-entry therapy.” NCT07839988 ↗ “Low dose tyrosine kinase inhibitor (lowTKI) (dasatinib 50 mg daily or imatinib 300 mg daily or nilotinib 300 mg daily) at investigators discretion” NCT05143840 ↗ “Imatinib was given at a dose of 400mg/d or 600mg/d, dasatinib at a dose of 100mg/d or 140mg/d, and nilotinib at a dose of 400mg twice daily.” NCT01883219 ↗ “In this study, the efficacy and safety of nilotinib (AMN107) as a third-line therapy for patients with GISTs was evaluated.” PMID 21456006 ↗ Mar 2011 “Trial Evaluating Nilotinib as Treatment for Newly Diagnosed CML Patients in Accelerated Phase.” NCT01605981 ↗ “The BCR-ABL TKIs that will be used include imatinib, dasatinib, nilotinib or bosutinib.” NCT03610971 ↗ “multiple doses of nilotinib on the pharmacokinetics and metabolism of midazolam” NCT01223898 ↗ “The study drug is nilotinib (Tasigna®).” NCT01261429 ↗ “Mitoxantrone, Nilotinib, Oxaliplatin” NCT02788201 ↗ “Nelarabine Nilotinib Paclitaxel” NCT02551718 ↗ |
| Known as | AMN107 | “Nilotinib (formerly AMN107), a highly selective BCR-ABL tyrosine kinase inhibitor, is effective in patients with Philadelphia chromosome-positive chronic myelogenous leukemia...” PMID 17715389 ↗ Aug 200713“Nilotinib (Tasigna®, code number AMN107) was first approved in 2007 for use in Philadelphia chromosome positive CML in the chronic or accelerated phase in patients resistant or...” NCT01201538 ↗ “A 24 Week, Randomized, Double Blind, Multicenter, Placebocontrolled Efficacy, Safety, Tolerability and PK Trial of Nilotinib (Tasigna®, AMN107) in Pulmonary Arterial Hypertension (PAH)” NCT01179737 ↗ “Nilotinib (Tasigna®, AMN107, Novartis, Switzerland) is approved by U.S. Food and Drug Administration (FDA) and is well tolerated for CML treatment at oral doses of 600-800mg daily.” NCT02954978 ↗ “Preclinical in vitro studies have shown that nilotinib (AMN107), a new BCR-ABL tyrosine kinase inhibitor, is more potent than imatinib against CML cells by a factor of 20 to 50.” PMID 16775235 ↗ Jun 2006 “Imatinib Mesylate (Glivec) as Maintenance Therapy After Cytogenetic Response With Nilotinib (AMN107, Tasigna) First Line in Newly Diagnosed Chronic Myelogenous Leukemia” NCT01316250 ↗ “Nilotinib (Tasigna®, AMN107, Novartis, Switzerland) is approved by the FDA and is well tolerated for CML treatment at oral doses of 600-800mg daily.” NCT04002674 ↗ “An Open-label Phase I/II (Proof of Concept) Trial of an Combination of Nilotinib (AMN 107) and RAD001 in Patients With Acute Myeloid Leukemia” NCT00762632 ↗ “Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas” NCT01275586 ↗ “Drug: Nilotinib (AMN107)” NCT00519090 ↗ “Drug: Nilotinib (AMN107)” NCT00785785 ↗ |
| Known as | Nilotinib (AMN107): Tasigna | “Nilotinib (AMN107): Tasigna” NCT01400074 ↗ |
| Known as | Nilotinib BE | “This study will investigate the safety and efficacy of a Tyrosine Kinase Inhibitor (TKI) called Nilotinib BE (bioequivalent) in individuals with Early Alzheimer's disease (EAD).” NCT05143528 ↗ |
| Known as | Nilotinib Hydrochloride Monohydrate | “Drug: Nilotinib Hydrochloride Monohydrate” NCT05554341 ↗ |
| Known as | Tasigna | “Nilotinib (Tasigna®, code number AMN107) was first approved in 2007 for use in Philadelphia chromosome positive CML in the chronic or accelerated phase in patients resistant or...” NCT01201538 ↗12“We evaluated the efficacy and safety of 1-year treatment with nilotinib (Tasigna®) in patients with autosomal dominant spinocerebellar ataxia (ADSCA) and the factors associated...” PMID 39009709 ↗ Jul 2024 “A 24 Week, Randomized, Double Blind, Multicenter, Placebocontrolled Efficacy, Safety, Tolerability and PK Trial of Nilotinib (Tasigna®, AMN107) in Pulmonary Arterial Hypertension (PAH)” NCT01179737 ↗ “Nilotinib (Tasigna®, AMN107, Novartis, Switzerland) is approved by U.S. Food and Drug Administration (FDA) and is well tolerated for CML treatment at oral doses of 600-800mg daily.” NCT02954978 ↗ “Nilotinib (Tasigna®): orally BID given continuously on Days 1- 28” NCT01887522 ↗ “Nilotinib (Tasigna ®), capsules of 150 mg” NCT02201459 ↗ “Nilotinib (Tasigna ®), capsules of 150 mg” NCT03942094 ↗ “The study drug is nilotinib (Tasigna®).” NCT01261429 ↗ “Nilotinib 150 MG [Tasigna]” NCT03228303 ↗ “Nilotinib (Tasigna)” NCT01166139 ↗ “Tasigna (nilotinib)” NCT07138378 ↗ |
| Known as | XS003 | “XS003 (nilotinib)” NCT07138378 ↗ |
| Action | Inhibit | “potently inhibits activated forms of KIT” PMID 26002753 ↗ May 2015 |
| Modality | Small molecule | “novel aminopyrimidine” NCT00633295 ↗ |
| Route | Oral | “nilotinib PO” NCT00036738 ↗ |
| Target | KIT | “Nilotinib is a tyrosine kinase inhibitor that potently inhibits activated forms of KIT.” PMID 26002753 ↗ May 2015 |