marked.bio

FLT3 inhibitor · small molecule

quizartinib

6 active trials across 2 of our biomarkers, targeting FLT3-ITD. 2 active approvals for a biomarker on marked.bio.

Also known as AC220, Vanflyta.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
quizartinib with standard induction and consolidation chemotherapy
FDA, 2023
newly diagnosed FLT3-ITD-positive, with induction and consolidation chemotherapy and as maintenance FDA approval 20/07/2023; boxed warning for QT prolongation (REMS). Companion test LeukoStrat CDx FLT3. Source
EU (EMA), 2023
newly diagnosed FLT3-ITD-positive, with cytarabine/anthracycline induction and cytarabine consolidation, then maintenance EU marketing authorisation 06/11/2023. Source
Vanflyta 		FLT3 mutation (ITD/TKD) AML Approved 2023 Approved 2023

QuANTUM-First vs placebo + chemotherapy, 268 vs 271 patients
CR 55% vs 55%OS HR 0.78 (0.62-0.98)

Stopped for side effects 20%Fatal 10%

Trial details and side effects
Patients
newly diagnosed FLT3-ITD AML, age 18-75 (NCT02668653)
Most common side effects
febrile neutropenia 44%, diarrhoea 42%, mucositis 38%, nausea 34%, abdominal pain 30%, sepsis 30%, neutropenia 29%, headache 28% (265 patients)
Notes
FDA approval summary (2023); median CR duration 38.6 vs 12.4 months; published median OS 31.9 vs 15.1 months. Side effects: Vanflyta US label, section 6 (QuANTUM-First, with chemotherapy).
Source
FDA approval summary

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
FLT3 mutation (ITD/TKD) 6 16 Phase 2
NPM1 mutation 1 1 Phase 1

Sponsors

Industry sponsors leading active biomarker-selected trials of quizartinib.

  1. Daiichi Sankyo 2
  2. Kura Oncology 1

Trials

6 of 16 trials shown

  1. Testing the Investigational Medication Combination of Azacitidine, Venetoclax, and Quizartinib Compared to the Usual Treatment of Cytarabine, Daunorubicin, and Quizartinib for Patients With FLT3-ITD Mutant Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)

    Not yet recruiting Phase 2 National Cancer Institute (NCI) Starts 2027Primary completion 2027-12 NCT07862517

    azacitidinecytarabinedaunorubicinquizartinib FDA EUvenetoclax

    Patient must have newly diagnosed acute myeloid leukemia (AML) with FLT3 ITD mutation as assessed by central myeloMATCH testing with variant allele frequency (VAF) ov…

    FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia

  2. Evaluation of Two Dose Levels of Quizartinib as Maintenance in FLT3-ITD (+) Acute Myeloid Leukemia Patients in Complete Remission

    Recruiting Phase 2 Daiichi Sankyo Started 2025Primary completion 2028-06 NCT06824168

    quizartinib FDA EU

    Participant has confirmed FLT3-ITD-positive (≥0.05 SR or ≥5% VAF) activating mutation from initial diagnosis in bone marrow …

    FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia, Leukemia

  3. CHIP-AML22/Quizartinib: Quizartinib + Chemotherapy in Newly Diagnosed Pediatric FLT3-ITD+ and NPM1wt AML Patients

    Recruiting Phase 2 Princess Maxima Center for Pediatric Oncology Started 2024Primary completion 2028-06 NCT06262438

    quizartinib FDA EUetoposideDexrazoxanemitoxantronecytarabinemethotrexatedaunorubicinfludarabine

    FLT3-ITD+ and wild-type NPM1:

    FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia in Children

  4. Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm With FLT3 or CBL Mutations

    Active, not recruiting Phase 1/2 M.D. Anderson Cancer Center Started 2020Primary completion 2027-12 NCT04493138

    azacitidinequizartinib FDA EU

    Detectable FLT3-ITD mutation in bone marrow and/or peripheral blood, or presence of CBL exon 8 or 9 deletions…

    FLT3 mutation (ITD/TKD)Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Myeloproliferative Neoplasm, Recurrent Chronic Myelomonocytic Leukemia

  5. Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the Blood

    Active, not recruiting Phase 1/2 Daiichi Sankyo Started 2018Primary completion 2027-05 NCT03793478

    quizartinib FDA EUfludarabinecytarabineIntrathecal (IT) triple chemotherapy prophylaxisetoposide

    Has presence of the FLT3-ITD activating mutation in bone marrow or peripheral blood as defined in the protocol

    FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia

  6. A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML

    Recruiting Phase 1 Kura Oncology Started 2023Primary completion 2030-04 NCT05735184

    ziftomenibvenetoclaxazacitidinedaunorubicincytarabinequizartinib FDA EU

    Those intending treatment with intensive chemotherapy in Arm C should be NPM1-m and FLT3-ITD+ with an allelic ratio ≥0.05 and eligible for FLT3-targeted treatment

    FLT3 mutation (ITD/TKD)NPM1 mutationAcute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation, Refractory AML, AML With Mutated NPM1