Last reviewed 2026-04-20 · How we verify

Vanflyta (QUIZARTINIB)

Vanflyta works by blocking the macrophage colony-stimulating factor 1 receptor, which is involved in the growth and survival of cancer cells.

Vanflyta (quizartinib) is a small molecule therapy originally developed by Daiichi Sankyo Inc, currently owned by the same company. It targets the macrophage colony-stimulating factor 1 receptor and has been FDA-approved since 2023 for the treatment of acute myeloid leukemia with FMS-like tyrosine kinase-3 mutation and acute myeloid leukemia. Vanflyta is a patented medication with no generic manufacturers available. Key safety considerations include its potential side effects, which may require monitoring. Vanflyta is a promising treatment option for patients with specific types of acute myeloid leukemia.

At a glance

Mechanism of action

Quizartinib is small molecule inhibitor of the receptor tyrosine kinase FLT3. Quizartinib and its major active metabolite AC886 bind to the adenosine triphosphate (ATP) binding domain of FLT3 with comparable affinity, and both had 10-fold lower affinity towards FLT3-ITD mutation compared to FLT3 in binding assay. Quizartinib and AC886 inhibited FLT3 kinase activity, preventing autophosphorylation of the receptor, thereby inhibiting downstream FLT3 receptor signaling and blocking FLT3-ITD-dependent cell proliferation. Quizartinib showed antitumor activity in mouse model of FLT3-ITD-dependent leukemia.

Approved indications

• Acute myeloid leukemia with FMS-like tyrosine kinase-3 mutation
• Acute myeloid leukemia, disease

Boxed warnings

• WARNING: QT PROLONGATION, TORSADES DE POINTES, and CARDIAC ARREST VANFLYTA prolongs the QT interval in a dose- and concentration-related manner [see Clinical Pharmacology (12.2) ] . Prior to VANFLYTA administration and periodically, monitor for hypokalemia or hypomagnesemia, and correct deficiencies. Perform ECGs to monitor the QTc at baseline, weekly during induction and consolidation therapy, weekly for at least the first month of maintenance, and periodically thereafter [see Dosage and Administration (2.3) and Warnings and Precautions (5.1) ]. Torsades de pointes and cardiac arrest have occurred in patients receiving VANFLYTA. Do not administer VANFLYTA to patients with severe hypokalemia, severe hypomagnesemia, or long QT syndrome [see Contraindications (4) and Warnings and Precautions (5.1) ] . Do not initiate treatment with VANFLYTA or escalate the VANFLYTA dose if the QT interval corrected by Fridericia's formula (QTcF) is greater than 450 ms [see Dosage and Administration (2.3) and Warnings and Precautions (5.1) ] . Monitor ECGs more frequently if concomitant use of drugs known to prolong the QT interval is required [see Dosage and Administration (2.3) and Warnings and Precautions (5.1) ] . Reduce the VANFLYTA dose when used concomitantly with strong CYP3A inhibitors, as they may increase quizartinib exposure [see Dosage and Administration (2.4) and Warnings and Precautions (5.1) ] . Because of the risk of QT prolongation, VANFLYTA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the VANFLYTA REMS [see Warnings and Precautions (5.2) ] . WARNING: QT PROLONGATION, TORSADES DE POINTES, and CARDIAC ARREST See full prescribing information for complete boxed warning. VANFLYTA prolongs the QT interval. ( 12.2 ) Prior to VANFLYTA administration and periodically, perform electrocardiograms (ECGs), monitor for hypokalemia or hypomagnesemia, and correct deficiencies. ( 2.3 , 5.1 ) Torsades de pointes and cardiac arrest have occurred in patients receiving VANFLYTA. Do not administer VANFLYTA to patients with severe hypokalemia, severe hypomagnesemia, or long QT syndrome. ( 4 , 5.1 ) Do not initiate treatment with VANFLYTA or escalate the VANFLYTA dose if the QT interval corrected by Fridericia's formula (QTcF) is greater than 450 ms. ( 2.3 , 5.1 ) Monitor ECGs more frequently if concomitant use of drugs known to prolong the QT interval is required. ( 2.3 , 5.1 ) Reduce the VANFLYTA dose when used concomitantly with strong CYP3A inhibitors, as they may increase quizartinib exposure. ( 2.4 , 5.1 ) VANFLYTA is available only through a restricted program called the VANFLYTA Risk Evaluation and Mitigation Strategy (REMS). ( 5.2 )

Common side effects

• Febrile neutropenia
• Thrombocytopenia
• Sepsis
• Diarrhea
• Mucositis
• Nausea
• Abdominal pain
• Vomiting
• Upper respiratory tract infection
• Fungal infection
• Herpesvirus infection
• Potassium decreased

Drug interactions

• Strong CYP3A Inhibitors
• Strong or Moderate CYP3A Inducers
• QT Interval Prolonging Drugs

Key clinical trials

• Trial to Compare Efficacy and Safety of Chemotherapy/Quizartinib vs Chemotherapy/Placebo in Adults FMS-like Tyrosine Kinase 3 (FLT3) Wild-type Acute Myeloid Leukemia (AML) (PHASE2)
• Azacytidine, Venetoclax Plus Minus Quizartinib for First Line Older/Unfit AML Patients (VENP-A-QUI) (PHASE3)
• 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 (PHASE1)
• Quizartinib or Placebo Plus Chemotherapy in Newly Diagnosed Patients With FLT3-ITD Negative AML (PHASE3)
• FLT3-ITD Targeted Therapy in Fit AML Patients (PHASE2,PHASE3)
• Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia (PHASE1)
• Quizartinib, Decitabine, and Venetoclax in Treating Participants With Untreated or Relapsed Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome (PHASE1,PHASE2)
• Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome (PHASE1,PHASE2)

Patents

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Vanflyta CI brief — competitive landscape report
• Vanflyta updates RSS · CI watch RSS
• Daiichi Sankyo Inc portfolio CI