VANFLYTA: Uses, Dosage & Side Effects
A selective FLT3 inhibitor used with chemotherapy for the treatment of FLT3-ITD positive acute myeloid leukemia (AML) in adults
VANFLYTA (quizartinib) is a prescription oral tyrosine kinase inhibitor used in combination with standard chemotherapy for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) that carries a FLT3 internal tandem duplication (ITD) mutation. VANFLYTA works by selectively blocking the FLT3-ITD receptor, stopping the uncontrolled proliferation of leukemic blasts and helping immature blood cells mature into healthy cells. Treatment may continue as maintenance therapy after bone marrow transplantation. VANFLYTA requires careful monitoring including regular ECG tests and blood work, as QT prolongation and myelosuppression are significant safety considerations.
Quick Facts: VANFLYTA
Key Takeaways
- VANFLYTA (quizartinib) is a targeted FLT3 inhibitor specifically designed for adults with newly diagnosed FLT3-ITD positive acute myeloid leukemia, used in combination with standard induction and consolidation chemotherapy.
- Your doctor will perform a genetic test on your cancer cells to confirm the FLT3-ITD mutation before prescribing VANFLYTA, as the drug is only effective in patients with this specific mutation.
- Regular ECG monitoring is mandatory because VANFLYTA can cause QT prolongation, a potentially serious heart rhythm disturbance. ECGs are performed weekly initially and then at reduced frequency.
- Significant drug interactions exist with strong CYP3A inhibitors (azole antifungals, certain antibiotics) which increase VANFLYTA levels, and CYP3A inducers (rifampicin, St. John’s Wort) which decrease its effectiveness.
- VANFLYTA must not be used during pregnancy or breastfeeding. Women must use effective contraception during treatment and for at least 7 months after the last dose; men for at least 4 months after the last dose.
What Is VANFLYTA and What Is It Used For?
VANFLYTA contains the active substance quizartinib, a type of anti-cancer medication belonging to the class known as protein kinase inhibitors. Specifically, quizartinib is a highly selective type II inhibitor of the FMS-like tyrosine kinase 3 (FLT3) receptor. It is prescribed in combination with standard chemotherapy for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) that has a specific mutation in the FLT3 gene called an internal tandem duplication (FLT3-ITD). This mutation is one of the most common genetic alterations found in AML, occurring in approximately 25–30% of all newly diagnosed cases, and is associated with a more aggressive disease course and poorer prognosis when treated with chemotherapy alone.
To understand how VANFLYTA works, it helps to first understand the disease it treats. Acute myeloid leukemia is a type of blood cancer that originates in the bone marrow, where blood cells are produced. In healthy individuals, immature blood cells (called blasts) in the bone marrow develop and mature into functional white blood cells, red blood cells, and platelets. In AML, a series of genetic mutations cause these blast cells to lose their ability to mature properly. Instead, they multiply uncontrollably, flooding the bone marrow and bloodstream with large numbers of abnormal, immature cells that cannot perform the functions of normal blood cells. This leads to life-threatening complications including severe infections (due to lack of functional white blood cells), anemia (due to reduced red blood cell production), and bleeding problems (due to insufficient platelets).
The FLT3 receptor is a protein found on the surface of blood cell precursors that normally helps regulate their growth and development. When the FLT3 gene acquires an internal tandem duplication mutation, the resulting FLT3-ITD receptor becomes constitutively active, meaning it is permanently switched on even without its normal activating signal. This continuous, unregulated signaling drives the leukemic cells to proliferate rapidly and resist the normal programmed cell death (apoptosis) mechanisms that would normally eliminate defective cells. Patients with FLT3-ITD positive AML historically have had higher relapse rates and shorter overall survival compared with FLT3-wildtype AML when treated with chemotherapy alone.
Quizartinib works by selectively binding to and blocking the kinase activity of the FLT3-ITD receptor. As a type II inhibitor, quizartinib binds to the inactive (DFG-out) conformation of the FLT3 kinase, effectively locking it in its off state. This blocks the downstream signaling pathways that promote leukemic cell survival and proliferation, including the RAS/MAPK, PI3K/AKT, and STAT5 pathways. By shutting down these abnormal growth signals, VANFLYTA achieves two important therapeutic effects: it slows or stops the division of leukemic blasts, and it allows the remaining immature cells to resume their normal differentiation process, maturing into functional blood cells.
Before starting treatment with VANFLYTA, your doctor will order a diagnostic test to analyze your cancer cells for changes in the FLT3 gene. This test specifically looks for FLT3-ITD mutations to confirm that VANFLYTA is an appropriate treatment for your particular form of AML. Only patients whose leukemia cells carry the FLT3-ITD mutation will benefit from VANFLYTA, as the drug is specifically designed to target this molecular abnormality.
The clinical efficacy of VANFLYTA was demonstrated in the pivotal phase III QuANTUM-First trial, a large, randomized, double-blind, placebo-controlled study that enrolled 539 patients with newly diagnosed FLT3-ITD positive AML aged 18 to 75 years. Patients were randomized to receive either quizartinib or placebo in combination with standard induction and consolidation chemotherapy, followed by maintenance therapy. The study met its primary endpoint, demonstrating a statistically significant improvement in overall survival for patients receiving quizartinib compared with placebo. The median overall survival was 31.9 months in the quizartinib group versus 15.1 months in the placebo group, representing a clinically meaningful improvement for this patient population.
Treatment with VANFLYTA can be continued after allogeneic hematopoietic stem cell transplantation (bone marrow transplant) as maintenance therapy, once the patient has recovered sufficiently. This is an important feature of the treatment strategy, as maintenance therapy after transplantation can help reduce the risk of disease relapse, which remains a significant concern in FLT3-ITD positive AML. VANFLYTA was first approved by the U.S. Food and Drug Administration (FDA) in July 2023 and subsequently by the European Medicines Agency (EMA), representing an important advance in the targeted treatment of AML.
The FLT3-ITD mutation is found in approximately 25–30% of adult AML cases. It causes the FLT3 receptor to be permanently activated, driving uncontrolled growth of leukemic cells. A simple blood or bone marrow test can identify this mutation. If you have FLT3-ITD positive AML, you are eligible for targeted therapy with VANFLYTA in combination with standard chemotherapy, which has been shown to significantly improve overall survival compared with chemotherapy alone.
What Should You Know Before Taking VANFLYTA?
Contraindications
There are specific situations in which VANFLYTA must not be used. These absolute contraindications exist because using the medication in these circumstances could pose serious health risks:
- Allergy to quizartinib: Do not take VANFLYTA if you are allergic to quizartinib or any of the other ingredients in the tablets (listed in the composition section below). If you think you may be allergic, consult your doctor before starting treatment.
- Congenital long QT syndrome: VANFLYTA must not be used in patients born with a heart condition called congenital long QT syndrome. This is a rare inherited disorder that affects the electrical activity of the heart, causing abnormal heart rhythms. Because VANFLYTA itself can prolong the QT interval, using it in patients who already have this condition would significantly increase the risk of dangerous cardiac arrhythmias.
- Breastfeeding: You must not breastfeed while taking VANFLYTA because it is not known whether quizartinib passes into breast milk, and the drug could potentially harm a nursing infant.
Warnings and Precautions
VANFLYTA can cause QT prolongation, a serious condition affecting the heart's electrical activity. This can lead to dangerous irregular heart rhythms, including cardiac arrest and ventricular fibrillation. Your doctor will perform ECG tests before and during treatment to monitor your heart rhythm. Contact your doctor immediately if you experience dizziness, lightheadedness, fainting, or an irregular heartbeat.
Before starting VANFLYTA, discuss the following with your healthcare provider:
- Heart conditions: Inform your doctor if you have or have ever had any heart problems, including arrhythmia (abnormal heart rhythm), heart attack within the past 6 months, heart failure, untreated angina (chest pain), or uncontrolled high blood pressure. These conditions may increase your risk of QT prolongation-related complications while taking VANFLYTA.
- Electrolyte imbalances: Tell your doctor if you have been informed that you have low potassium or magnesium levels in your blood (hypokalemia or hypomagnesemia). Low electrolyte levels can further increase the risk of QT prolongation. Your doctor will monitor your electrolyte levels regularly and more frequently if you develop diarrhea or vomiting.
- QT-prolonging medications: If you are taking other medications known to prolong the QT interval, inform your doctor, as the combination with VANFLYTA may further increase this risk.
- Strong CYP3A inhibitors: Tell your doctor if you are taking strong CYP3A inhibitors, as these can significantly increase VANFLYTA blood levels and the risk of side effects.
- Signs of infection: If you have or have had fever, cough, chest pain, shortness of breath, fatigue, or pain during urination, tell your doctor, as these may be signs of infection that could worsen during treatment.
Monitoring During Treatment
Your doctor will conduct several regular monitoring tests throughout your treatment with VANFLYTA to ensure the medication is working safely and effectively:
Blood tests: Regular blood tests will be performed to monitor your blood cell counts (white blood cells, red blood cells, and platelets), as VANFLYTA in combination with chemotherapy commonly causes myelosuppression (reduced bone marrow function). Your doctor will also check your electrolyte levels (including sodium, potassium, magnesium, chloride, and bicarbonate), and will monitor these more frequently if you experience diarrhea or vomiting, as these conditions can further deplete electrolytes.
ECG monitoring: Before and during treatment, your doctor will monitor your heart using electrocardiograms (ECGs) to ensure that your heart rhythm remains normal. ECGs will be performed once weekly during the initial treatment period and then at reduced frequency as determined by your doctor. If you are also taking other medications that prolong the QT interval, ECG monitoring will be performed more frequently.
Infections in patients over 65: Older patients face a greater risk of very serious infections compared with younger patients, particularly during the early treatment period. If you are over 65 years of age, you will be monitored closely for severe infections during the initial phase of treatment.
Children and Adolescents
VANFLYTA must not be given to children or adolescents under 18 years of age. There is insufficient evidence regarding the use of quizartinib in this age group, and the safety and efficacy have not been established in pediatric patients. Acute myeloid leukemia in children may require different treatment approaches, and healthcare providers should consider age-appropriate treatment alternatives.
Pregnancy and Breastfeeding
Pregnancy: You must not take VANFLYTA during pregnancy. Based on its mechanism of action, quizartinib may cause harm to an unborn baby. Women of childbearing potential must have a pregnancy test within 7 days before starting treatment with VANFLYTA. Effective contraception must be used during the following periods:
- Women: During treatment with VANFLYTA and for at least 7 months after the last dose.
- Men: During treatment with VANFLYTA and for at least 4 months after the last dose.
If you are pregnant, think you may be pregnant, or are planning to have a baby, consult your doctor, pharmacist, or nurse before using this medicine.
Breastfeeding: You must not breastfeed during treatment with VANFLYTA and for at least 5 weeks after the last dose. It is not known whether quizartinib passes into breast milk, and a risk to the nursing infant cannot be excluded.
Fertility: VANFLYTA may reduce fertility in both women and men. Discuss fertility preservation options with your doctor before starting treatment, especially if you plan to have children in the future.
Driving and Operating Machinery
VANFLYTA is not expected to affect the ability to drive or operate machinery. However, if you experience any side effects that could impair your ability to drive or use machines (such as dizziness or fatigue), refrain from these activities until you feel well enough to do so safely.
How Does VANFLYTA Interact with Other Drugs?
Unlike many biological therapies, VANFLYTA is a small molecule that is extensively metabolized by the cytochrome P450 (CYP) enzyme system, particularly CYP3A. This means that other medications which affect the activity of CYP3A enzymes can significantly alter the blood levels of quizartinib, either increasing the risk of side effects or reducing its therapeutic efficacy. It is essential that you inform your doctor, pharmacist, or nurse about all medications you are currently taking, have recently taken, or might take, including prescription medications, over-the-counter drugs, vitamins, antacids, and herbal remedies.
Major Interactions (Avoid or Use with Caution)
The following categories of medications can significantly affect how VANFLYTA works and may require dose adjustments or avoidance:
| Drug Category | Examples | Effect on VANFLYTA |
|---|---|---|
| Azole antifungals | Itraconazole, posaconazole, voriconazole | Significantly increases VANFLYTA blood levels; dose reduction required |
| Macrolide antibiotics | Clarithromycin, telithromycin | Increases VANFLYTA blood levels; increased risk of side effects |
| Antidepressants | Nefazodone | Increases VANFLYTA blood levels |
| HIV medications | Ritonavir | Increases VANFLYTA blood levels and side effect risk |
If you are taking strong CYP3A inhibitors, your doctor may start you on a lower dose of VANFLYTA (one 17.7 mg tablet once daily instead of the standard starting dose) to reduce the risk of side effects, particularly QT prolongation.
| Drug Category | Examples | Effect on VANFLYTA |
|---|---|---|
| Anti-tuberculosis drugs | Rifampicin | Significantly reduces VANFLYTA effectiveness |
| Anticonvulsants | Carbamazepine, primidone, phenobarbital, phenytoin | Reduces VANFLYTA effectiveness |
| Prostate cancer drugs | Apalutamide, enzalutamide | Reduces VANFLYTA effectiveness |
| Other inducers | Bosentan, mitotan | Reduces VANFLYTA effectiveness |
| Herbal products | St. John’s Wort (Hypericum perforatum) | Reduces VANFLYTA effectiveness; must be avoided |
| HIV medications | Efavirenz, etravirine | Reduces VANFLYTA effectiveness |
QT-Prolonging Medications
Using VANFLYTA together with other medications that are known to prolong the QT interval can further increase the risk of serious heart rhythm disturbances. If possible, your doctor will avoid prescribing QT-prolonging medications alongside VANFLYTA. If co-administration is necessary, your heart will be monitored more frequently with ECGs. Examples of QT-prolonging medications include:
- Antifungal agents: Azole antifungals (which are also CYP3A inhibitors)
- Antiemetics: Ondansetron, granisetron
- Antibiotics: Azithromycin, moxifloxacin, doxycycline, pentamidine
- Antiprotozoals: Atovaquone
- Antipsychotics/antiemetics: Prochlorperazine
- Immunosuppressants: Tacrolimus
Because VANFLYTA has significant drug interactions, always give your doctor a complete list of all medications, herbal products, vitamins, and supplements you are taking. Do not start, stop, or change the dose of any medication during VANFLYTA treatment without first consulting your doctor or pharmacist. This includes over-the-counter medications and herbal remedies such as St. John’s Wort.
What Is the Correct Dosage of VANFLYTA?
Always take VANFLYTA exactly as your doctor or pharmacist has instructed. Do not change the dose or stop taking VANFLYTA without speaking with your doctor first. The dose of VANFLYTA varies depending on the phase of treatment (with chemotherapy, after chemotherapy, or as maintenance after transplantation), and your doctor may adjust the dose based on your blood test results, any side effects you experience, and other medications you are taking.
During Chemotherapy
Standard Chemotherapy Phase Dosing
The usual starting dose is 35.4 mg (two 17.7 mg tablets) taken once daily for 14 days during each chemotherapy cycle. The maximum recommended dose during this phase is 53 mg once daily.
If you are taking strong CYP3A inhibitors (such as certain antifungal medications), your doctor may start you on a lower dose of 17.7 mg (one tablet) once daily.
After Chemotherapy (Continuation/Maintenance)
Post-Chemotherapy Maintenance Dosing
After your chemotherapy is completed, your doctor may adjust your dose to 26.5 mg (one 26.5 mg tablet) once daily for two weeks, and then increase the dose to 53 mg (two 26.5 mg tablets) once daily on an ongoing basis, depending on how you respond to treatment.
After Stem Cell Transplantation
If you undergo an allogeneic stem cell transplantation (bone marrow transplant), your doctor will stop VANFLYTA before the procedure. After the transplant, once you have recovered sufficiently, your doctor will tell you when to restart VANFLYTA as maintenance therapy. The timing and dosing will depend on your individual recovery and clinical status.
| Treatment Phase | Dose | Duration | Notes |
|---|---|---|---|
| Induction/Consolidation | 35.4 mg once daily | 14 days per cycle | With chemotherapy; max 53 mg/day |
| With CYP3A inhibitors | 17.7 mg once daily | 14 days per cycle | Reduced starting dose |
| Post-chemotherapy (initial) | 26.5 mg once daily | 2 weeks | Titration period |
| Maintenance | 53 mg once daily | Ongoing | Until progression or intolerance |
How to Take VANFLYTA
- Take VANFLYTA by mouth (orally) with or without food.
- Take VANFLYTA at approximately the same time each day. This helps you remember to take your medication consistently.
- Swallow the tablets whole. Do not crush, chew, or break the tablets.
- If you vomit after taking VANFLYTA, do not take additional tablets until it is time for your next scheduled dose.
Missed Dose
If you forget to take VANFLYTA, take it as soon as you remember on the same day. Take your next dose at the usual time the following day. Do not take a double dose (two doses on the same day) to make up for a missed dose.
Overdose
If you accidentally take more tablets than prescribed, or if someone else accidentally takes your medication, contact your doctor immediately or go to the nearest hospital emergency department. Bring the medication package with you. You may need medical monitoring and treatment, particularly for QT prolongation.
Stopping VANFLYTA treatment without your doctor’s approval may cause your condition to worsen. Do not stop taking your medication unless your doctor tells you to do so. If you have any questions about how long to continue treatment, speak with your doctor or pharmacist.
What Are the Side Effects of VANFLYTA?
Like all medicines, VANFLYTA can cause side effects, although not everyone experiences them. Because VANFLYTA is used in combination with chemotherapy for the treatment of acute myeloid leukemia, many of the side effects overlap with those of the underlying disease and the chemotherapy itself. Your medical team will monitor you closely throughout treatment and manage any side effects that arise.
Serious Side Effects
Contact your doctor, pharmacist, or nurse immediately if you experience any of the following:
- Dizziness, feeling faint, or lightheadedness – these may be signs of QT prolongation, an abnormal electrical activity in the heart that affects its rhythm.
- Fever, cough, chest pain, shortness of breath, fatigue, or pain during urination – these may be signs of an infection or febrile neutropenia (low white blood cell count with fever).
Side Effects by Frequency
Very Common
May affect more than 1 in 10 people
- Elevated alanine aminotransferase (a liver enzyme)
- Thrombocytopenia (low platelet count)
- Anemia (low red blood cell count)
- Neutropenia (low neutrophil count, a type of white blood cell)
- Diarrhea
- Nausea
- Abdominal pain (stomach pain)
- Headache
- Vomiting
- Edema (swelling of the face, arms, and legs)
- Upper respiratory tract infections (nose and throat infections)
- Decreased appetite
- Epistaxis (severe nosebleeds)
- Fungal infections
- Herpes infections
- Dyspepsia (indigestion)
- Bacteremia (bacteria in the blood)
Common
May affect up to 1 in 10 people
- Pancytopenia (low levels of all blood cell types)
Uncommon
May affect up to 1 in 100 people
- Cardiac arrest (the heart stops beating)
- Ventricular fibrillation (dangerous, irregular, and uncoordinated contractions of the heart’s ventricles)
The side effects listed above are those that have been specifically identified with VANFLYTA treatment. Because VANFLYTA is given in combination with intensive chemotherapy, patients may also experience additional side effects related to the chemotherapy regimen itself. Your medical team will discuss the complete spectrum of potential side effects before you begin treatment.
If you experience any side effects, including any not listed above, tell your doctor, pharmacist, or nurse. You can also report side effects directly to your national medicines regulatory authority. Reporting helps to provide ongoing information about the benefit-risk balance of medications after they have been approved.
Many side effects of VANFLYTA and chemotherapy combination therapy can be managed with supportive care. Your doctor may temporarily pause your treatment, reduce your dose, or provide additional medications to manage specific side effects. Blood transfusions may be given for severe anemia or thrombocytopenia. Antimicrobial medications may be prescribed to prevent or treat infections. Never adjust your dose or stop treatment on your own – always consult your medical team.
How Should You Store VANFLYTA?
Proper storage of VANFLYTA is important to ensure the medication remains effective and safe throughout its shelf life. Follow these storage guidelines carefully:
- Keep out of reach of children: Store VANFLYTA in a location that is out of the sight and reach of children to prevent accidental ingestion.
- Expiry date: Do not use VANFLYTA after the expiry date printed on the carton and blister pack (after “EXP”). The expiry date refers to the last day of the month indicated.
- Storage conditions: No special storage conditions are required. VANFLYTA can be stored at room temperature.
- Packaging integrity: Do not use the medicine if you notice that the packaging is damaged or shows signs of tampering.
- Disposal: Do not dispose of medications via wastewater or household waste. Ask your pharmacist about how to properly dispose of medications you no longer use. These measures help protect the environment.
VANFLYTA is supplied in perforated unit-dose blister packs made of aluminium/aluminium, which helps protect the tablets from moisture and light. Keep the tablets in their blister packaging until you are ready to take them.
What Does VANFLYTA Contain?
Understanding the complete composition of your medication can be important, particularly if you have known allergies or intolerances to specific pharmaceutical ingredients. Below is the full ingredient list for both strengths of VANFLYTA:
Active Ingredient
The active substance is quizartinib, supplied as quizartinib dihydrochloride. Each VANFLYTA 17.7 mg tablet contains 17.7 mg of quizartinib, and each VANFLYTA 26.5 mg tablet contains 26.5 mg of quizartinib.
Inactive Ingredients (Excipients)
| Component | 17.7 mg Tablet | 26.5 mg Tablet |
|---|---|---|
| Tablet core | Hydroxypropyl betadex, microcrystalline cellulose, magnesium stearate | Hydroxypropyl betadex, microcrystalline cellulose, magnesium stearate |
| Film coating | Hypromellose, talc, triacetin, titanium dioxide | Hypromellose, talc, triacetin, titanium dioxide, yellow iron oxide |
| Appearance | White, round, imprinted “DSC 511” | Yellow, round, imprinted “DSC 512” |
| Pack sizes | 14 × 1 or 28 × 1 tablets | 14 × 1, 28 × 1, or 56 × 1 tablets |
Both tablet strengths are packaged in perforated unit-dose aluminium/aluminium blister packs. Not all pack sizes may be marketed in every country.
VANFLYTA is manufactured by Daiichi Sankyo Europe GmbH (Luitpoldstrasse 1, 85276 Pfaffenhofen, Germany) and the marketing authorization is held by Daiichi Sankyo Europe GmbH (Zielstattstrasse 48, 81379 Munich, Germany). For additional information about this medicine, consult the European Medicines Agency website or your local regulatory authority.
Frequently Asked Questions About VANFLYTA
The FLT3-ITD (FMS-like tyrosine kinase 3 internal tandem duplication) mutation is one of the most common genetic changes found in acute myeloid leukemia (AML), present in approximately 25–30% of newly diagnosed cases. This mutation causes the FLT3 receptor protein on blood cell surfaces to become permanently activated, sending continuous growth signals that drive the uncontrolled proliferation of leukemic cells. Patients with FLT3-ITD positive AML historically have had worse outcomes with chemotherapy alone, including higher relapse rates and shorter overall survival. The identification of this mutation through genetic testing is essential because it determines eligibility for targeted therapies like VANFLYTA, which specifically block the abnormal FLT3-ITD signaling. Your doctor will test your cancer cells for this mutation before prescribing VANFLYTA.
The duration of VANFLYTA treatment depends on your individual clinical situation and response to therapy. During the induction and consolidation chemotherapy phases, VANFLYTA is taken for 14 days during each chemotherapy cycle. After chemotherapy is completed, VANFLYTA may continue as maintenance therapy on an ongoing basis. If you undergo a bone marrow transplant, VANFLYTA is stopped before the procedure and restarted after you have recovered. Your doctor will regularly review your treatment and determine how long you should continue taking VANFLYTA. Do not stop taking the medication without your doctor’s guidance, as this may allow the leukemia to return or progress.
Regular ECG (electrocardiogram) monitoring is a mandatory part of VANFLYTA treatment because the medication can cause QT prolongation, a condition where the electrical recovery phase of the heartbeat takes longer than normal. If the QT interval becomes excessively prolonged, it can trigger dangerous heart rhythm disturbances, including ventricular fibrillation and cardiac arrest. ECGs are typically performed once a week during the initial treatment period and then at reduced frequency as determined by your doctor. If you are also taking other medications that can prolong the QT interval (such as certain antifungal drugs or antibiotics), your doctor will monitor your heart even more frequently. Additionally, your electrolyte levels (potassium and magnesium) will be checked regularly, as low levels can worsen QT prolongation.
Yes, VANFLYTA can be taken with or without food. There are no dietary restrictions associated with VANFLYTA, and food does not significantly affect the absorption of the medication. The most important thing is to take VANFLYTA at approximately the same time each day to maintain consistent blood levels and to help you remember your daily dose. Swallow the tablets whole – do not crush, chew, or break them. If you vomit shortly after taking your dose, do not take a replacement dose; instead, wait until your next scheduled dose.
If you experience any side effects while taking VANFLYTA, contact your medical team promptly. Some side effects, such as mild gastrointestinal symptoms, may be manageable with supportive care. However, certain side effects require immediate medical attention, particularly signs of heart rhythm problems (dizziness, fainting, lightheadedness) or serious infection (fever, chest pain, difficulty breathing). Never adjust your dose or stop taking VANFLYTA on your own. Your doctor has several strategies for managing side effects, including temporary treatment pauses, dose reductions, or additional supportive medications. Blood cell count abnormalities may be managed with transfusions, growth factors, or antimicrobial prophylaxis. Always report all side effects, even mild ones, so your medical team can provide the best possible care.
The clinical efficacy of VANFLYTA was demonstrated in the landmark QuANTUM-First trial, a large phase III randomized controlled study. In this trial, patients with newly diagnosed FLT3-ITD positive AML who received quizartinib plus chemotherapy had a median overall survival of 31.9 months, compared with 15.1 months for patients who received placebo plus chemotherapy. This represents a clinically and statistically significant improvement in survival for this patient population. The benefit of VANFLYTA was observed across multiple subgroups and included both the induction/consolidation and maintenance phases of treatment. VANFLYTA is one of several FLT3 inhibitors now available, and your doctor will determine the most appropriate treatment based on your specific clinical situation, mutation profile, and overall health status.
References
- European Medicines Agency (EMA). VANFLYTA (quizartinib) – Summary of Product Characteristics. 2024. Available at: www.ema.europa.eu
- U.S. Food and Drug Administration (FDA). VANFLYTA (quizartinib) – Prescribing Information. July 2023.
- Erba HP, Montesinos P, Kim HJ, et al. Quizartinib plus chemotherapy in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia (QuANTUM-First): a randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet. 2023;401(10388):1571–1583. doi:10.1016/S0140-6736(23)00464-6
- National Comprehensive Cancer Network (NCCN). Clinical Practice Guidelines in Oncology: Acute Myeloid Leukemia. Version 1.2025.
- Döhner H, Wei AH, Appelbaum FR, et al. Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN. Blood. 2022;140(12):1345–1377. doi:10.1182/blood.2022016867
- World Health Organization (WHO). WHO Model List of Essential Medicines. 23rd Edition, 2023.
- Daver N, Schlenk RF, Russell NH, Levis MJ. Targeting FLT3 mutations in AML: review of current knowledge and evidence. Leukemia. 2019;33(2):299–312. doi:10.1038/s41375-018-0357-9
- British National Formulary (BNF). Quizartinib. Available at: bnf.nice.org.uk
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