In this article
FDA Approval of Vanflyta for FLT3-ITD Positive AML
August 2023: The treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3 internal tandem duplication (ITD)-positive, as detected by an FDA-approved test, with standard cytarabine and anthracycline induction and cytarabine consolidation, as well as as maintenance monotherapy following consolidation chemotherapy, has been approved by the Food and Drug Administration.
LeukoStrat CDx FLT3 Mutation Assay was additionally given FDA approval as a Vanflyta companion diagnostic.
QuANTUM-First Clinical Trial Efficacy and Results
In QuANTUM-First (NCT02668653), a randomised, double-blind, placebo-controlled trial involving 539 patients with newly diagnosed FLT3-ITD positive AML, the effectiveness of quizartinib in combination with chemotherapy was assessed. A clinical trial assay was used to establish the FLT3-ITD status prospectively, and the companion diagnostic LeukoStrat CDx FLT3 Mutation Assay was used to confirm it after the fact.
According to the initial assignment, patients were randomised (1:1) to receive quizartinib (n = 268) or a placebo (n = 271) with induction, consolidation, and maintenance monotherapy. At the start of post-consolidation therapy, there was no re-randomization. After hematopoietic stem cell transplantation (HSCT) recovery, patients who underwent HSCT started maintenance therapy.
Overall survival (OS), calculated from the randomization date until death from any cause, served as the primary efficacy outcome measure. After at least 24 months had passed since the final patient was randomly assigned, the major analysis was carried out. In the trial, the quizartinib arm showed a statistically significant increase in OS [hazard ratio (HR) 0.78; 95% CI: 0.62, 0.98; 2 sided p=0.0324]. With a median duration of 38.6 months (95% CI: 21.9, NE), the quizartinib arm’s CR rate was 55% (95% CI: 48.7, 60.9), and the placebo group’s CR rate was 55% (95% CI: 49.2, 61.4) with a median duration of 12.4 months (95% CI: 8.8, 22.7).
Following allogeneic HSCT, quizartinib is not recommended as maintenance monotherapy. Quiartinib has not been shown to increase OS in this situation.
Cardiac Safety Boxed Warnings and Vanflyta REMS
QT prolongation, torsades de pointes, and cardiac arrest are all mentioned in the boxed warning for quizartinib. Only the Vanflyta REMS, a restricted programme under a Risk Evaluation and Mitigation Strategy (REMS), offers quizartinib. For a comprehensive list of side effects, consult the prescribed information.
Recommended Quizartinib Dosage and Administration Schedule
The recommended quizartinib dose is as follows:
- Induction: 35.4 mg orally once daily on Days 8-21 of “7 + 3” (cytarabine [100 or 200 mg/m2/day] on Days 1 to 7 plus daunorubicin [60 mg/m2/day] or idarubicin [12 mg/m2/day] on Days 1 to 3) and on Days 8-21 or 6-19 of an optional second induction (“7 + 3” or “5 + 2” [5 days cytarabine plus 2 days daunorubicin or idarubicin], respectively),
- b) Consolidation: 35.4 mg orally once daily on Days 6-19 of high‑dose cytarabine (1.5 to 3 g/m2 every 12 hours on Days 1, 3 and 5) for up to 4 cycles, and
- c) Maintenance: 26.5 mg orally once daily on Days 1 to 14 and 53 mg once daily, thereafter, for up to thirty-six 28-day cycles.
- View full prescribing information for Vanflyta
How CancerFax Helps
CancerFax is a specialist cancer access and patient-navigation platform. We help patients and families understand their options, organise medical records, coordinate hospital communication, and support cross-border treatment planning where appropriate.
We help collect and organise reports, scans, pathology, biomarker results, and treatment history for structured case review.
We communicate with hospitals or trial teams to assess whether a case may be suitable for further screening.
We support appointment coordination, document submission, translation, and direct communication with international departments.
For international patients, we help with practical coordination — travel planning, hospital admission guidance, and local support.
If this option is not suitable, we help explore other relevant treatments, clinical trials, or advanced care pathways.
From inquiry through to follow-up, our coordinators provide a single point of contact for the family.
CancerFax does not guarantee treatment access, eligibility, or clinical outcome. Our role is to help patients access accurate information, structured review, and appropriate specialist pathways.
About Dr. Nishant Mittal
Dr. Nishant Mittal is a highly accomplished researcher with over 13 years of experience in the fields of cardiovascular biology and cancer research. Significant contributions to stem cell biology, developmental biology, and innovative research techniques mark his career. Research Highlights Dr. Mittal's research has focused on several key areas: 1) Cardio…
✓ Reviewed for medical accuracy by the CancerFax review panel.
Medical Disclaimer
This article is for educational purposes only and should not replace medical advice, diagnosis, or treatment from a qualified oncology specialist. Every patient's case is different. Treatment decisions should always be made after a review of complete medical records by the treating medical team.
Treatment availability, eligibility, timelines, and access can change. Any clinical trial participation depends on detailed review and approval by the trial hospital or investigator.