Acute myeloid leukaemia (AML) is the most common adult leukaemia, affecting approximately a third of newly diagnosed adult leukaemia patients, giving them a 5-year overall survival of ~28%.1


AML diagnosis is complex, but important

AML is a genetically heterogeneous clonal neoplasm disorder where different mutations can have an impact on prognosis:2

  • The most commonly detected single gene mutations in AML are in the FMS-like tyrosine kinase-3 (FLT3) gene:2
    • Internal tandem duplication (ITD) is the most common, affecting ~25–30% of AML patients, and is associated with a poor prognosis2
    • Tyrosine kinase domain (TKD), affecting ~7% of AML patients,2 with an unclear impact on prognosis3


FLT3+ AML treatment: time for a change

Over the past 30 years, improvements in the outlook for AML patients have been mainly due to developments in supportive care, rather than treatment evolution.4,5


What’s changed?

RYDAPT® (midostaurin) is the first and only FLT3 inhibitor approved for newly diagnosed FLT3+ AML patients eligible for intensive chemotherapy.4,6,7 Approved by both NICE and the SMC in 2018,8,9 RYDAPT induces cell cycle arrest and apoptosis in leukaemic cells.10


What does that mean for your patients?

RYDAPT provides a targeted treatment option for your FLT3+ AML patients eligible for intensive chemotherapy, and gives them the opportunity of an extra 4 years of life.11

RYDAPT is supported by results from the international, randomised controlled trial RATIFY. RYDAPT with standard of care* (SOC) resulted in significant improvements vs. placebo with SOC* in newly diagnosed FLT3+ AML patients:†,11

  • Increase in overall survival (OS) (median OS 25.6 months with placebo and SOC (95% CI, 31.5–NR) vs. 74.7 months with RYDAPT and SOC (95% CI, 18.6–42.9: p=0.009)‡,11
  • 22% relative reduction in risk of death (HR=0.78; 95% CI, 0.63–0.96: p=0.009)§,11
  • Signficant increase in event-free survival (EFS) (median EFS 3.0 months for placebo and SOC (95% CI, 1.9–5.9) vs. 8.2 months for RYDAPT and SOC (95% CI, 5.4–10.7: p=0.002),11
  • Safety profile similar to that of placebo11


Getting the FLT3 test result in time

FLT3 mutation status determination has previously formed part of a prognostic work-up, with no sense of real urgency as the patient will already be receiving the best SOC. Now, in order to catch the critical treatment window for RYDAPT initiation, quick testing turnaround times are essential.11


Professor Schlenk on the importance of FLT3 testing

Diagram which shows how timely FLT3 testing allows patients to be put on the most suitable treatment sooner

Go to the FLT3 testing page for more information on the testing process and how to ensure all your newly diagnosed FLT3+ AML patients have the opportunity to benefit from RYDAPT.11




AML, acute myeloid leukaemia; CI, confidence interval; EFS, event-free survival; HR, hazard ratio; ITD, internal tandem duplication; IV, intravenous; NICE, National Institute for Health and Care Excellence; NR, not reached; OS, overall survival; SMC, Scottish Medicines Consortium; TKD, tyrosine kinase inhibitor; q12h, every 12 hours.

* SOC: Chemotherapy dosing during induction was cytarabine IV, 200 mg/m2/d on Days 1–7, and daunorubicin IV, 60 mg/m2/d on Days 1–3. During consolidation, high-dose cytarabine was given at a dose of 3 g/m2/d IV q12h on Days 1, 3 and 5.

† Non-censored for stem cell transplant.

One-sided p by stratified log-rank test.

§ One-sided p by stratified score test.


  1. Howlader N, Noone AM, Krapcho M, et al. SEER Cancer Statistics Review, 1975–2016, National Cancer Institute. Bethesda, MD. Available at: Last accessed May 2020.
  2. Patel JP, Gönen M, Figueroa ME, et al. N Engl J Med. 2012;22;366(12):1079–1089.
  3. Mead AJ, Linch DC, Hills RK, et al. Blood. 2007;110(4):1262–1270.
  4. Ferrara F, Schier CA. Lancet. 2013;381(9865):484–495.
  5. Lin TL, Levy MY. Clin Med Insights Oncol. 2012;6:205–217.
  6. European Medicines Agency. Rydapt. Available at: Last accessed May 2020.
  7. US Food and Drug Administration. RYDAPT approval. Available at: Last accessed May 2020.
  8. National Institute for Health and Care Excellence. Midostaurin Final Appraisal Determination. Available at: Last accessed May 2020.
  9. Scottish Medicines Consortium. Midostaurin (Rydapt) for AML. Available at: Last accessed May 2020.
  10. Rydapt Summary of Product Characteristics. Novartis Pharma AG 2018.
  11. Stone RM, Mandrekar SJ, Sanford BL, et al. N Engl J Med. 2017;377(5):454–464.
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