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Press Release

NICE recommends Novartis’ Rydapt® (midostaurin)? for patients with newly diagnosed FLT3-mutated AML, the first new treatment in more than 25 years1,2

Posted on: 11 May 18

·       Midostaurin treatment regimen in FLT3-mutated AML demonstrated a significant improvement in overall survival with a 22% reduction in the risk of death3

·       Testing for different genetic mutations in AML is critical to predicting outcomes and determining treatment strategies4

·       A FLT3 gene mutation in AML can result in faster disease progression, higher relapse rates and lower rates of survival than other forms of AML4-6


Camberley, May 04, 2018 Novartis today announced Rydapt® (midostaurin) has been recommended by the National Institute for Health and Care Excellence (NICE) as a cost-effective treatment option for adult patients newly diagnosed with acute myeloid leukemia (AML) who are FMS-like tyrosine kinase 3 mutation-positive (FLT3+) in combination with chemotherapy and for patients with complete response followed by Rydapt® single agent maintenance therapy.7 Midostaurin will be routinely available on the NHS with immediate effect.


AML is a rare and aggressive cancer of the blood and bone marrow8 that affects over 3,000 patients in the UK each year.9  AML prevents white blood cells from maturing, causing an accumulation of “blasts;” the rapid proliferation of these blasts prevents normal blood cells from forming.8


“Acute Myeloid Leukaemia is a rapidly progressing and often fatal disease. Overall only around 20 per cent of patients with AML will survive for five years or more after they are diagnosed,” said Zack Pemberton-Whiteley, Campaigns and Advocacy Director at Leukaemia Care. “As new treatment options become available, we urgently need to ensure that timely testing is done to match the right patient with the right treatment, especially for those with aggressive forms of the disease.”


Mutations in specific genes are found in many cases of AML and genetic testing in newly diagnosed AML patients can help to determine prognosis and potential treatment strategies4. Around one-third of patients with AML have a mutation in the FLT3 gene.4 The FLT3 gene mutation can result in faster disease progression, higher relapse rates and lower rates of survival than other forms of AML.4-6


The NICE recommendation is based on results from the Phase III RATIFY (CALGB 10603 [Alliance]) clinical trial, which demonstrated that newly diagnosed FLT3+ patients who received midostaurin in combination with chemotherapy followed by midostaurin monotherapy after complete response as maintenance therapy experienced significant improvement in overall survival with a 22% reduction in the risk of death compared with chemotherapy alone (hazard ratio [HR] = 0.78, 95% confidence interval [CI], 0.63, 0.95; 2 sided p=0.016).3


In addition, event-free survival (EFS; event defined as no complete remission within 60 days of the start of induction therapy, relapse or death) was significantly higher for midostaurin plus chemotherapy versus chemotherapy alone (median of 8.2 months compared to 3.0 months, HR = 0.78, 95% CI 0.66, 0.93 and 2 sided p=0.004).3


“As we’ve come to understand more about the different mutations associated with AML and their effect on prognosis, such as the FLT3 mutation, it has become essential that clinicians use genetic testing to guide treatment decisions” said Professor Paresh Vyas, Professor of Haematology and Honorary Consultant Haematologist and Group Leader MRC Molecular Haematology Unit, University of Oxford. “The overall survival advantage that midostaurin plus chemotherapy demonstrated in clinical trials represents a significant advancement for newly diagnosed AML patients with the FLT3 mutation and helps to establish a new standard of care for these patients with a worse prognosis.”


“The NICE positive recommendation represents a remarkable advance for patients in England and Wales affected by this high-risk blood cancer. They will now have access to a targeted medicine that could significantly extend their lives,” said Mari Scheiffele, General Manager of Novartis Oncology UK & Ireland. “We are proud to build on our heritage in haematology to have the first new treatment for AML in more than 25 years.”


Midostaurin is indicated for use in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy, and for patients in complete response followed by midostaurin single agent maintenance therapy, for adult patients with newly diagnosed AML who are FLT3 mutation-positive.7


In RATIFY, the most frequent adverse reactions (incidence greater than or equal to 20%) in the midostaurin plus chemotherapy arm were febrile neutropenia, nausea, vomiting, mucositis, headache, musculoskeletal pain, petechiae (small red skin spots), device-related infection, epistaxis, hyperglycemia and upper respiratory tract infections. The most frequent Grade 3/4 adverse reactions (incidence greater than or equal to 10%) were febrile neutropenia, device-related infection and mucositis. The safety profile of the midostaurin arm is comparable to the placebo arm in the clinical trial.3,7


The NICE recommendation has been published in its final appraisal determination. Novartis is awaiting publication of the NICE Technology Appraisal Guidance, the final step within the NICE approval process, which is scheduled for publication in June 2018 and will be available via the NICE website.



About AML

AML is the most common acute leukemia in adults; in the UK, it accounts for about 1 in 3 cases of leukaemia in adults overall and about 9 in 10 cases of acute leukaemia.9


AML prevents white blood cells from maturing, causing an accumulation of “blasts,” which do not allow room for the normal blood cells.8 Mutations in specific genes are found in many cases of AML,1 and genetic testing for mutations in newly diagnosed AML patients can help to determine prognosis and potential treatment strategies.10


About Rydapt® (midostaurin)

Midostaurin is an oral, multi-targeted inhibitor of multiple kinases, including FLT3 and KIT, which help regulate many essential cell processes, interrupting cancer cells’ ability to grow and multiply.7


Midostaurin is available in 25 mg capsules and the recommended dosage is 50 mg orally twice on days 8 to 21 each cycle of induction therapy and on days 8 to 21 each cycle of consolidation therapy. For patients in complete response every day as single agent maintenance therapy, it is dosed until relapse for up to 12 cycles of 28 days each. In patients receiving a haematopoietic stem cell transplant (SCT), midostaurin should be discontinued 48 hours prior to the conditioning regimen for SCT.7


About Novartis

Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2017, the Group achieved net sales of USD 49.1 billion, while R&D throughout the Group amounted to approximately USD 9.0 billion. Novartis Group companies employ approximately 124,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world. For more information, please visit


In the UK, Novartis develops, manufactures and markets innovative medicines, devices and diagnostic tests which help improve patient outcomes. Based on four sites across the north and south of England, we employ approximately 1,500 people to serve healthcare needs across the whole of the UK, as well as supporting the global operations of Novartis by manufacturing the active pharmaceutical ingredients used worldwide in many medicines. In 2016 Novartis in the UK invested almost £40million in R&D and is the largest commercial sponsor of clinical trials. For more information, please visit


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  1.        Schiller GJ. High-risk acute myelogenous leukemia: treatment today ... and tomorrow. Hematology Am Soc Hematol Educ Program. 2013; 2013:201-208.
  2.        Lin TL, Levy MY. Acute myeloid leukemia: focus on novel therapeutic strategies. Clin Med Insights Oncol. 2012;6:205-217.
  3.        Stone RM, Mandrekar S, Sanford BL, et al. Midostaurin plus chemotherapy for acute myeloid leukaemia with a FLT3 mutation. N Engl J Med. 2017;377(5):454-464
  4.        Patel JP, Gönen M, Figueroa ME, et al. Prognostic relevance of integrated genetic profiling in acute myeloid leukemia. N Engl J Med. 2012; 22;366(12):1079-1089.
  5.        Gilliland DG, Griffin JD. The roles of FLT3 in hematopoiesis and leukemia. Blood. 2002;100(5): 1532-1542.
  6.        Yanada M, Matsuo K, Suzuki T, et al. Prognostic significance of FLT3 internal tandem duplication and tyrosine kinase domain mutations for acute myeloid leukemia: a meta-analysis. Leukemia. 2005;19(8):1345–1349.
  7.        Rydapt® (midostaurin) Summary of Product Characteristics. Camberley, United Kingdom: Novartis Europharm Limited; 2017.
  8.        National Institutes of Health (NIH) National Cancer Institute (NCI). Adult Acute Myeloid Leukemia Treatment (PDQ®) Accessed 26 April 2018.
  9.        Leukaemia Care. Adult Acute Myeloid Leukaemia: A Guide for Patients. (2018) Accessed 26 April 2018.
  10.     DA, Borowitz MJ, Cessna M, et al. Initial Diagnostic Workup of Acute Leukemia: Guideline from the College of American Pathologists and the American Society of Hematology. Arch Pathol Lab Med. 2017 Oct;141(10):1342-1393.
Editor's Details

Mike Wood

Last updated on: 11/05/2018

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