Novartis’ Rydapt ®▼ (midostaurin) is the first and only treatment to receive NICE recommendation for routine funding for patients in England and Wales living with advanced systemic mastocytosis (AdvSM)
Advanced systemic mastocytosis (AdvSM) comprises rare hematologic neoplasms that are associated with a poor prognosis and lack effective treatment options.1
Rydapt®▼ is the first and only licensed product recommended by NICE for the treatment of people living with AdvSM.
The positive recommendation follows significant dialogue between NICE, NHS England, Novartis, Patient Advocacy Organisations and clinical experts.
London, UK, 19th August 2021 – Novartis today welcomed the news that the National Institute for Health and Care Excellence (NICE) has issued a Final Appraisal Determination recommending Rydapt®▼ (midostaurin) for the treatment of aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasms or mast cell leukaemia (nb – collectively called AdvSM).2 Midostaurin is the first and only licensed treatment for AdvSM in the UK and has been shown to provide therapeutic benefits to adults with the disease.1
AdvSM affects between 1 in 10,000 – 30,000 people.3 The life-threating blood disorder is characterised by uncontrolled growth and accumulation of mast cells – mediators of allergic responses – in one or more organs.4 These cells accumulate in such high quantities that they begin to cause organ damage.3 The wide-ranging symptoms of the disease can have a devastating impact on an individual’s quality of life.3 Symptoms include itching, fever, abdominal pain, nausea and vomiting.5 Until today, patients in the UK could only access drugs to treat their symptoms, not targeted therapy that inhibits KIT signalling, cell proliferation and histamine release, and induce apoptosis in mast cells.6
Currently, there are significant challenges in the methodologies for appraisal of rare medicines. Novartis has a strong legacy in rare diseases and remained committed to securing a positive outcome and ensuring patient access to this much needed option, despite the foreseeable challenges associated with midostaurin being appraised via the Single Technology Appraisal (STA) route, rather than the Highly Specialised Technology (HST) route.7 For patients living with rare diseases like AdvSM, these challenges can delay access to new treatments for their condition.
The Final Appraisal Determination by NICE recommending midostaurin for AdvSM (a collective term for aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasms or mast cell leukaemia) follows significant dialogue and a commitment to finding a solution to ensure patient access, between all stakeholders involved including NICE, NHS England, Novartis, Patient organisations and clinical experts.
Novartis is pleased that an outcome that benefits patients could be found through open dialogue and collaboration across all parties, it is crucial to work together and bring innovation to patients.
Jess Hobart, Chair and Trustee at patient organisation UK Masto, comments: “For many years, people living with advanced systemic mastocytosis (AdvSM) have had no specific treatment options available to them. This is a complex disease, which has a huge impact on the lives of those living with the condition, their family and friends. Midostaurin is the first licensed and reimbursed treatment for AdvSM and the approval is a welcomed acknowledgement from regulatory bodies that patients with rare conditions like AdvSM deserve to have access to innovative therapy options despite the challenges of assessing medications in small patient populations.”
Dr Deepti Radia, Consultant Haematologist at Guy's and St Thomas' Hospital, London comments: “Treatment options for advanced systemic mastocytosis (AdvSM) have been limited and until today, clinicians have used symptomatic and non-targeted cytoreductive treatments. The NICE recommendation of midostaurin provides patients and healthcare professionals with greater choice and access to the first licensed treatment for this life-threatening blood disorder that could help to improve outcomes.”
Mari Scheiffele, General Manager for Oncology UK & Ireland, Novartis, says: “We are pleased that NICE has recommended midostaurin as the first licensed treatment of advanced systemic mastocytosis (AdvSM) in England and Wales. The approval represents a huge milestone for patients and the AdvSM community where, to-date, there have been limited treatment options. Novartis is committed to securing the best outcomes for patients and worked collaboratively with NICE, NHS England, Patient Advocacy Groups and clinical experts to ensure midostaurin is available to patients living with this life-threatening disease.”
Notes to Editors:
About Rydapt®▼ (midostaurin)
Rydapt®▼ (midostaurin) is indicated as monotherapy for the treatment of adult patients with haematological neoplasm (SM‐AHN), or mast cell leukaemia (MCL). Advanced SM is a complex heterogenous blood cancer, characterised by: an accumulation of abnormal neoplastic mast cells in the bone marrow and other organ systems, which produces organ damage.8,9
This medicine is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse events. Reporting forms and information can be found at mhra.gov.uk/yellowcard.
About advanced systemic mastocytosis (AdvSM)
Advanced SM is a group of rare diseases in which uncontrolled growth and accumulation of mast cells (a type of white blood cell) occurs in one or more organs, including the GI tract, spleen, lymph nodes and bone marrow.1 Mast cells release substances, such as histamine, which can lead to symptoms including itching, fever, abdominal pain, nausea and vomiting.5 The uncontrolled growth and reproduction of mast cells is caused in many people by a KIT mutation. KIT D816V is the most common mutation in SM, occurring in about 90% of patients.10
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1 Gotlib, J. et al. Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis. New England Jorunal of Medicine, 374(26), pp.2530-2541 2 National Institute for Health and Care Excellence (2021), Final Appraisal Document: Midostaurin for treating advanced systemic mastocytosis. 3 UK Masto. Mastocytosis. Available at: https://ukmasto.org/about-mcd/mastocytosis/ [Accessed August 2021]. 4 National Institute for Health and Care Excellence (2019). Appendix B Final scope for the appraisal of midostaurin for treating advanced systemic mastocytosis. [online] . Available at: https://www.nice.org.uk/guidance/gid-ta10503/documents/final-scope [Accessed August 2021]. 5 Cancer.net. (2012). Mastocytosis - Symptoms and Signs. Available at: https://www.cancer.net/cancer-types/mastocytosis/symptoms-and-signs [Accessed August 2021]. 6 Rydapt Summary of Product Characteristics. Novartis Pharma AG 2018. 7 National Institute for Health and Care Excellence (2019). Committee papers. Available at: https://www.nice.org.uk/guidance/gid-ta10503/documents/committee-papers [Accessed August 2021]. 8 Valent P, Sperr WR, Akin C. How I treat patients with advanced systemic mastocytosis. Blood. 2010;116(26):5812-5817. 9 Pardanani A. Systemic mastocytosis in adults: 2017 update on diagnosis, risk stratification and management. Am J Hematol. 2016;91(11):1146-1159. 10 Chatterjee, A., Ghosh, J., & Kapur, R. (2015). Mastocytosis: a mutated KIT receptor induced myeloproliferative disorder. Oncotarget, 6(21), 18250–18264. https://doi.org/10.18632/oncotarget.4213