The National Institute for Health and Care Excellence (NICE) has recommended Novartis’ Rydapt (midostaurin) for the treatment of aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasms or mast cell leukaemia (three different subtypes of advanced SM).
Rydapt is the first and only licensed treatment for advanced SM in the UK and has been shown to provide therapeutic benefits to adults with the disease.
Patients in the UK could formerly only access drugs to treat their symptoms and were not able to receive targeted therapy that would inhibit KIT signalling, cell proliferation and histamine release, and induce apoptosis in mast cells.
Around 170 patients in England and Wales with AdvSM will now be eligible for treatment with Rydapt through the NHS following the NICE decision.
Rydapt was approved by the FDA for treatment of a form of acute myeloid leukaemia (AML) in 2017 and has been approved in the UK for AML since 2018.
NICE said that clinical trials showed an increase in overall survival of 23% for people with advanced SM when treated with Rydapt compared to other treatments, which include interferon alpha, cladribine and imatinib.
Deepti Radia, consultant haematologist at Guy’s and St Thomas’ Hospital in London said: “Treatment options for advanced systemic mastocytosis have been limited… 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.”
Novartis reported that the decision followed “significant dialogue between NICE, NHS England, Novartis, patient advocacy organisations and clinical experts”. The company also noting that there were “significant challenges in the methodologies for appraisal of rare medicines” in the UK.
The NICE approval of Novartis’ Rydapt comes as the evidence-based HTA body shares its intention to review and revamp its processes and evaluation methods.