The National Institute for Health and Care Excellence (NICE) has issued a Final Appraisal Determination recommending Novartis’ Rydapt®▼ (midostaurin) for the treatment of aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasms or mast cell leukaemia (collectively called AdvSM).
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.
AdvSM affects between 1 in 10,000 – 30,000 people. The life-threatening blood disorder is characterised by uncontrolled growth and accumulation of mast cells – mediators of allergic responses – in one or more organs. These cells accumulate in such high quantities that they begin to cause organ damage.
The wide-ranging symptoms of the disease can have a devastating impact on an individual’s quality of life. Symptoms include itching, fever, abdominal pain, nausea and vomiting. 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.
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 dialogue between all stakeholders involved including NICE, NHS England, Novartis, Patient organisations and clinical experts.
Jess Hobart, Chair and Trustee at patient organisation UK Masto, said: “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 commented: “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, said: “The approval represents a huge milestone for patients and the AdvSM community where, to-date, there have been limited treatment options.”