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 (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.

Patients in the UK could formerly only access drugs to treat their symptoms, not targeted therapy that inhibits KIT signalling, cell proliferation and histamine release, and induces apoptosis in mast cells.

Dr Deepti Radia, consultant haematologist at Guy's and St Thomas' Hospital, London commented: “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.”

Jess Hobart, Chair and Trustee at patient organisation UK Masto, added: “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.”