Rydapt receives NICE recomendation to treat agressive systemic mastocytosis – Novartis

Novartis 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).

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-threating 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.