UK – NICE recommends Blueprint Medicines’ Ayvakyt to treat rare blood disorder

The National Institute for Health and Care Excellence (NICE) has recommended Blueprint Medicines’ Ayvakyt (avapritinib) to treat patients with the rare blood disorder systemic mastocytosis (SM).

The drug can now be used as a monotherapy to treat patients in England and Wales with the three subtypes of advanced SM: aggressive SM (ASM), SM with an associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL).

Approximately one in 10,000 people in the UK are thought to be living with SM, with advanced SM accounting for up to 10% of cases.

The disease is caused by the abnormal buildup of mast cells, a key part of the body’s immune system, and leads to symptoms including skin lesions, diarrhoea, bone pain, fatigue and anaphylaxis…