New treatment for rare leukaemia not approved in Scotland: what you need to know

Yes, avapritinib was approved in 2024 by the National Institute for Health and Care Excellence (NICE).

Wales and Northern Ireland usually follow NICE recommendations. This can take time. Your healthcare team can tell you whether avapritinib is available where you are treated.

Avapritinib works by blocking abnormal proteins that signal mast cells to grow and multiply.

Mast cells are a type of white blood cell. They are an important part of the immune system. Advanced systemic mastocytosis (AdvSM) is a collection of illnesses that causes you to make too many mast cells. Mast cell leukaemia (MCL) is a form of AdvSM and it is a blood cancer.

Making too many mast cells causes the symptoms of AdvSM, including MCL.

MCL does not have many existing treatment options. The other targeted treatment for MCL, midostaurin, is also only available in England.

Currently MCL patients mainly have treatment designed for other leukaemia types. This does not always work well and outcomes can be poor. People with MCL need more treatments that are targeting the mast cells specifically.

Leukaemia Care worked with UK Mastocytosis Support Group to provide the patient experience of having MCL and other types of ASM.

This helped decision-makers at the SMC understand how access to treatment can affect everyday life, not just clinical outcomes. We also wanted equal access to treatment across the UK.

If you have been diagnosed with MCL and would like to know whether this treatment might be suitable for you, speak to your healthcare team. They can talk you through your options and help you decide what’s right for you. They can also tell you if it’s available where you’re being treated.

At Leukaemia Care, our mission is to support you and your loved ones - especially when navigating new and potentially complex treatment options. Here’s how we can help: