Ayvakyt

Ayvakyt is a cancer medicine used to treat adults with gastrointestinal stromal tumour (GIST), a cancer of the stomach and bowel, that cannot be removed by surgery and has spread to other parts of the body. Ayvakyt is used when the cancer cells have a D842V mutation, a change in the gene for the platelet-derived growth factor receptor alpha (PDGFRA). Ayvakyt is also used in adults to treat systemic mastocytosis, a blood disorder where the body makes too many abnormal mast cells (a type of white blood cell), which can build up in the skin, bones, joints, lymph nodes, liver, spleen, the stomach and gut. It is used to treat the following advanced forms of systemic mastocytosis: aggressive systemic mastocytosis, systemic mastocytosis associated with a haematological neoplasm (blood cancer), or mast cell leukaemia. It is used after the patient has received at least one systemic treatment (treatment with medicines affecting the whole body). Ayvakyt is also used to treat adults with moderate to severe symptoms of indolent systemic mastocytosis (ISM), a slow-growing form of systemic mastocytosis. It is used if the patient has not responded to symptomatic treatment (treatment that eases the symptoms of a disease without addressing its cause). These diseases are rare, and Ayvakyt was designated an ‘orphan medicine’ (a medicine used in rare diseases) on [17 July 2017](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3171889) (GIST) and on [26 October 2018](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182074) (mastocytosis). Ayvakyt contains the active substance avapritinib.