Basic Information
Ayvakyt
Regulatory Information
EMEA/H/C/005208
September 24, 2020
July 23, 2020
9
April 18, 2024
Company Information
Netherlands
Gustav Mahlerplein 2 1082MA Amsterdam
Blueprint Medicines (Netherlands) BV
Drug Classification
Active Substances Detail
EMA Resources
Detailed Information
Therapeutic Indication
### Therapeutic indication Unresectable or metastatic gastrointestinal stromal tumour (GIST) AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation. Advanced systemic mastocytosis (AdvSM) AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL), after at least one systemic therapy. Indolent systemic mastocytosis (ISM) AYVAKYT is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment (see section 5.1).
Overview Summary
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.