following a full submission assessed under the orphan medicine process:
avapritinib (Ayvakyt®) is not recommended for use within NHSScotland.
Indication under review: 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).
In a non-randomised phase II study, 73% of patients with response-evaluable ASM, SM-AHN or MCL who received avapritinib achieved an overall response.
The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust economic analysis to gain acceptance by SMC.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Medicine details
- Medicine name:
- avapritinib (Ayvakyt)
- SMC ID:
- SMC2849
- Indication:
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).
- Pharmaceutical company
- Blueprint Medicines
- BNF chapter
- Malignant disease and immunosuppression
- Submission type
- Full
- Status
- Not recommended
- Date advice published
- 13 July 2026