Avapritinib monotherapy [AVA1]

For the treatment of aggressive systemic mastocytosis or aggressive systemic mastocytosis with an associated haematological neoplasm or mast cell leukaemia where the following criteria have been met:

1. This application for avapritinib monotherapy is being made by and the first cycle of systemic anti-cancer therapy with avapritinib monotherapy will be prescribed by a consultant specialist specifically trained and accredited in the use of systemic anti-cancer therapy.
2. The patient is an adult and has a pathologically-confirmed diagnosis of aggressive systemic mastocytosis (ASM) or aggressive systemic mastocytosis with an associated haematological neoplasm (ASM-AHN) or mast cell leukaemia.
3. The patient has advanced disease and requires systemic therapy for this condition.
4. The patient has previously received systemic therapy for this condition or not. Please mark below whether the patient has/has not previously received any systemic therapy for this condition:

• no, this patient has not received any previous systemic therapy for this condition
• yes, this patient has been previously treated with systemic therapy for this condition

5. The patient has previously received midostaurin or not. Please mark below whether the patient has previously received treatment with midostaurin or not:

• no, this patient has not received previous midostaurin
• yes, this patient has received previous midostaurin

6. The patient has not previously received treatment with avapritinib unless this was via a company early access scheme and all treatment criteria on this form are complied with.
7. The patient has an ECOG performance status (PS) of 0 or 1 or 2 or 3 and is fit enough for treatment with avapritinib.
8. Avapritinib will be administered as monotherapy.
9. Avapritinib will be continued until loss of clinical benefit or the development of unacceptable toxicity or withdrawal of patient consent, whichever occurs first.
10. The prescribing clinician is aware of the need for caution and potential dose changes in the prescribing of avapritinib with strong or moderate CYP3A inhibitors and inducers, as set out in the avapritinib Summary of Product Characteristics (SPC).
11. The prescribing clinician is aware that before initiating treatment with avapritinib the risk of intracranial haemorrhage should be carefully considered in patients with relevant risk factors such as severe thrombocytopenia, vascular aneurysm and a history of intracranial haemorrhage, stroke or TIA. 9 9
12. The prescribing clinician is aware that 2-weekly full blood counts are necessary for the first 8 weeks of treatment, then 2-weekly if the platelet count is <75 x 10 /L, 4-weekly if the platelet count is 75-100 x 10 /L and as required 9 if the platelet count is >100 x 10 /L.
13. A formal medical review as to how avapritinib is being tolerated and whether avapritinib should continue or not will be scheduled to occur at least by the end of the second 4-weekly cycle of treatment.
14. When a treatment break of more than 6 weeks beyond the expected 4-weekly cycle length is needed, a treatment break approval form will be completed to restart treatment.
15. Avapritinib will otherwise be used as set out in its Summary of Product Characteristics (SPC).

NHS funded From: 04 February 2025

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CDF Managed Access: NA

NICE Technology Appraisal: TA1012 (06 November 2024)

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