NICE – Single Technology Appraisal (STA) – Avapritinib for treating unresectable or metastatic gastrointestinal stromal tumours – Terminated…

We are very disappointed to report that the NICE single technology appraisal of avapritinib (Ayvakit) for the treatment of unresectable or metastatic PDGFRA D842V-mutant GIST has been terminated.
PDGFRA D842V-mutant GIST is an ultra-rare form of GIST affecting 5-10 newly diagnosed GIST cancer patients each year in the UK.

After a significant amount of work from all parties over the past year, including representation from GIST Patients, Specialist GIST Clinicians and GIST Cancer UK, it has not been possible to reach agreement for the supply of avapritinib for GIST patients in the UK at this time. Blueprint Medicines remains committed to finding a way to work with NICE and NICE is open to reviewing the situation when more evidence has been gathered, as stated on the NICE website.

All patients currently receiving avapritinib through the named-patient access program will continue to receive treatment.

Doctors treating other PDGFRA D842V-mutant GIST patients will need to complete an NHS Individual funding request (IFR) to apply for access to prescribe avapritinib. Should this process be unsuccessful they should contact Blueprint Medicines directly at:

Global Contact Center


+31 85 064-4001
US & Corporate Contact Center

GIST Cancer UK will continue to work with all parties to find a solution.

Posted in GIST News, Medication, Treatment and tagged , .