Extended indication AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metasta
Therapeutic value No estimate possible yet
Total cost 1,050,000.00
Registration phase Registered and reimbursed


Active substance Avapritinib
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication Stomach cancer
Extended indication 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.
Proprietary name Ayvakyt
Manufacturer Blueprint Medicines
Mechanism of action Tyrosine kinase inhibitor
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)


Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date July 2019
Expected Registration September 2020
Orphan drug Yes
Registration phase Registered and reimbursed
Additional comments Positieve CHMP-opinie in juli 2020.

Therapeutic value

Current treatment options imatinib, sunitinib en regorafenib
Therapeutic value No estimate possible yet
Substantiation In de VOYAGER fase 3 studie werd een PFS van 4,2 maanden in de avapritinib groep versus 5,6 maanden in de regorafenib groep gevonden, een niet statistisch significant verschil. De overall response rate (ORR) was 17% versus 7%.
Frequency of administration 1 times a day
Dosage per administration 300 mg
References NCT03465722 (VOYAGER)

Expected patient volume per year

Patient volume


Market share is generally not included unless otherwise stated.

References NKR; Mei L. Gastrointestinal Stromal Tumors: The GIST of Precision Medicine. Trends Cancer 2018.; Lasota J. KIT and PDGFRA mutations in gastrointestinal stromal tumors (GISTs). Semin Diagn Pathol 2006.
Additional comments In 2016 waren er 50 diagnoses van GIST stadium IV. Avapritinib wordt vanwege de studieresultaten enkel verder ontwikkeld voor GIST met een PDGFRa exon 18 mutatie. In ongeveer 6% van alle patiënten met GIST wordt deze mutatie aangetoond. Dit betekent dat het patiëntvolume in Nederland rond de 3 patiënten zal bedragen.

Expected cost per patient per year

Cost < 350,000.00
References https://www.medscape.com/viewarticle/923624
Additional comments In de Verenigde Staten is de prijs voor avapritinib gesteld op $32.000 per maand ($384.000 per jaar).

Potential total cost per year

Total cost


This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References AdisInsight
Additional comments Ook onderzocht bij: Solide tumoren en Systemic mastocytosis. Echter, nog geen fase 3 studies dus komende 2 jaar deze indicatie uitbreidingen nog niet verwacht.

Other information

There is currently no futher information available.