Active substance
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Stomach cancer
Extended indication
Treatment of patients with unresectable or metastatic gastrointestinal stromal tumors (GIST)

1. Product

Blueprint Medicines
Mechanism of action
Tyrosine kinase inhibitor
Route of administration
Therapeutical formulation
Budgetting framework
Intermural (MSZ)

2. Registration

Registration route
Centralised (EMA)
Type of trajectory
Normal trajectory
Submission date
July 2019
Expected Registration
August 2020
Orphan drug
Registration phase
Registration application pending
Additional comments
Estimated Primary Completion Date 04-2021 phase III trial.

3. Therapeutic value

Current treatment options
imatinib, sunitinib en regorafenib
Therapeutic value
No judgement
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%.
Duration of treatment
Not found
Frequency of administration
1 times a day
Dosage per administration
300 mg
NCT03465722 (VOYAGER)

4. Expected patient volume per year

Patient volume


Market share is generally not included unless otherwise stated.

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.

5. Expected cost per patient per year

< 350,000
Additional comments
In de Verenigde Staten is de prijs voor avapritinib gesteld op $32.000 per maand ($384.000 per jaar).

6. 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.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

Indication extension
Additional comments
Ook onderzocht bij: Solide tumoren en Systemic mastocytosis. Echter, nog geen fase III studies dus komende 2 jaar deze indicatieuitbreidingen nog niet verwacht.

9. Other information

There is currently no futher information available.