Avapritinib

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.
Therapeutic value	No estimate possible yet
Total cost	1,050,000.00
Registration phase	Registered

Product

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

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
Submission date	July 2019
Expected Registration	September 2020
Orphan drug	Yes
Registration phase	Registered
Additional remarks	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	3 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 remarks	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.

Patient volume

3

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 remarks

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 remarks	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	1,050,000.00 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.

Total cost

1,050,000.00

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 remarks	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.