Avapritinib Medicine 10 December 2019 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 and reimbursed 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. Current proprietary name Already available biosimilars / generics Proprietary name Ayvakyt Manufacturer Blueprint Medicines Portfolio holder Mechanism of action Tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date July 2019 Expected Registration September 2020 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in juli 2020. Therapeutic value Current treatment options imatinib, sunitinib en regorafenib Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. 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%. Duration of treatment Frequency of administration 1 times a day Dosage per administration 300 mg References NCT03465722 (VOYAGER) Additional remarks Expected patient volume per year Patient volume 3 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 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. 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 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions 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 Additional remarks