Avapritinib Medicine 10 December 2019 Extended indication Treatment of patients with unresectable or metastatic gastrointestinal stromal tumors (GIST) Therapeutic value No judgement Total cost Registration phase Registration application pending Product Active substance Avapritinib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Stomach cancer Extended indication Treatment of patients with unresectable or metastatic gastrointestinal stromal tumors (GIST) Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Blueprint Medicines Portfolio holder Mechanism of action Tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Unknown 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 August 2020 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Estimated Primary Completion Date 04-2021 phase III trial. Therapeutic value Current treatment options imatinib, sunitinib en regorafenib Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration 300 mg References NCT03465722 Additional remarks Expected patient volume per year Patient volume 25 - 50 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR Additional remarks In 2016 waren er 50 diagnoses van GIST stadium IV. Inschatting: 25-50 patiënten per jaar, ervan uitgaande dat niet iedereen in de derdelijn behandeld wordt. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost 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 III studies dus komende 2 jaar deze indicatie-uitbreidingen nog niet verwacht. Other information Additional remarks