Pexidartinib Medicine 13 December 2018 Extended indication Treatment of adult patients with symptomatic tenosynovial giant cell tumour (TGCT), also referred to as giant cell tumour of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS), which is associated with severe morbidity or functional limitations, and which is not amenable to improvement with surgery. Therapeutic value No judgement Total cost Registration phase Registration application pending Product Active substance Pexidartinib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Oncology other Extended indication Treatment of adult patients with symptomatic tenosynovial giant cell tumour (TGCT), also referred to as giant cell tumour of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS), which is associated with severe morbidity or functional limitations, and which is not amenable to improvement with surgery. Current proprietary name Already available biosimilars / generics Proprietary name Turalio Manufacturer Plexxikon Portfolio holder Mechanism of action CFS1R inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks CFS1R tyrosine kinase remmer. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date April 2019 Expected Registration August 2020 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Fabrikant verwacht registratie in Q2 2020. Therapeutic value Current treatment options Chirurgie Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation ENLIVEN trial shows reduced tumor size. Duration of treatment Frequency of administration 2 times a day Dosage per administration 400 mg References NCT02371369 Additional remarks Patiënten ontvangen 1.000 mg (5 capsules per dag) gedurende 2 weken, daarna 800 mg (4 capsules per dag) gedurende 22 weken. Expected patient volume per year Patient volume < 30 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR; kanker.nl Additional remarks In 2016 werden er 28 diagnoses gesteld van wekedelensarcomen van overige en ongespecificeerde lokalisaties. Volgens kanker.nl is de incidentie per jaar 2-3 patiënten per miljoen inwoners (34-51 in Nederland). De inschatting van de werkgroep is dat het aantal patiënten wat hiervoor in aanmerking komt verwaarloosbaar is. 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 Lopende fase 2 studies. Other information Additional remarks