Pexidartinib Medicine 13 December 2018 Extended indication Pigmented villo-nodular synovitis (PVNS) and giant cell tumour of tendon sheath 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 Pigmented villo-nodular synovitis (PVNS) and giant cell tumour of tendon sheath Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Plexxikon Portfolio holder Mechanism of action CFS1R inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Unknown 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 May 2020 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options 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 5 times a day Dosage per administration 200 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 Additional remarks Other information Additional remarks