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 Clinical trials 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 Unknown 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 Unknown Particularity Unknown ATMP Unknown Submission date Expected Registration October 2019 Orphan drug No Registration phase Clinical trials 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 1000 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 - 50 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). 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 Unknown Indication extensions References Additional remarks Other information Additional remarks