Active substance Pexidartinib
Domain Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen New medicine (specialité)
Main indication Other oncology
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.


Proprietary name Turalio
Manufacturer Plexxikon
Mechanism of action CFS1R inhibitor
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Intermural (MSZ)
Additional comments CFS1R tyrosine kinase remmer.


Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date April 2019
Expected Registration August 2020
Orphan drug No
Registration phase Registration application pending
Additional comments Fabrikant verwacht registratie in Q2 2020.

Therapeutic value

Current treatment options Chirurgie
Therapeutic value No judgement
Substantiation ENLIVEN trial shows reduced tumor size.
Duration of treatment Not found
Frequency of administration 2 times a day
Dosage per administration 400 mg
References NCT02371369
Additional comments 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.

References NKR;
Additional comments In 2016 werden er 28 diagnoses gesteld van wekedelensarcomen van overige en ongespecificeerde lokalisaties. Volgens 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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References adisinsight
Additional comments Lopende fase 2 studies.

Other information

There is currently no futher information available.