Pexidartinib

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.

1. Product

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.

2. Registration

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.

3. 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.

4. Expected patient volume per year

Patient volume

< 30

Market share is generally not included unless otherwise stated.

References
NKR; kanker.nl
Additional comments
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.

5. Expected cost per patient per year

There is currently nothing known about the expected cost.

6. Potential total cost per year

There is currently nothing known about the possible total cost.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

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

9. Other information

There is currently no futher information available.