Pexidartinib

Extended indication	Pigmented villo-nodular synovitis (PVNS) and giant cell tumour of tendon sheath
Therapeutic value	No judgement
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
Manufacturer	Plexxikon
Mechanism of action	CFS1R inhibitor
Route of administration	Oral
Therapeutical formulation	Capsule
Additional remarks	CFS1R tyrosine kinase remmer.

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
Submission date	April 2019
Expected Registration	May 2020
Orphan drug	No
Registration phase	Registration application pending

Therapeutic value

Therapeutic value	No judgement
Substantiation	ENLIVEN trial shows reduced tumor size.
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 Market share is generally not included unless otherwise stated.
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.

Patient volume

< 30

Market share is generally not included unless otherwise stated.

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

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

Other information

There is currently no futher information available.