Pembrolizumab

Extended indication	Amyotrophic lateral sclerosis (ALS), add on in patients on a stable dose of riluzole.
Therapeutic value	No estimate possible yet
Registration phase	Registration application pending

Product

Active substance	Masitinib
Domain	Neurological disorders
Reason of inclusion	New medicine (specialité)
Main indication	ALS
Extended indication	Amyotrophic lateral sclerosis (ALS), add on in patients on a stable dose of riluzole.
Proprietary name	Alsitek
Manufacturer	AB Science
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)
Additional remarks	Al als diergeneesmiddel op de markt sinds 2009 voor behandeling van mast-cel tumoren (Masivet).

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	August 2022
Expected Registration	September 2023
Orphan drug	Yes
Registration phase	Registration application pending
Additional remarks	Negatieve opinie CHMP april 2018. Herbeoordeling gestart in april 2018. Fabrikant heeft de aanvraag tot herbeoordeling ingetrokken in mei 2018. Opnieuw ingediend bij de EMA.

Therapeutic value	No estimate possible yet
Duration of treatment	continuous
Frequency of administration	1 times a day
Dosage per administration	4,5 of 6,0 mg/kg lichaamsgewicht
References	NCT03127267
Additional remarks	masitinib (1x daags) wordt samen gegeven met 50mg riluzole (2x daags)

Patient volume	< 1,280 Market share is generally not included unless otherwise stated.
References	GIP databank
Additional remarks	1.280 gebruikers riluzol (GIP databank 2021), maximaal volume.

Patient volume

< 1,280

Market share is generally not included unless otherwise stated.

References

GIP databank

Additional remarks

1.280 gebruikers riluzol (GIP databank 2021), maximaal volume.

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

Indication extension	Yes
Indication extensions	Onderzocht in verschillende indicatiegebieden. De eerstvolgende indicatie-uitbreidingen zijn op de scan opgenomen.

There is currently no futher information available.