Extended indication Spinocerebellar degeneration
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Troriluzole
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Neurological disorders other
Extended indication Spinocerebellar degeneration
Manufacturer Biohaven
Portfolio holder Biohaven
Route of administration Oral
Budgetting framework Extramural (GVS)
Additional remarks Excitatory amino acid antagonist; Glutamate release inhibitor; Voltage-gated sodium channel inhibitor.

Registration

Registration route Centralised (EMA)
ATMP No
Submission date October 2023
Expected Registration November 2024
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options Er bestaat geen behandeling die de ziekteprogressie bij SCA afremt.
Therapeutic value No estimate possible yet
Substantiation De fase 3 studie is afgerond, maar nog niet gepubliceerd. (n=213, SCA 1,2,3,6,7,8,10). Persberichten melden geen statistisch significant verschil in fSARA (modified functional Scale for the Assessment and Rating of Ataxia) score. Er werden wel significant minder valpartijen bij de ambulante patiënten geconstateerd. In een subgroep van SCA3 patiënten zonder loophulpmiddelen/rolstoel lijkt wel een significant verschil in fSARA.
Dosage per administration 200mg
References NCT03701399

Expected patient volume per year

Patient volume

340 - 680

Market share is generally not included unless otherwise stated.

References erfelijkheid.nl;
Additional remarks Genetische cerebellaire ataxie omvat progressieve degeneratie van het cerebellum. Hun algemene prevalentie wordt geschat op 2 tot 4 gevallen per 100.000 mensen (1). SCA types 1,2,3,6,7,8,10 zijn geïncludeerd in de studie. Hieronder vallen de types die het meest voorkomen.

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.