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Govorestat

Extended indication Sorbitol dehydrogenase deficiency
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Govorestat
Domain Metabolism and Endocrinology
Reason of inclusion New medicine (specialité)
Main indication Metabolic diseases
Extended indication Sorbitol dehydrogenase deficiency
Manufacturer Applied Therapeutics
Route of administration Oral
Budgetting framework Extramural (GVS)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date January 2025
Expected Registration February 2026
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Govorestat is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI).

Therapeutic value

Therapeutic value No estimate possible yet
Substantiation De trial loopt nog. De12 maanden interim analyse is nog niet gepubliceerd behalve in een press release van applied therapeutics, waarbij een significante afname van sorbitol spiegels wordt gerapporteerd en klinisch een trend op een samengesteld eindpunt, wat niet overeen lijkt te komen met het vooraf vastgestelde primaire eindpunt. Het primaire eindpunt is 10 minuten loop/ren test.
Frequency of administration 1 times a day
References INSPIRE (NCT05397665); https://www.clinicaltrialsregister.eu/ctr-search/search?query=AT-007.

Expected patient volume per year

Patient volume

< 0

Market share is generally not included unless otherwise stated.
References Amsterdam UMC ECZA
Additional remarks ECZA is het Amsterdam UMC voor neuromusculaire aandoeningen inclusief Charcot-Marie-Tooth. SORD is pas sinds 2020 ontdekt als oorzaak van neuropathie. Lijkt extreem zeldzaam: geen patiënten bekend.

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.