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Govorestat

Extended indication Patients with Classic Galactosaemia aged 2 to 65 years.
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Govorestat
Domain Metabolism and Endocrinology
Reason of inclusion New medicine (specialité)
Main indication Metabolic diseases
Extended indication Patients with Classic Galactosaemia aged 2 to 65 years.
Manufacturer Applied Therapeutics
Route of administration Oral
Budgetting framework Extramural (GVS)
Additional remarks Govorestat (AT-007) is a novel oral aldose reductase inhibitor that can cross the blood-brain barrier into the CNS.

Registration

Registration route Centralised (EMA)
ATMP No
Submission date 2024
Expected Registration 2025
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options Galactose-beperkt dieet.
Therapeutic value No estimate possible yet
Substantiation Er zijn nog geen gepubliceerde resultaten in peer-reviewed tijdschriften. De ACTION-Galactosemia Kids trial is uitgevoerd bij 47 kinderen (2 tot 17 jaar) en liet op het primaire eindpunt geen significant klinisch verschil zien (composite endpoint: sum of change comprised of four endpoints: OWLS-2 Oral Expression (OE), OWLS-2 Listening Comprehension (LC), BASC-3 Behavior Symptoms Index (BSI) and the BASC-3 Activities of Daily Living (ADL)).
References https://ir.appliedtherapeutics.com/news-releases/news-release-details/applied-therapeutics-announces-clinical-benefit-govorestat-007/
Additional remarks NCT05418829

Expected patient volume per year

Patient volume

< 180

Market share is generally not included unless otherwise stated.
References ECZA Amsterdam UMC
Additional remarks In Nederland zijn 180 patiënten bekend met deze vorm van galactosemie

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 Yes
Indication extensions Sorbitol Dehydrogenase (SORD) Deficiency; Phosphomannomutase 2 deficiency
References https://www.drugs.com/clinical_trials/applied-therapeutics-announces-positive-results-12-month-interim-analysis-govorestat-007-ongoing-21340.html

Other information

There is currently no futher information available.