Govorestat Medicine 04 June 2024 Extended indication Patients with Classic Galactosaemia aged 2 to 65 years. Therapeutic value No estimate possible yet Total cost Registration phase No registration expected 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. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Applied Therapeutics Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Unknown Budgetting framework Extramural (GVS) Centre of expertise 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) Type of trajectory Unknown Particularity Unknown ATMP No Submission date December 2023 Expected Registration 2025 Orphan drug Yes Registration phase No registration expected Reimbursement Medicine sluice Additional remarks Teruggetrokken januari 2025: the company stated that it needed more time to collect further data to support the assessment of Nugalviq in the proposed indication. Therapeutic value Current treatment options Galactose-beperkt dieet. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. 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)). Duration of treatment Frequency of administration Dosage per administration 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. Maximum patient volume for sluice References ECZA Amsterdam UMC Additional remarks In Nederland zijn 180 patiënten bekend met deze vorm van galactosemie. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions 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 Additional remarks Other information Additional remarks