Venglustat Medicine 09 January 2018 Extended indication Ziekte van Fabry Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Venglustat Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Ziekte van Fabry Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Sanofi Portfolio holder Mechanism of action Enzyme inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Ook bekend onder de naam ibiglustat. Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP No Submission date 2026 Expected Registration 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Formeel nog geen weesgeneesmiddelstatus verkregen van de EMA. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Nog geen fase 3 resultaten bekend. Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertisecentrum Additional remarks In Nederland zijn ongeveer 300 patiënten met de ziekte van Fabry bekend. 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 Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks