Migalastat Medicine 03 June 2025 Extended indication Migalastat monotherapy for treatment of Fabry’s disease in adults and elderly with amenable GLA variants and end-stage renal disease who are receiving either standard hemodialysis or hemodiafiltration. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Migalastat Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Metabolic diseases Extended indication Migalastat monotherapy for treatment of Fabry’s disease in adults and elderly with amenable GLA variants and end-stage renal disease who are receiving either standard hemodialysis or hemodiafiltration. Current proprietary name Already available biosimilars / generics Proprietary name Galafold Manufacturer Amicus Portfolio holder Mechanism of action Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise AMC (SPHINX, Amsterdam lysosome center) Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date March 2027 Expected Registration October 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT04020055 (AT1001-025); Expertopinie Additional remarks Dit middel wordt niet vergoed voor Fabry. Er loopt een verkenning voor een voorlopig toelating traject. Niemand komt in aanmerking voor enzymbehandeling want er is risico op blijvende orgaanschade. Dit middel wordt wel in het buitenland gegeven. Er is een laag niveau van bewijs. Bij dit risico op orgaanschade is er geen meerwaarde. Expected patient volume per year Patient volume < 0 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie Additional remarks In Nederland wordt de prevalentie van de ziekte van Fabry geschat op ongeveer 1 op 40.000 tot 1 op 117.000 mensen, wat betekent dat er naar schatting tussen de 150 en 250 mensen met Fabryʼs disease zouden kunnen zijn. De verwachting van de werkgroep is dat het niet ingezet gaat worden. 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