Mercaptamine Medicine 03 December 2024 Extended indication Extension of indication to include treatment of children from 6 months of age with paediatric cystinosis Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Mercaptamine Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Eye disorders Extended indication Extension of indication to include treatment of children from 6 months of age with paediatric cystinosis Current proprietary name Already available biosimilars / generics Proprietary name Cystadrops Manufacturer Recordati Portfolio holder Mechanism of action Unknown Route of administration Ocular Therapeutical formulation Eye drops Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Mercaptamine kan neerslagen van cystine in de cornea verminderen. Dit betreft de indicatieuitbreiding voor 6 maanden tot 2 jaar. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date November 2024 Expected Registration September 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Verwachte registratie op basis van gemiddelde doorlooptijd van 10 maanden voor een indicatieuitbreiding. 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 4 times a day Dosage per administration 1 druppel in ieder oog References NCT04125927 (CYT-C2-00) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost € 43,728.00 - 45,117.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References GIPdatabank Additional remarks Van 2019 t/m 2023 was de gemiddelde vergoeding per gebruiker Cystadrops €43.728-€45.117. 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 Unknown Indication extensions References Additional remarks Other information Additional remarks