Mercaptamine Medicine 03 December 2024 Extended indication Cystadrops is indicated for the treatment of corneal cystine crystal deposits in adults and children from 6 months of age with cystinosis. Therapeutic value Possible added value Total cost Registration phase Registered Product Active substance Mercaptamine Domain Neurological disorders Reason of inclusion Indication extension IND Main indication Eye disorders Extended indication Cystadrops is indicated for the treatment of corneal cystine crystal deposits in adults and children from 6 months of age with cystinosis. Current proprietary name Already available biosimilars / generics Proprietary name Cystadrops Manufacturer Recordati Portfolio holder Mechanism of action 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 zes maanden tot twee jaar. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date November 2024 Expected Registration June 2025 Orphan drug Yes Registration phase Registered Reimbursement Reimbursed from the basic package Medicine sluice Additional remarks Positieve CHMP-opinie in april 2025. Therapeutic value Current treatment options Alle patienten gebruiken nu cysteamine oogdruppels. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Mercaptamine (cysteamine) oogdruppels moeten vele malen per dag toegediend worden. Cystadrops kunnen twee tot vier keer per dag worden toegediend, omdat het middel visceuzer is. NB: beperkte kwaliteit van de studies: ongeblindeerd omdat de viscositeit blindering onmogelijk maakte. NB: concentratie vergelijkende middel laag (mercaptamine 0,10%; gebruikelijk in Nederland: 0,55%; cystadrops is 0,5%). Twee studies: 1) Open-label, 6 mo- 60 mo; safety dosing; n = 8 vnl kinderen; uitkomst: daling IVCM score (kristal deposit score) van 11,4 naar 7,9. 2) open label randomised versus CH 0,10%: 3 mo: 32 patiënten (15 versus 16): 4 dd: uitkomst IVCM score -4,6 versus -0,46 (p< 0.0001). Opmerking: is dus vergeleken met lage dosering en niet de gebruikelijke dosering van 0,55%. 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 Declaratiedata Additional remarks Van 2019 t/m 2023 waren er tussen de 47 en 52 gebruikers van Cystadrops. Het patiëntaantal dat in aanmerking komt voor deze indicatieuitbreiding van zes maanden tot twee jaar zal gering zijn. Expected cost per patient per year Cost € 43,700.00 - 45,200.00 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 tot en met 2023 was de gemiddelde vergoeding per gebruiker Cystadrops €43.728 tot €45.117. 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