Mitapivat Medicine 03 December 2024 Extended indication Treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassaemia Therapeutic value No estimate possible yet Total cost € 5,640,000.00 Registration phase Registration application pending Product Active substance Mitapivat Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other non-oncological hematological medications Extended indication Treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassaemia Current proprietary name Already available biosimilars / generics Proprietary name Pyrukynd Manufacturer Agios Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date December 2024 Expected Registration December 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Verwachte registratie op basis van IHSI-inschatting. Therapeutic value Current treatment options TDT: Bloedtransfusies, ijzerchelatie en luspatercept. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er zijn nog geen resultaten bekend van de fase 3 ENERGIZE en ENERGIZE-T studie. Duration of treatment Frequency of administration 2 times a day Dosage per administration 100 mg References NCT04770753 (ENERGIZE); NCT04770779 (ENERGIZE-T) Additional remarks Expected patient volume per year Patient volume 20 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie (1) Additional remarks Er zijn mogelijk rond de 100 transfusie-afhankelijke alfa- of bèta-thalassemie patiënten (TDT) en 100 niet-transfusie-afhankelijke alfa- of bèta-thalassemie patiënten (NTDT), het exacte aantal is niet bekend. Een ruwe inschatting is dat er 20 patiënten zijn (TDT en NTDT samen) die langdurig op mitapivat zullen staan (1). Expected cost per patient per year Cost € 282,000.00 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 GoodRX Additional remarks Een 28 daags voorraad van 5mg per tablet is in de Verenigde Staten $25.664.97. Jaarlijks zullen deze kosten neerkomen op $307,906 per patiënt per jaar. Omgerekend kost het €282.000 per patiënt per jaar. Mogelijk zullen de kosten in Nederland lager uitvallen. Potential total cost per year Total cost € 5,640,000.00 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