Mitapivat Medicine 03 June 2025 Extended indication Mitapivat monotherapy for treatment of pyruvate kinase deficiency anaemia in infants and toddlers over 1 year of age, children and adolescents who are not regularly receiving blood transfusion. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Mitapivat Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other non-oncological hematological medications Extended indication Mitapivat monotherapy for treatment of pyruvate kinase deficiency anaemia in infants and toddlers over 1 year of age, children and adolescents who are not regularly receiving blood transfusion. Current proprietary name Already available biosimilars / generics Proprietary name Pyrukynd Manufacturer Avanzanite Portfolio holder Mechanism of action Route of administration Oral Therapeutical formulation Granulate Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Werkingsmechanisme: pyruvaat kinase activator Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New therapeutical formulation ATMP No Submission date June 2026 Expected Registration June 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 NCT05175105 (ACTIVATE-Kids) Additional remarks De ACTIVATE-Kids is een RCT, placebo-gecontroleerde studie in kinderen met PKD (1 tot 18 jaar, niet transfusie afhankelijk). Laat zien dat 32% van de kinderen het primaire eindpunt van de studie hebben gehaald (Hb stijging minder dan1,5 g/dL). Expected patient volume per year Patient volume < 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie Additional remarks De prevalentie is ongeveer 1 op 9 per 100.000. Er wordt verwacht dat er in Nederland maximaal 50 personen in aanmerking zullen komen. Het aandeel patiënten van 1 tot 18 jaar zal hier een klein stuk van zijn. De werkgroep verwacht maximaal tien patiënten. Expected cost per patient per year Cost References Mitapivat kost in de Verenigde Staten gemiddeld $26,075 (28 dagen tweemaal daags 5mg). De kosten zullen $339,906 per patiënt per jaar bedragen. Omgerekend kost het €316.000 per patiënt per jaar. 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