Mitapivat Medicine 08 June 2021 Extended indication Pyruvate kinase deficiency in transfusion-dependent and transfusion-independent adults Therapeutic value Possible added value Total cost Registration phase Registration application pending Product Active substance Mitapivat Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Pyruvate kinase deficiency in transfusion-dependent and transfusion-independent adults Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Agios Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Unknown Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Werkingsmechanisme: "First-in-class, small molecule stimulator of pyruvate kinase". Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date July 2021 Expected Registration August 2022 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Geen. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Er zijn nog geen huidige behandelingen en daardoor kan mitapivat belangrijk zijn in het verhogen van de Hb-waarde en het voorkomen van transfusies en van patiënten. De eerste eindpunten van de fase 3 studie zijn behaald. Verdere resultaten worden afgewacht. Duration of treatment Frequency of administration 2 times a day Dosage per administration 5mg-50mg References NCT03548220 Additional remarks Expected patient volume per year Patient volume < 50 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References orphanet Additional remarks De prevalentie is ongeveer 1-9 op de 100.000. Er wordt verwacht dat er in Nederland maximaal 50 personen in aanmerking zullen komen. 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 Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Sikkelcel ziekte References Adisinsight Additional remarks Lopende fase 1 studie. Indien mitapivat geregistreerd wordt voor deze indicatie zal het patiëntvolume een aanzienlijk groter volume krijgen. Other information Additional remarks