Dordaviprone Medicine 03 December 2024 Extended indication Dordaviprone monotherapy for treatment of newly diagnosed H3 K27M-mutant diffuse glioma in children, adolescents, adults and elderly following completion of radiotherapy. Therapeutic value No estimate possible yet Total cost € 1,008,000.00 Registration phase Clinical trials Product Active substance Dordaviprone Domain Oncology Reason of inclusion New medicine (specialité) Main indication Brain cancer Extended indication Dordaviprone monotherapy for treatment of newly diagnosed H3 K27M-mutant diffuse glioma in children, adolescents, adults and elderly following completion of radiotherapy. Current proprietary name Already available biosimilars / generics Proprietary name Modeyso Manufacturer Jazz Portfolio holder Jazz Mechanism of action Other, see general comments Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Werkingsmechanisme: dopamine receptor D2 antagonist / caseinolytic mitochondrial matrix peptidase proteolytic subunit agonist Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date November 2026 Expected Registration December 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Indieningsdatum en verwachte registratie aan de hand van IHSI-inschatting. Therapeutic value Current treatment options Bestraling Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times every 2 weeks Dosage per administration 625mg References NCT05580562 (ACTION) Additional remarks Patiënten met een gewicht van meer dan 52,5kg: 625mg eenmaal per een of twee weken. Patiënten met een gewicht van minder dan 52,5 kg: een dosering (en bijbehorende aantal capsules) aangepast aan lichaamsgewicht en afgerond tot incrementen van 125mg. Expected patient volume per year Patient volume 8 - 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References El-Khouly. PhD-Thesis Vrije Universiteit Amsterdam. 2022 (1) Additional remarks Jaarlijks krijgen acht tot tien kinderen in Nederland de diagnose diffuus intrinsiek ponsglioom (DIPG) (1). Expected cost per patient per year Cost € 112,000.00 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 (1) drugs.com; (2) jazzpharma.com/pi/modeyso.en.USPI.pdf; (3) IHSI inschatting op basis van dordviprine gebruik Additional remarks Er is nog geen Nederlandse lijstprijs bekend. In de Verenigde Staten kost een verpakking van 10 capsules (125mg) $31.265,50 (1). Uitgaande van een wekelijkse dosering van 625mg (2) en een gemiddelde behandelduur van 56 dagen (3) worden de kosten per patiënt ingeschat op $130,000 (ofwel €112.000). Potential total cost per year Total cost € 1,008,000.00 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