Mavacamten Medicine 03 June 2025 Extended indication Mavacamten for treatment of symptomatic (NYHA class II or III) non-obstructive hypertrophic cardiomyopathy in adults. Therapeutic value No estimate possible yet Total cost € 31,500,000.00 Registration phase No registration expected Product Active substance Mavacamten Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other medication for cardiovascular diseases Extended indication Mavacamten for treatment of symptomatic (NYHA class II or III) non-obstructive hypertrophic cardiomyopathy in adults. Current proprietary name Already available biosimilars / generics Proprietary name Camzyos Manufacturer BMS Portfolio holder BMS Mechanism of action Other, see general comments Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Small-molecule, selective allosteric inhibitor of cardiac myosin ATPase. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date 2025 Expected Registration 2026 Orphan drug No Registration phase No registration expected Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Medicijnen die de symptomen bestrijden. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Het geneesmiddel laat bij eerdere indicaties een verbetering van de kwaliteit van leven zien. Hierdoor is er minder septum ablatie therapie nodig. Deze indicatie gaat over het toedienen voordat de obstructie ontstaat. Resultaten moeten afgewacht worden. Duration of treatment Frequency of administration 1 times a day Dosage per administration 2,5mg, 5mg, 10mg of 15mg References ODYSSEY-HCM (NCT05582395) Additional remarks Expected patient volume per year Patient volume < 1,800 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References (1) CBS, (2) ZIN pakketadvies mavacamten (Camzyos) 1 mei 2024, (3) Massera et al. How common is hypertrophic cardiomyopa-thy …. Really? Disease prevalence revisited 27 years after CARDIA. International Journal of Cardiology, 2023;382:64-67., (4) Lu et al. Clinical Outcomes in Patients With Nonobstructive, Labile, and Obstructive Hypertrophic Cardiomyopathy. J Am Heart Assoc, 2018;7(5):e006657. doi: 10.1161/JAHA.117.006657., (5) Schoonvelde et al. Loss of quality of life and increased societal costs in patients with hypertrophic cardiomyopathy: the AFFECT-HCM study. Eur Heart J Qual Care Clin Outcomes, 2024:qcae092. Additional remarks De gehele volwassen populatie in Nederland was (>18 jaar) was ongeveer 14,6 miljoen op 1 januari 2024(1). De klinische prevalentie van HCM bedraagt 0,058% n=8.468 (2,3). Ongeveer 69% van de klinisch gediagnosticeerde HCM patiënten hebben non-obstructieve HCM n=5.843 (2). Van de populatie patiënten met non-obstructieve HCM, heeft zo'n 35% NYHA functionele klasse II en III n=2.045 (5). Ongeveer 88% van de patiënten met non-obstructieve HCM en NYHA functionele klasse II en III heeft een LVEF≥55% n=1,800 (7). Dit betekent dat er maximaal 1.800 non-obstructieve HCM patiënten in aanmerking zullen komen voor mavacamten. Expected cost per patient per year Cost € 17,500.00 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 G-standaard januari 2025 Additional remarks De prijs in Nederland is €1.340,70 per verpakking met 28 harde capsules voor alle sterktes (2,5mg; 5mg; 10mg; 15mg). Kosten per patiënt per jaar zijn €1.340,70/28*365,25= €17.488,95. Potential total cost per year Total cost € 31,500,000.00 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