Eplontersen Medicine 04 June 2026 Extended indication Transthyretin amyloid cardiomyopathy (ATTR-CM). Therapeutic value No estimate possible yet Total cost € 158,800,000.00 Registration phase Clinical trials Product Active substance Eplontersen Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other medication for cardiovascular diseases Extended indication Transthyretin amyloid cardiomyopathy (ATTR-CM). Current proprietary name Already available biosimilars / generics Proprietary name Wainzua Manufacturer AstraZeneca Portfolio holder AstraZeneca Mechanism of action Other, see general comments Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Werkingsmechanisme: ligand‑geconjugeerd antisense‑oligonucleotide (LICA) Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP No Submission date December 2026 Expected Registration July 2027 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Tafamidis (hoge dosering) Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation CARDIO-TTRansform studie loopt nog. Duration of treatment Frequency of administration Dosage per administration References NCT04136171 Additional remarks Expected patient volume per year Patient volume < 400 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Grogan et al. J Card Fail. 2017 Aug;23(8):S73; GIPdatabank; expertopinie Additional remarks De verwachting van het aantal patiënten is vergelijkbaar met tafamidis. Volgens GIP data werden in 2023 circa 400 patiënten voor (ATTR-CM) behandeld met tafamidis (20 en 61mg). Werkelijke inschatting van aantal patiënten is lastig, vooral gezien het tijdsbestek en andere concurrenten. Er komen namelijk andere potentiële geneesmiddelen op de markt voor ATTR-CM, die nu worden onderzocht. Denk aan TTR-syntheseremmers (kleine iRNA moleculen) zoals patisiran (al goedgekeurd voor ATTR-PN) en vutrisiran en ook de andere antisense-oligonucleotide "inotersen". Expected cost per patient per year Cost € 397,000.00 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 medicijnkosten.nl Additional remarks De prijs per 0,8ml pen (56mg/ml) van eplontersen (Wainzua) is €33.083. Bij maandelijks gebruik komen de jaarlijkse behandelkosten uit op €396.996. Potential total cost per year Total cost € 158,800,000.00 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