Elamipretide Medicine 04 December 2025 Extended indication Elamipretide monotherapy for treatment of Barth syndrome in male adolescents, adults and elderly. Therapeutic value Total cost Registration phase Clinical trials Product Active substance Elamipretide Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Elamipretide monotherapy for treatment of Barth syndrome in male adolescents, adults and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Pharmanovia Portfolio holder Pharmanovia Mechanism of action Route of administration Subcutaneous Therapeutical formulation Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date December 2025 Expected Registration January 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT03098797 (TAZPOWER) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet Additional remarks In Nederland wordt het zeldzame Barth-syndroom naar schatting aangetroffen bij ongeveer 10 tot 80 jongens en mannen. Dit syndroom is een erfelijke aandoening die uitsluitend voorkomt bij mannen en wordt veroorzaakt door een mutatie op het X-chromosoom. De wereldwijde prevalentie is geschat op 1 tot 9 per miljoen inwoners, wat betekent dat ook in Nederland het aantal patiënten uiterst beperkt is. Door de zeldzaamheid is er beperkte informatie beschikbaar. 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 Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks