Elamipretide Medicine 10 December 2019 Extended indication Elamipretide monotherapy for treatment of primary Mitochondrial myopathy in adults and elderly with pathogenic nuclear DNA mutations. Therapeutic value No estimate possible yet 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 primary Mitochondrial myopathy in adults and elderly with pathogenic nuclear DNA mutations. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Stealth Portfolio holder Mechanism of action Other Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date August 2025 Expected Registration September 2026 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Het staat nog ter discussie of deze er gaat komen. De MMPOWER-3 klinische studie toonde geen significant voordeel van elamipretide behandeling in een genotypisch diverse populatie van volwassenen met primaire mitochondriale myopathie (PMM). Duration of treatment Frequency of administration 1 times a day Dosage per administration 60 mg References NCT05162768 (NuPOWER) (1); Expertopinie (2). 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 Spierziekten.nl (1); Expertopinie (2) Additional remarks In Nederland zijn er ongeveer 400 tot 500 mensen geregistreerd met een mitochondriële aandoening (1). Het zal om tientallen patiënten gaan (2). 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 Fase 3 voor mitochondrial myopathies References AdisInsight Additional remarks Other information Additional remarks