Lonapegsomatropine Medicine 03 June 2025 Extended indication Lonapegsomatropin monotherapy for treatment of adult-onset Growth hormone deficiency in adults over 23 years of age and elderly. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Lonapegsomatropine Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Unknown Extended indication Lonapegsomatropin monotherapy for treatment of adult-onset Growth hormone deficiency in adults over 23 years of age and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Skytrofa Manufacturer Ascendis Portfolio holder Mechanism of action Receptor agonist Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date 2025 Expected Registration July 2025 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Expertopinie Additional remarks De frequentie van toedienen is lager dan die van huidige groeihormonen. Hierdoor is het makkelijker voor patiënten. Echter, zijn er wel meer bijwerkingen. Expected patient volume per year Patient volume < 340 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.ntvg.nl/system/files/publications/a4288.pdf?utm_source=chatgpt.com Additional remarks Groeihormoondeficiëntie (GHD) bij volwassenen is een zeldzame aandoening. In Nederland wordt de prevalentie geschat op ongeveer 175 gevallen per 1.000.000 mensen. In 2023 werden groeihormonen bij 340 patiënten voorgeschreven. 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 Unknown Indication extensions References Additional remarks Other information Additional remarks