Somapacitan Medicine 04 December 2025 Extended indication Somapacitan monotherapy for treatment of Short stature in children and adolescents either born small for gestational age or with Turnerʼs syndrome, Noonan syndrome, or idiopathic short stature. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Somapacitan Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Metabolic diseases Extended indication Somapacitan monotherapy for treatment of Short stature in children and adolescents either born small for gestational age or with Turnerʼs syndrome, Noonan syndrome, or idiopathic short stature. Current proprietary name Already available biosimilars / generics Proprietary name Sogroya Manufacturer Novo Nordisk Portfolio holder Novo Nordisk Mechanism of action Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date December 2025 Expected Registration February 2026 Orphan drug 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 Er is nog onvoldoende data bekend. In de studie wordt het effect en de veiligheid van eenmaal per week toegediende somapacitan vergeleken met dagelijkse Norditropin®. Als hier een gelijke waarde uit blijkt dan is met name het toegenomen gebruiksgemak een voordeel. Duration of treatment Frequency of administration 1 times a day Dosage per administration References NCT05330325 (REAL 8); NCT05723835 (REAL 9) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Tijtgat et al., J Pediatr Endocrinol Metab, 2022. Additional remarks Jaarlijks worden circa 2.500 kinderen in Nederland verwezen voor evaluatie van korte lengte. Het zal waarschijnlijk gaan om een kleine subset van kinderen met een forse groeiachterstand (SD -2,5) die zich nog niet in de puberteit bevinden. 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