Navepegritide Medicine 05 December 2023 Extended indication Treatment of achondroplasia in children Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Navepegritide Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Other metabolism and Endocrinology Extended indication Treatment of achondroplasia in children Current proprietary name Already available biosimilars / generics Proprietary name TransCon CNP Manufacturer Ascendis Portfolio holder Ascendis Mechanism of action Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise UMC Utrecht Skeletal Dysplasia unit. Erasmus Rotterdam(Toekomstig). Additional remarks TransCon CNP is an investigational long-acting prodrug of C-type natriuretic peptide (CNP). Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date October 2025 Expected Registration November 2026 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Op dit moment is er geen behandeling voor achondroplasia. Vosoritide is wel geregistreerd voor patiënten ouder dan twee jaar, maar nog niet op de markt. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De data dient nog afgewacht te worden. Het lijkt wel zo te zijn dat het geneesmiddel effectiever is des te jonger de patiënt is. De verwachting is dat met name neurologische problemen voorkomen kunnen worden. Duration of treatment Frequency of administration 1 times a week Dosage per administration 100μg/kg References NCT04085523; Breinholt, VM, Mygind, PH, Christoffersen, ED, et al. Phase 1 safety, tolerability, pharmacokinetics and pharmacodynamics results of a long-acting C-type natriuretic peptide prodrug, TransCon CNP. Br J Clin Pharmacol. 2022; 88( 11): 4763- 4772. doi:10.1111/bcp.15369 Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Birth prevalence of achondroplasia: A systematic literature review and meta‐analysis. Pamela K. Foreman. Am J Med Genet A. 2020 (1); Additional remarks De incidentie van achondroplasie wordt wereldwijd geschat op ongeveer 1 op de 25.000 levendgeborenen. Dit betekent dat er een prevalente pool van ongeveer 135 patiënten van 0 tot 17 jaar oud is. Door de fabrikant van vosoritide werden er zeven nieuwe patiënten verwacht met achondroplasie per jaar in Nederland op basis van een expertopinie. 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