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Synonyms: TransCon CNP
Compound class:
Peptide
Comment: Navepegritide (TransCon CNP) is a prodrug of a human C-type natriuretic peptide (CNP) analogue [1]. It comprises CNP(1-38) attached to an inert polyethylene glycol carrier via a cleavable linker [3]. CNP is a validated therapeutic agent for the treatment of patients with achondroplasia (see the approved CNP analogue vosoritide) [2]. Navepegritide is designed to provide sustained hormone release and continuous CNP exposure as a once-weekly therapy that is proposed to counterbalance the overactivated FGFR3 signaling pathway in achondroplasia [1].
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| References |
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1. Breinholt VM, Rasmussen CE, Mygind PH, Kjelgaard-Hansen M, Faltinger F, Bernhard A, Zettler J, Hersel U. (2019)
TransCon CNP, a Sustained-Release C-Type Natriuretic Peptide Prodrug, a Potentially Safe and Efficacious New Therapeutic Modality for the Treatment of Comorbidities Associated with Fibroblast Growth Factor Receptor 3-Related Skeletal Dysplasias. J Pharmacol Exp Ther, 370 (3): 459-471. [PMID:31235532] |
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2. Galetaki DM, Merchant N, Dauber A. (2024)
Novel therapies for growth disorders. Eur J Pediatr, 183 (3): 1121-1128. [PMID:37831302] |
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3. Savarirayan R, Hoernschemeyer DG, Ljungberg M, Zarate YA, Bacino CA, Bober MB, Legare JM, Högler W, Quattrin T, Abuzzahab MJ et al.. (2023)
Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial. EClinicalMedicine, 65: 102258. [PMID:37823031] |
