Academic Journal
Navepegritide: First Approval.
| Τίτλος: | Navepegritide: First Approval. |
|---|---|
| Συγγραφείς: | Kang C; Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com. |
| Πηγή: | Drugs [Drugs] 2026 Aug; Vol. 86 (8), pp. 1385-1391. Date of Electronic Publication: 2026 Jun 03. |
| Τύπος έκδοσης: | Journal Article; Review |
| Γλώσσα: | English |
| Στοιχεία περιοδικού: | Publisher: Adis, Springer International Country of Publication: New Zealand NLM ID: 7600076 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1179-1950 (Electronic) Linking ISSN: 00126667 NLM ISO Abbreviation: Drugs Subsets: MEDLINE |
| Imprint Name(s): | Publication: Auckland : Adis, Springer International Original Publication: New York, ADIS Press [etc.] |
| Ιατρικοί όροι (MeSH): | Natriuretic Peptide, C-Type*/therapeutic use , Natriuretic Peptide, C-Type*/administration & dosage , Natriuretic Peptide, C-Type*/pharmacology , Natriuretic Peptide, C-Type*/adverse effects , Natriuretic Peptide, C-Type*/pharmacokinetics , Achondroplasia*/drug therapy , Limb Deformities, Congenital*/drug therapy, Receptor, Fibroblast Growth Factor, Type 3/metabolism ; Receptor, Fibroblast Growth Factor, Type 3/antagonists & inhibitors ; Bone and Bones/abnormalities ; Prodrugs/therapeutic use ; Humans ; Drug Approval ; United States ; Dwarfism ; Lordosis |
| Περίληψη: | Navepegritide (YUVIWEL®) is a prodrug of C-type natriuretic peptide (CNP) developed by Ascendis Pharma for the treatment of achondroplasia and is being investigated for hypochondroplasia. Navepegritide is administered once weekly and designed to provide sustained release and continuous exposure of active CNP, which is needed to counteract the overactive fibroblast growth factor receptor 3 signaling in achondroplasia, resulting in improved growth velocity. Navepegritide recently received accelerated approval in the USA for increasing linear growth in paediatric patients aged ≥ 2 years with achondroplasia with open epiphyses. This article summarizes the milestones in the development of navepegritide leading to this first approval for achondroplasia. (© 2026. Springer Nature Switzerland AG.) |
| Competing Interests: | Declarations. Authorship and Conflict of Interest: During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Connie Kang is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to this article and are responsible for its content. Ethics Approval, Consent to Participate, Consent to Publish, Availability of Data and Material, Code Availability: Not applicable. |
| References: | McDonnell C, Hove HB, Irving M, et al. Longitudinal observation of children with achondroplasia: findings from a global natural history study (ACHieve). Horm Res Paediatr. 2026. https://doi.org/10.1159/000550169 . Ascendis Pharma. YUVIWEL<sup>®</sup> (navepegritide) for injection, for subcutaneous use: US prescribing information. 2026. https://ascendispharma.us/products/pi/yuviwel/yuviwel_pi.pdf . Accessed 9 Apr 2026. Ascendis Pharma. FDA approves once-weekly YUVIWEL<sup>®</sup> (navepegritide) for children with achondroplasia aged 2 years and older [media release]. Feb 27 2026. https://investors.ascendispharma.com/news-releases/news-release-details/fda-approves-once-weekly-yuviwelr-navepegritide-children . Ascendis Pharma. Ascendis Pharma provides business and strategic roadmap update at 43rd Annual J.P. Morgan Healthcare Conference [media release]. 12 Jan 2025. https://ascendispharma.com/ . Ascendis Pharma. Ascendis Pharma announces formation of VISEN Pharmaceuticals to develop and commercialize TransCon<sup>TM</sup> endocrinology rare disease therapies in China [media release]. 8 Nov 2018. http://www.ascendispharma.com . Ascendis Pharma. Ascendis Pharma announces strategic partnership with Teijin Limited in Japan [media release]. 29 Nov 2023. http://www.ascendispharma.com . Specialised Therapeutics. Specialised Therapeutics signs exclusive agreement with Ascendis Pharma A/S for distribution and commercialisation of three endocrinology therapies in Australia and select South-East Asia countries [media release]. 1 Jul 2024. http://www.stbiopharma.com . Er-Kim. Er-Kim extends exclusive distribution agreement with Ascendis Pharma A/S to expand commercialization of its endocrinology portfolio to Eurasian markets [media release]. 10 Dec 2024. http://www.er-kim.com . United States Securities and Exchange Commission. SEC File 2023 - Ascendis Pharma. 2023. https://www.sec.gov/ix?doc=/Archives/edgar/data/1612042/000095017023003068/asnd-20221231.htm . Accessed 10 Apr 2026. Breinholt VM, Rasmussen CE, Mygind PH, et al. 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. 2019;370(3):459–71. (PMID: 10.1124/jpet.119.25825131235532) Savarirayan R, Hoernschemeyer DG, Ljungberg M, et al. Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial. EClinicalMedicine. 2023;65:102258. (PMID: 10.1016/j.eclinm.2023.1022583782303110562841) 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):4763–72. (PMID: 10.1111/bcp.15369354817079796269) Savarirayan R, McDonnell C, Bacino CA, et al. Once-weekly navepegritide in children with achondroplasia: the APPROACH randomized clinical trial. JAMA Pediatr. 2026;180(1):18–25. (PMID: 10.1001/jamapediatrics.2025.47714124775412624480) Visen Pharmaceuticals. Once-weekly TransCon(tm) CNP achieved primary efficacy objective in ACcomplisH China phase 2 trial [media release]. 16 Nov 2023. http://www.visenpharma.com . VISEN Pharmaceuticals. Navepegritide achieved primary efficacy endpoint with sustained clinical benefits in Chinese children with achondroplasia: 104 weeks topline results in ACcomplisH China phase 2 trial [media release]. 15 Aug 2025. https://www.visenpharma.com/en/News/NewsDetail/20250815_293.html . McDonnell M, Irving M, Nolting LA, et al. Once-weekly lonapegsomatropin (TransCon hGH) added to once-weekly navepegritide (TransCon CNP) in children with achondroplasia: 26-week results from the phase 2 COACH trial. In: The American Society for Bone and Mineral Research. 2025. Ascendis Pharma. Week 52 COACH trial topline results confirm consistent and durable treatment benefits in children with achondroplasia (ACH) [media release]. 9 Jan 2026. http://www.ascendispharma.com . |
| Substance Nomenclature: | 127869-51-6 (Natriuretic Peptide, C-Type) EC 2.7.10.1 (Receptor, Fibroblast Growth Factor, Type 3) 0 (Prodrugs) |
| SCR Disease Name: | Hypochondroplasia |
| Entry Date(s): | Date Created: 20260603 Date Completed: 20260716 Latest Revision: 20260716 |
| Update Code: | 20260717 |
| DOI: | 10.1007/s40265-026-02341-x |
| PMID: | 42234372 |
| Βάση Δεδομένων: | MEDLINE |
| ISSN: | 1179-1950 |
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| DOI: | 10.1007/s40265-026-02341-x |