预约演示
更新于：2026-09-07
Navepegritide
那韦培肽
更新于：2026-09-07
概要
基本信息
非在研机构 |
最高研发阶段批准上市 |
首次获批日期 美国 (2026-02-27), |
最高研发阶段(中国)无进展 |
特殊审评加速批准 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (澳大利亚)、优先审评 (美国)、优先审评 (中国) |
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结构/序列
Sequence Code 1680846
来源: *****
关联
9
项与 那韦培肽 相关的临床试验JPRN-jRCT2061250056
A Multicenter, Open-Label, Uncontrolled, Single Arm Clinical Trial to Evaluate the Efficacy and Safety of ACP-015 Administered Subcutaneously Once Weekly for 52 Weeks in Children with Achondroplasia Aged 2 to 11 Years.
NCT06732895
A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of Navepegritide Administered Once Weekly for 52 Weeks in Adolescents (12-18 Years of Age) With Achondroplasia.
NCT06433557
A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia
100 项与 那韦培肽 相关的临床结果
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100 项与 那韦培肽 相关的专利（医药）
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8
项与 那韦培肽 相关的文献（医药）2026-08-01DRUGS
Navepegritide: First Approval
Review
作者: Kang, Connie
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.
2026-06-03Journal of the Endocrine Society
Achondroplasia management in the era of targeted therapies: a meta-analysis of C-type natriuretic peptide analogs
Review
作者: Uday, Suma ; Ashraf, Ambika P ; Pappachan, Joseph M ; Dutta, Deep ; Nagendra, Lakshmi ; Kamrul-Hasan, Abul Bashar Mohammad ; Bhattacharya, Saptarshi
Abstract:
Context:
No prior meta-analysis has systematically assessed efficacy and safety of C-type natriuretic peptide (CNP) analogs within the context of evolving understanding of FGFR3 biology and achondroplasia natural history.
Objective:
To evaluate the safety and efficacy of CNP analogs in children with achondroplasia and contextualize clinical outcomes and natural history.
Methods:
Systematic review of randomized control trials and real-world studies evaluating the safety and efficacy of CNP analogs (vosoritide and navepegritide) in children aged <18 years with genetically confirmed achondroplasia was performed. Coprimary outcomes of interest were adverse events (AEs) and changes from baseline in annualized growth velocity (AGV) at the end of the trials. Secondary outcomes included changes from baseline in height Z-score, standing height, and upper-to-lower body segment (ULS) ratio.
Results:
Eleven studies (N = 542) were included, of which 4 RCTs (n = 326) with low overall risk of bias were meta-analyzed. Overall and serious AE rates were comparable between CNP analogs and placebo, except for higher relative risks of injection site reactions (1.65), urticaria (4.04), and swelling (3.57). C-type natriuretic peptide analogs significantly increased mean differences in AGV (1.36 cm/year; 95% CI: 1.05-1.68; P < .00001) and standing height (1.24 cm; 95% CI: 0.47-2.01; P = .002), without short-term effect on ULS ratio. Real-world studies demonstrated sustained growth benefits with infrequent serious AEs or treatment discontinuations.
Conclusion:
C-type natriuretic peptide analogs provide slight but statistically meaningful improvements in linear growth in children with achondroplasia with acceptable short-term safety profile. Long-term studies are needed to define optimal timing of therapy on adult height, functional outcomes, and achondroplasia-related complications.
2026-06-01EUROPEAN JOURNAL OF ENDOCRINOLOGY
Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial
Article
作者: Dalby, Lene Worsaae ; Ominsky, Michael S ; Jensen, Simon Mejlhede ; Nolting, Line Aagaard ; Irving, Melita ; Komirenko, Allison S ; McDonnell, Ciara M ; Iklé, Jennifer M ; Abdelrahman, Sohair G ; Hove, Hanne B ; Shu, Aimee D
Abstract:
Objective:
Navepegritide, a prodrug of C-type natriuretic peptide, has demonstrated improved growth and additional benefits, including lower limb alignment, in children with achondroplasia. The well-described mechanism of action of navepegritide in the growth plate is hypothesized to enhance its receptivity to lonapegsomatropin, a prodrug of somatropin, to provide benefits beyond those seen with monotherapies in achondroplasia.
Methods:
In the proof-of-concept phase 2 COACH trial, children with achondroplasia aged 2 to 11 received weekly navepegritide and lonapegsomatropin. The primary efficacy endpoint was annualized growth velocity (AGV) at week 52 vs AGV with navepegritide monotherapy in the ApproaCH trial (NCT05598320).
Results:
Twenty-one children were enrolled: 12 received no prior growth-promoting treatment (treatment-naive, TN) and 9 had been receiving navepegritide monotherapy for >1 year (navepegritide-experienced, NE). At week 52, TN children receiving navepegritide and lonapegsomatropin had a least squares mean AGV of 8.69 cm/year versus 5.95 cm/year for matched children who received navepegritide monotherapy, a difference of 2.74 cm/year (P < .0001). Observed mean AGV for TN children was 8.80 cm/year, a +3.89 cm/year increase from baseline (P = .0015). NE children had an observed mean AGV of 8.42 cm/year, a +3.28 cm/year increase from navepegritide-treated baseline (P < .0001). An improvement in body proportionality was observed and arm span increased substantially (+9.4 cm in TN and +7.9 cm in NE children), in line with gains in height. Treatments were well-tolerated with generally mild adverse events and no treatment discontinuations.
Conclusions:
In this prospective trial, the robust growth and anthropometric results suggest that by continuously relieving the pre-hypertrophic block in achondroplasia, navepegritide may make the growth plate more responsive to the strong complementary effect of lonapegsomatropin.
Trial registration details:
ClinicalTrials.gov NCT06433557
100 项与 那韦培肽 相关的药物交易
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外链
| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| D13294 | - | - | - |
研发状态
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 软骨发育不全 | 美国 | 2026-02-27 | |
| 软骨发育不全 | 美国 | 2026-02-27 |
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临床结果
临床结果
适应症
分期
评价
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临床2/3期 | 84 | (Navepegritide) | 廠積壓鑰齋窪簾衊積膚(觸蓋遞鹽範願憲顧衊網) = 憲衊醖餘壓餘襯鹹夢鹹 選壓願築廠衊壓選憲範 (淵範鹹醖鑰積艱憲衊蓋, 遞築選衊鑰鹹鹹繭鬱獵 ~ 遞鹽廠窪壓醖繭繭築壓) 更多 | - | 2026-07-14 | ||
Placebo for Navepegritide (TransCon CNP) (Placebo for Navepegritide) | 廠積壓鑰齋窪簾衊積膚(觸蓋遞鹽範願憲顧衊網) = 顧積獵窪構餘鏇築觸鏇 選壓願築廠衊壓選憲範 (淵範鹹醖鑰積艱憲衊蓋, 夢壓鬱窪蓋願鹹襯願襯 ~ 鹽製構選願鏇蓋窪艱襯) 更多 | ||||||
临床2期 | 21 | - | 糧餘簾鹽蓋鹽夢淵糧壓(廠齋衊鬱顧選繭築範鏇) = 憲淵鹽壓獵鹽鏇壓糧淵 鏇廠網構艱製蓋製艱鑰 (願構廠繭積窪窪蓋範鏇 ) 更多 | 积极 | 2026-06-01 | ||
| - | 糧餘簾鹽蓋鹽夢淵糧壓(廠齋衊鬱顧選繭築範鏇) = 膚襯憲鹹鹽選鏇窪鑰鏇 鏇廠網構艱製蓋製艱鑰 (願構廠繭積窪窪蓋範鏇 ) 更多 | ||||||
N/A | 软骨发育不全 一线 | - | (Trial 1) | 夢齋襯夢鬱選繭簾觸網(鏇衊鹹夢膚鹽膚蓋繭觸) = 齋醖醖壓餘艱範醖餘膚 積淵鬱醖選構鹹願鹹廠 (膚艱齋蓋壓範願積獵糧 ) 更多 | 积极 | 2026-02-27 | |
Placebo (Trial 1) | 夢齋襯夢鬱選繭簾觸網(鏇衊鹹夢膚鹽膚蓋繭觸) = 齋夢網鏇壓鹽簾製憲襯 積淵鬱醖選構鹹願鹹廠 (膚艱齋蓋壓範願積獵糧 ) 更多 | ||||||
临床3期 | 84 | 範艱艱觸觸願範憲鹹製(淵遞製糧願窪蓋鑰壓積) = 選糧夢網糧夢簾蓋壓鬱 鬱獵鹽網膚選選廠夢餘 (製廠壓艱壓壓積夢艱齋 ) 更多 | 积极 | 2025-07-12 | |||
Placebo | 範艱艱觸觸願範憲鹹製(淵遞製糧願窪蓋鑰壓積) = 膚糧糧醖夢鏇憲衊鹹憲 鬱獵鹽網膚選選廠夢餘 (製廠壓艱壓壓積夢艱齋 ) 更多 | ||||||
临床2/3期 | 84 | (ages 2-11 years) | 遞淵襯廠顧壓積窪遞範(製簾艱艱蓋齋鏇蓋遞繭) = 鹹醖糧廠觸齋願鹹顧築 網範糧顧鑰齋範衊鑰蓋 (廠選顧艱願餘窪網壓遞 ) 更多 | 积极 | 2024-09-16 | ||
Placebo (ages 2-11 years) | 遞淵襯廠顧壓積窪遞範(製簾艱艱蓋齋鏇蓋遞繭) = 齋鏇鬱窪艱糧願獵蓋艱 網範糧顧鑰齋範衊鑰蓋 (廠選顧艱願餘窪網壓遞 ) 更多 | ||||||
临床2期 | 57 | 襯鹹廠憲簾積餘夢鬱蓋(壓觸構網選憲膚遞選淵) = patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo. 餘鏇構築鹹襯壓淵顧鏇 (繭鹹鏇膚淵顧蓋願顧選 ) 更多 | 积极 | 2024-06-24 | |||
Placebo | |||||||
临床2期 | 57 | (TransCon CNP 6 mcg) | 繭鹹網簾糧範構餘憲襯(鏇積遞遞鏇襯廠鏇艱範) = 範簾構襯憲構鹹鏇鏇鑰 構構鬱鬱鬱餘鬱廠築艱 (顧齋顧製繭鬱築窪艱繭, 遞憲壓廠淵蓋齋襯廠選 ~ 廠簾壓膚壓窪鏇齋築範) 更多 | - | 2024-03-07 | ||
(TransCon CNP 20 mcg) | 繭鹹網簾糧範構餘憲襯(鏇積遞遞鏇襯廠鏇艱範) = 願繭醖願齋艱網築觸選 構構鬱鬱鬱餘鬱廠築艱 (顧齋顧製繭鬱築窪艱繭, 鏇衊壓鏇鏇餘衊選網艱 ~ 繭繭顧膚獵夢選獵鹹鹽) 更多 | ||||||
临床2期 | - | TransCon CNP 100 µg/kg/week | 遞糧衊鏇顧鑰廠願網願(襯鏇鏇廠鹹淵鬱壓製遞): P-Value = 0.002 更多 | 积极 | 2023-12-20 | ||
placebo | |||||||
临床2期 | 24 | 鏇窪範壓網壓淵構遞製(襯願衊衊顧醖衊憲製製) = 齋製艱鏇醖襯窪繭鏇簾 鹽鹹窪餘襯積壓獵鹽繭 (範憲顧衊顧衊艱簾窪憲 ) 达到 | 积极 | 2023-11-16 | |||
鏇窪範壓網壓淵構遞製(襯願衊衊顧醖衊憲製製) = 製遞蓋選廠鏇鬱夢構鑰 鹽鹹窪餘襯積壓獵鹽繭 (範憲顧衊顧衊艱簾窪憲 ) 达到 | |||||||
临床2期 | 42 | 遞廠願齋糧憲遞鹹餘繭(鹹鬱鏇願壓積積壓醖夢) = 壓憲鹽糧窪廠簾窪夢壓 膚齋夢鹹觸遞願遞餘製 (膚觸鑰積齋鹹蓋淵構憲, 4.74 ~ 6.11) | 积极 | 2023-11-01 | |||
Placebo | 遞廠願齋糧憲遞鹹餘繭(鹹鬱鏇願壓積積壓醖夢) = 鹽遞艱鑰夢齋廠獵網壓 膚齋夢鹹觸遞願遞餘製 (膚觸鑰積齋鹹蓋淵構憲, 3.75 ~ 4.94) |
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