伏索利肽(Vosoritide) - 药物靶点：NPRB_在研适应症：软骨发育不全，软骨发育不良，努南综合征_专利_临床

概要

基本信息

药物类型

重组多肽

别名

Modified C-natriuretic peptide、Modified recombinant human C-type natriuretic peptide、Vosoritide (genetical recombination) (JAN)

+ [6]

靶点

NPRB

作用方式

激动剂

作用机制

NPRB激动剂(心房钠尿肽受体B型激动剂)

治疗领域

遗传病与畸形内分泌与代谢疾病皮肤和肌肉骨骼疾病+ [3]

在研适应症

软骨发育不全软骨发育不良努南综合征+ [5]

非在研适应症-

原研机构

BioMarin Pharmaceutical, Inc.

在研机构

BioMarin Pharmaceutical, Inc.BioMarin International Ltd.Children's National Research Institute

+ [2]

非在研机构-

权益机构-

最高研发阶段批准上市

首次获批日期

欧盟 (2021-08-26),

软骨发育不全

最高研发阶段(中国)-

特殊审评孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (日本)、孤儿药 (韩国)、优先审评 (澳大利亚)、儿科研究计划 (欧盟)、优先审评 (美国)、加速批准 (美国)、孤儿药 (澳大利亚)

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结构/序列

Sequence Code 1680765

来源: *****

关联

17

项与伏索利肽相关的临床试验

NCT07126262

/ Recruiting临床2期

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months

The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to < 36 months over a 52-week period.

开始日期2025-07-30

申办/合作机构

BioMarin Pharmaceutical, Inc.

[+1]

NCT07073014

/ Enrolling by invitation临床3期

A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

开始日期2025-06-20

申办/合作机构

BioMarin Pharmaceutical, Inc.

CTIS2024-513129-22-00

/ Recruiting临床3期

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children with Hypochondroplasia - 111-303

开始日期2025-01-16

申办/合作机构

BioMarin Pharmaceutical, Inc.

100 项与伏索利肽相关的临床结果

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100 项与伏索利肽相关的转化医学

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100 项与伏索利肽相关的专利（医药）

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71

项与伏索利肽相关的文献（医药）

2025-09-01·JOURNAL OF PEDIATRIC ORTHOPAEDICS

Changes in the Alignment of the Spine and Lower Limb in Children With Achondroplasia Treated With Vosoritide: A Single-center, 1-year Follow-up Prospective Study

Article

作者: Mishima, Kenichi ; Kamiya, Yasunari ; Sawamura, Kenta ; Imagama, Shiro ; Kitoh, Hiroshi ; Matsushita, Masaki

Background::

Achondroplasia (ACH) is the most common skeletal dysplasia and is characterized by a short-limbed short stature, sagittal spinal malalignment, and genu varum. Vosoritide promotes longitudinal bone growth in children with ACH; however, its effects on various disease-specific complications, other than short stature, are unknown. This study aimed to investigate the therapeutic effects of vosoritide on spinal and lower limb malalignment in children with ACH.

Methods::

This single-center, open-label, prospective study included patients with ACH aged younger than or equal to 15 years who received vosoritide treatment and had a minimum follow-up period of 1 year. To evaluate alignment after vosoritide treatment, radiologic parameters were measured from sagittal radiographs of the spine and anteroposterior radiographs of the bilateral lower limbs before the administration of vosoritide and 12 months after treatment. Paired t tests were used to compare parameters before and after vosoritide treatment.

Results::

Seventeen patients (mean age, 7.6±2.7 y) were included. After 1-year treatment of vosoritide, the mean height increased by 5.4±1.3 cm. Changes in spinal alignment after 1 year of vosoritide treatment were 1.5 degrees for cervical lordosis, −1.3 degrees for thoracic kyphosis, −2.8 degrees for thoracolumbar kyphosis, −5.2 degrees for lumbar lordosis (LL), −2.2 degrees for pelvic tilt, −2.6 degrees for pelvic incidence, −0.4 degrees for sacral slope, and 2.6 mm for C7 sagittal vertical axis. Alignment changes in the lower limbs were −3.4 degrees for mechanical axis angle (MAA), 1.7 degrees for mechanical lateral proximal femoral angle (mLPFA), −2.8 degrees for mechanical lateral distal femoral angle (mLDFA), −0.2 degrees for medial proximal tibial angle, and −0.5 degrees for lateral distal tibial angle. The LL, MAA, mLPFA, and mLDFA levels showed statistically significant changes towards the normal range after treatment.

Conclusions::

One-year treatment of vosoritide decreased the exaggerated LL and improved genu varum deformity in children with ACH. Vosoritide therapy may not only increase longitudinal bone growth but also improve spinal and lower limb malalignment in children with ACH.

Level of Evidence::

Level II: prospective comparative study.

2025-08-01·ADVANCES IN THERAPY

Real-World Safety and Effectiveness of Vosoritide in Achondroplasia: Results from a Single Center in Portugal

Article

作者: Sousa, Sérgio B ; Rua, Inês B ; Pais, Maria J ; Mirante, Alice ; Beger, Christoph ; Silva, Isabel ; Gomes, Cristina

INTRODUCTION:

Achondroplasia, the most common skeletal dysplasia, is caused by autosomal dominant gain-of-function pathogenic variants in the fibroblast growth factor receptor 3 (FGFR3) gene. Vosoritide, a C-type natriuretic peptide analog, is a first-in-class targeted treatment for achondroplasia that counteracts overactive FGFR3 signaling to stimulate endochondral bone growth. This retrospective cohort study evaluated growth, safety, and treatment compliance in children with achondroplasia receiving vosoritide under an early access program in Portugal.

METHODS:

Twenty-seven children aged 2-14 years with a genetically confirmed diagnosis of achondroplasia were treated with vosoritide at a single Portuguese center for at least 6 months between January 2022 and June 2024. The analysis included children with severe achondroplasia-associated complications. Anthropometric measurements collected to characterize the effect of vosoritide on growth included height standard deviation score (SDS) and annualized growth velocity (AGV). Student's t test was used for statistical comparisons. Safety and tolerability endpoints included adverse drug reactions and treatment adherence.

RESULTS:

In total, 15 children completed at least 24 months of treatment. After 24 months of treatment, mean variation in height SDS increased from baseline by + 0.95 SD (P ≤ 0.0001), referenced to an untreated achondroplasia-specific population, and + 0.56 SD (P ≤ 0.0001) relative to children of average stature. Additionally, mean AGV from baseline was 5.87 cm/year (95% confidence interval 5.14-6.60), resulting in a significant increase of + 1.62 cm/year (P ≤ 0.0001). Injection site reactions were the most common adverse drug reaction observed (n = 14); no serious adverse drug reactions were reported. There were no discontinuations due to adverse drug reactions.

CONCLUSION:

Vosoritide showed long-term effectiveness in a real-world Portuguese population of patients with achondroplasia. Vosoritide was also well tolerated, and patients showed good adherence to treatment. These findings were consistent with the outcomes of clinical trials and existing real-world experience.

2025-08-01·CURRENT OPINION IN PEDIATRICS

Genetics of short stature

Review

作者: Navardauskaite, Ruta ; Nicolae, Ruxandra ; Jee, Youn Hee

Purpose of review:

This review highlights recent genetic discoveries and therapeutic advancements in evaluating and managing children with short stature. With an increasing diagnostic yield from genetic testing and the emergence of genotype-specific treatments, a comprehensive update is necessary for timely application in clinical practice.

Recent findings:

New pathogenic variants have been identified in genes, including FBN1, IHH, NPR2, ACAN, FGFR3, COMP, MATN3, EXT2, and LZTR1, associated with syndromic and nonsyndromic short stature. Their clinical significance is underscored by sequencing studies showing a diagnostic yield of up to 33% in idiopathic short stature. Concurrently, targeted pharmacologic agents, such as vosoritide or infigratinib, are advanced treatment options for genetically defined achondroplasia. In addition, more data is available on growth hormone responses in children with various genetic short statures.

Summary:

Incorporating genetic testing into the routine investigation of short stature improves diagnostic accuracy, enables early discussion of prognostic outcomes, and facilitates precision therapy. Timely identification of specific gene variants helps avoid ineffective treatments – such as growth hormone in resistant genotypes – and supports the adoption of personalized medicine interventions.

156

项与伏索利肽相关的新闻（医药）

2025-08-05

·FiercePharma

BioMarin touts promising early result for potential Voxzogo successor

BioMarin is projecting sales of Voxzogo to fall between $900 million and $935 million this year. It also has a potential replacement for the achondroplasia treatment in its pipeline which could hit the market by 2030, the company says. Competition is coming for BioMarin’s potential blockbuster Voxzogo, but the California biotech may be able to answer with a longer-acting version of the C-type natriuretic peptide (CNP), which treats a form of dwarfism in children called achondroplasia.BioMarin revealed topline results for the early-stage candidate, BMN 333, that suggest it is a “potentially superior version of Voxzogo,” according to a research note from Evercore ISI analyst Cory Kasimov.In a phase 1 study of healthy adult volunteers, BMN 333 demonstrated area-under-the-curve (AUC) levels greater than three times those observed in other long-acting CNP studies, with no safety signals found, BioMarin said in an August 4 press release. AUC is a pharmacokinetic measure indicating the total exposure to a drug over time, the company added.With the result, BioMarin said that it plans to begin the dose-finding arm of its phase 2/3 registration-enabling program in the first half of next year, with the potential for a 2030 launch if the results are positive.Since close of the market on Friday, BioMarin's share price has increased by 12%.While BioMarin said that its goal with BMN 333 is to demonstrate its superiority to Voxzogo, which was approved in 2021, R&D chief Greg Friberg was quick to point out during the company’s second-quarter conference call on Monday that the result was “three times greater than the AUC levels reported for another long-acting CNP.”Friberg’s reference was to Ascendis’ TransCon CNP, which has demonstrated comparable results to Voxzogo in a phase 3 trial of achondroplasia patients and is scheduled for an FDA approval decision by November 30.One potential advantage of the Ascendis treatment, which is also known as navepegritide, is that it is injected weekly as opposed to daily for Voxzogo.As for BMN 333, the drug's profile so far demonstrates "a potential best-in-class molecule with the opportunity to drive even greater improvements in growth parameters versus available therapies, and by extension, to further improve measures of health and wellness in children with achondroplasia,” Friberg added on Monday. Another achondroplasia candidate to consider is Kyowa Kirin and BridgeBio’s daily oral FGFR3 inhibitor infigratinib. BridgeBio is conducting a phase 3 study in achondroplasia patients and is testing the treatment in hypochondroplasia, which is a form of short-limbed dwarfism.As for Voxzogo, which is the lone medicine on the market for achondroplasia, second-quarter sales came in at $221 million, a 20% increase year over year, bringing its first-half tally to $404 million. The company is projecting 2025 Voxzogo sales at a range of $900 million to $935 million, which would account for nearly 30% of its revenue.

临床结果临床3期上市批准临床1期

2025-05-16

·BioSpace

BioMarin Expands Enzyme Expertise With $270M Inozyme Buy

iStock, Dmitrii_Guzhanin The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency. BioMarin is acquiring rare disease specialist Inozyme Pharma for $270 million to build out its portfolio of enzyme therapies, the biotech announced on Friday. BioMarin will pay $4 for each outstanding share of Inozyme. The companies expect to complete the transaction in the third quarter, pending customary closing conditions such as anti-trust clearances and the tender of at least a majority of Inozyme shares. The Board of Inozyme has unanimously recommended that stockholders tender their shares. The centerpiece of Friday’s deal is Inozyme’s INZ-701, an investigational enzyme replacement therapy currently in late-stage development for ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency. Caused by mutations to the ENPP1 gene, ENPP1 deficiency is a rare and progressive disease that can affect a variety of organs, presenting differently from patient to patient. Common symptoms include early-onset osteoporosis, a heightened risk of fractures and calcification of the tendons. Patients with ENPP1 deficiency are also more susceptible to cardiovascular mortality, severe rickets and softening of the bones. There are currently no approved treatments for the condition. Delivered subcutaneously, INZ-701 combines the active site of the ENPP1 enzyme with a specific fragment of a human antibody, according to Inozyme’s website . This structure allows INZ-701 to be distributed throughout the body, essentially replacing the deficient ENPP1 enzyme and carrying out its function. Preclinical data have supported this mechanism of action, showing that INZ-071 could prevent abnormal mineralization. Aside from ENPP1 deficiency, Inozyme is also testing INZ-701 for ABCC6 deficiency and calcific uremic arteriopathy, both rare diseases. The acquisition comes after some rocky times for BioMarin. Last year the company reconfigured its c-suite, laid off 225 employees and axed four molecules as part of a broader reorganization plan. “As BioMarin continues our transformation and delivers on our corporate strategy, we will continue to evaluate external innovation alongside internal innovation,” said BioMarin’s president and CEO Alexander Hardy in a statement announcing the deal. “We are in a strong financial position to bring in additional assets as we accelerate the development of medicines for patients with significant unmet need.” The Inozyme acquisition also follows BioMarin’s first-quarter earnings call, where Hardy said that the biotech would conduct “at least one business development deal this year.” BioMarin made $745 million in Q1, which represents a 15% year-on-year increase and was ahead of the analyst consensus of $738.1 million. BioMarin’s enzyme therapy portfolio accounted for much of its topline, bringing in $484 million in the quarter, an 8% jump from the same period last year. Voxzogo, its closely watched C-type natriuretic peptide analog indicated for achondroplasia with open epiphyses, surged 40% year-on-year to bring in $214 million. With a strong start to 2025, BioMarin reaffirmed its full-year outlook. The company expects total revenues to hit $3.1 billion, with Voxzogo contributing at least $900 million. Still, analysts at William Blair remain cautious about BioMarin. In a May 2 note to investors, the analysts flagged “uncertainty” regarding the biotech’s “current revenue drivers” and whether they have “the potential to continue delivering significant value creation in the near term.”

并购IPO基因疗法

2025-05-15

·抗体圈

大肠杆菌生产药物的关键技术与挑战

摘要：大肠杆菌凭借重组 DNA 技术成为生物制药领域的核心工具，从首个重组胰岛素的生产至今，已在激素、酶、疫苗、抗体等多个领域展现重要价值。尽管存在翻译后修饰不足和毒性等局限，但其低成本、高产量和成熟的生产体系使其仍是首选宿主。本文系统梳理了大肠杆菌生产的各类生物药物，包括已获批的激素、酶、肽类、疫苗及融合蛋白等，并探讨其临床应用、优势与未来发展方向，展现其在生物制药产业中的关键地位与潜力。一、引言大肠杆菌在重组 DNA 技术的推动下，成为生物制药的重要宿主。自 1980 年代首个重组胰岛素获批以来，其凭借快速生长、低成本和高产量等优势，广泛应用于多种生物药物生产。尽管面临复杂蛋白折叠困难和缺乏糖基化修饰等挑战，但其在激素、酶、疫苗等领域的成果显著，已成为全球生物制药产业的基石之一（参考图 1: General method for producing recombinant biopharmaceuticals in Escherichia coli，了解大肠杆菌生产重组生物药物的通用流程）。二、大肠杆菌生产的生物药物类型2.1激素类药物大肠杆菌是生产重组激素的主力，涵盖胰岛素、生长激素、干扰素等。例如，首个重组人胰岛素Humulin由礼来公司开发，1982 年获 FDA 批准，彻底改变糖尿病治疗模式。随后，长效胰岛素类似物Lantus、生长激素Sogroya等相继问世，显著提升患者生活质量（表1: Approved recombinant hormones produced in Escherichia coli and their mechanism of action列出了主要激素药物及其作用机制）。干扰素 α-2b（Intron A）则用于肝炎、癌症治疗，其改良型Besremi对真性红细胞增多症疗效显著。2.2酶类药物酶类药物在代谢疾病和癌症治疗中至关重要。Krystexxa（聚乙二醇化尿酸酶）用于慢性痛风，通过降解尿酸缓解症状；Voraxaze（葡糖羧肽酶）可快速清除化疗药物甲氨蝶呤，降低肾损伤风险；Asparlas（聚乙二醇天冬酰胺酶）则用于白血病治疗，延长患者生存期（表2: Approved recombinant enzymes produced in Escherichia coli and their mechanism of action详细展示了相关酶类药物）。2.3肽类药物肽类药物在心血管和代谢疾病中应用广泛。Natrecor（奈西立肽）用于急性心力衰竭，通过扩张血管改善症状；Voxzogo（vosoritide）针对软骨发育不全，通过调节信号通路促进骨骼生长；Oxervate（神经生长因子）则用于治疗神经萎缩性角膜炎，修复角膜损伤（表3: Approved recombinant peptides produced in Escherichia coli and their mechanism of action列举了代表性肽类药物）。2.4疫苗类药物大肠杆菌在疫苗生产中表现突出，尤其在病毒样颗粒（VLP）疫苗领域。Hecolin是首个基于大肠杆菌的戊肝疫苗，在中国获批上市；Cecolin（HPV 疫苗）有效预防生殖器病变；兽用疫苗如Strangvac（马链球菌疫苗）和Leucogen（猫白血病疫苗）也展现重要价值（表4: Approved recombinant vaccines produced in Escherichia coli总结了人用及兽用疫苗）。2.5融合蛋白与抗体片段融合蛋白和抗体片段是癌症与免疫疾病的关键疗法。Lucentis（雷珠单抗）靶向 VEGF，用于湿性黄斑变性；Kimmtrak（ Tebentafusp）通过双特异性结合 T 细胞和肿瘤抗原，治疗转移性葡萄膜黑色素瘤；Cimzia（ certolizumab pegol）作为 TNF-α 抑制剂，有效缓解类风湿关节炎（表5: Approved recombinant antibodies and fusion proteins produced in Escherichia coli列出了主要抗体及融合蛋白药物）。2.6其他生物药物包括粒细胞集落刺激因子Neupogen（治疗中性粒细胞减少症）、瘦素类似物Myalept（治疗脂肪营养不良）等，均通过大肠杆菌生产，满足罕见病和免疫疾病治疗需求。三、大肠杆菌的优势与挑战优势：遗传背景清晰：便于基因操作和表达系统优化，如利用已知的克隆技术和表达载体高效生产目标蛋白。生产成本低：培养基简单（仅需基础营养物质），适合大规模发酵，例如 mRNA 新冠疫苗的低成本生产模式可复制至其他生物药。生产周期短：生长繁殖速度快，可在短时间内实现蛋白大量表达，如重组胰岛素从基因设计到生产仅需数周。挑战：翻译后修饰局限：无法进行复杂糖基化、二硫键形成等修饰，影响部分蛋白（如全长单克隆抗体）的功能，需依赖哺乳动物细胞补位（参考表5中部分抗体需哺乳动物细胞生产的限制）。内毒素风险：细胞壁成分可能引发免疫反应，需额外纯化工艺去除内毒素，增加生产成本（如疫苗生产中的严格纯化流程）。蛋白折叠问题：复杂蛋白易形成包涵体，需优化表达条件或采用共表达伴侣蛋白辅助折叠（如图1中纯化步骤的重要性）。四、未来发展方向技术优化：通过基因工程改造大肠杆菌，引入糖基化通路或共表达折叠辅助因子，提升复杂蛋白生产能力。例如，利用合成生物学技术构建 “设计型” 大肠杆菌，实现更接近天然蛋白的修饰。联合疗法拓展：大肠杆菌生产的疫苗与免疫检查点抑制剂（ICIs）联用，如mRNA 肿瘤疫苗 + PD-1 单抗的协同效应，已在黑色素瘤等适应症中展现潜力（参考前文KEYNOTE-942 研究数据）。兽用与罕见病药物：针对动物传染病（如马链球菌疫苗Strangvac）和罕见病（如脂肪营养不良药物Myalept），开发高性价比的大肠杆菌生产药物，填补市场空白。可持续生产：探索无血清培养基和连续生产工艺，降低环境负担，符合绿色制药趋势。五、结论大肠杆菌凭借低成本、高可及性和成熟的工业化体系，在生物制药领域占据不可替代的地位。尽管面临修饰能力有限等挑战，但其在激素、酶、疫苗等领域的成功案例（如表1-5中多款 FDA/EMA 批准药物）及技术优化潜力，使其持续成为创新药物开发的核心平台。未来，随着合成生物学和联合疗法的突破，大肠杆菌将进一步推动生物制药向高效、经济、普惠方向发展。识别微信二维码，添加抗体圈小编，符合条件者即可加入抗体圈微信群！请注明：姓名+研究方向！本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

疫苗免疫疗法微生物疗法

100 项与伏索利肽相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11190	伏索利肽	-	DB11928

研发状态

批准上市

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适应症	国家/地区	公司	日期
软骨发育不全	欧盟	BioMarin International Ltd.	2021-08-26
软骨发育不全	冰岛	BioMarin International Ltd.	2021-08-26
软骨发育不全	列支敦士登	BioMarin International Ltd.	2021-08-26
软骨发育不全	挪威	BioMarin International Ltd.	2021-08-26

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
软骨发育不良	申请上市	加拿大	BioMarin Pharmaceutical, Inc.	2025-02-01
努南综合征	临床2期	美国	BioMarin Pharmaceutical, Inc.	2024-11-22
努南综合征	临床2期	法国	BioMarin Pharmaceutical, Inc.	2024-11-22
努南综合征	临床2期	意大利	BioMarin Pharmaceutical, Inc.	2024-11-22
努南综合征	临床2期	西班牙	BioMarin Pharmaceutical, Inc.	2024-11-22
矮身材同源框基因缺乏症	临床2期	美国	BioMarin Pharmaceutical, Inc.	2024-11-22
矮身材同源框基因缺乏症	临床2期	法国	BioMarin Pharmaceutical, Inc.	2024-11-22
矮身材同源框基因缺乏症	临床2期	意大利	BioMarin Pharmaceutical, Inc.	2024-11-22
矮身材同源框基因缺乏症	临床2期	西班牙	BioMarin Pharmaceutical, Inc.	2024-11-22
特发性矮小症	临床2期	美国	BioMarin Pharmaceutical, Inc.	2024-10-21

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适应症

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


OPAL (Company_Website) 人工标引	N/A	软骨发育不全	-	VOXZOGO® (vosoritide)	壓壓遞顧壓製襯夢齋衊(願鑰繭獵選蓋廠壓艱廠) = 夢廠簾膚鏇蓋構餘糧壓壓簾齋餘鑰繭觸獵糧糧 (醖願觸窪襯蓋觸繭範網 )	积极	2025-03-20
Company_Website 人工标引	N/A	软骨发育不全	63	VOXZOGO	顧齋餘構鏇鹹獵築鹹膚(繭製範遞鏇願範膚願範) = None 鑰餘繭觸鹽淵構壓鬱窪 (壓積積築鏇齋選遞鑰積 )	积极	2025-03-20
NCT03424018 \| NCT03197766 (CLARITY achondroplasia study, Pubmed \| Med)	临床3期	软骨发育不全 C-type natriuretic peptide analog	119	Vosoritide 15 μg/kg	淵醖鬱齋艱鹽鑰艱蓋築(憲鹹夢顧蓋鏇遞蓋網膚) = 淵鬱蓋願鑰積鏇獵獵壓鹹窪製選餘衊膚廠夢構 (築顧顧顧願襯願壓範觸, 0.38) 更多	积极	2024-12-01
	临床3期	软骨发育不全 C-type natriuretic peptide analog	119	vosoritide (Untreated Control)	淵醖鬱齋艱鹽鑰艱蓋築(憲鹹夢顧蓋鏇遞蓋網膚) = 醖鏇憲積鏇衊構壓鑰醖鹹窪製選餘衊膚廠夢構 (築顧顧顧願襯願壓範觸, 0.63)	积极	2024-12-01
NCT04219007 (phase 2, Pubmed \| EClinicalMedicine)	临床2期	软骨发育不良	26	Vosoritide 15 μg/kg/day	構繭淵餘積網艱廠窪廠(構積選構鬱簾夢衊積構) = 鬱壓獵範醖獵糧壓鹽積願壓觸艱獵淵簾顧憲觸 (窪憲壓膚鬱淵獵膚壓糧 )	积极	2024-04-01
NCT03989947 (Study 111-206, PRNewswire) 人工标引	临床2期	软骨发育不全	-	VOXZOGO	醖繭襯繭遞鹽顧選淵選(醖鏇築鹹觸壓繭廠簾壓) = 憲鹹襯艱蓋製積製選願餘襯廠範窪蓋鬱鏇願蓋 (蓋鏇顧築蓋鹽醖壓淵蓋, 0.46 ~ 1.93) 更多	积极	2023-10-20
NCT03989947 \| NCT03583697 (111-206, ESPE2023)	临床2期	软骨发育不全	34	Vosoritide 15 mg/kg	觸鹽齋製鑰繭醖膚憲艱(窪鬱繭網鬱簾夢鏇膚齋) = 觸醖餘襯襯夢廠選餘觸蓋願簾壓夢觸獵積蓋蓋 (範憲窪襯積鹽壓窪遞鹹, 2.98 ~ 9.56)	积极	2023-09-21
OR18-5 (ENDO2022)	临床2期	侏儒症	35	Vosoritide 15 mcg/kg/day	築蓋積膚廠艱願築糧糧(淵壓餘鑰網廠膚繭範鏇) = 遞鬱鑰網糧蓋製鏇製鏇積衊鑰獵製艱衊選襯簾 (積醖窪憲鹽簾鏇窪築廠, 2.2 ~ 4.6)	积极	2022-11-01
ESPE2022	临床2期	软骨发育不良	26	Vosoritide	夢鬱鑰願艱鑰築膚鹹醖(顧獵齋鬱鏇淵窪鬱選蓋) = 觸範衊繭簾選憲願鏇餘鏇鬱鑰膚鹹鹽壓艱壓蓋 (構廠網襯積鹹醖網範淵, 6.8 ~ 8)	-	2022-09-15
ATU (ESPE2022)	N/A	软骨发育不全	29	Vosoritide	簾遞壓餘壓廠艱製襯壓(蓋襯範鬱鹹艱製廠憲獵) = The majority of AEs were mild and included injection site reactions and vomiting, with the most common being injection site rash (4 events) 襯襯築積壓艱構蓋窪鹹 (範鹹窪艱遞繭願構鹹壓 ) 更多	积极	2022-09-15
ESPE2022	临床2期	糖尿病性黄斑水肿 \| 努南综合征 NPR2 mutations \| PTPN11 mutations	6	Vosoritide 15 mg/kg/day	夢齋觸選壓廠糧鏇鏇觸(製艱繭艱築繭鬱網獵獵) = 艱鑰餘醖獵願廠積窪獵鑰遞構鹽鹽窪願艱鑰獵 (窪餘蓋憲廠艱蓋鑰構遞 )	积极	2022-09-15