BioMarin在两场国际内分泌学会议上展示了VOXZOGO®（vosoritide）用于治疗软骨发育不全症及其他骨骼疾病儿童的新数据-动脉网

BioMarin Presents New Data for VOXZOGO® (vosoritide) in Children with Achondroplasia and Other Skeletal Conditions at Two International Endocrinology Meetings

CISION 等信源发布 2025-05-12 21:05

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可切换为仅中文

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New data in children under 5 years with achondroplasia showed treatment with VOXZOGO was associated with improvement in tibial bowing, a common cause of pain and impaired function

新数据显示，5岁以下患有软骨发育不全的儿童使用VOXZOGO治疗与胫骨弯曲改善相关，而胫骨弯曲是导致疼痛和功能受损的常见原因。

Data from the largest retrospective, multi-year observational study of more than 600 children and adults with hypochondroplasia found significantly higher rates of comorbidities, surgeries and overall doctor visits

数据来自对600多名儿童和成人进行的最大规模的回顾性多年观察研究，结果显示，患有软骨发育不全症的人群中，并发症、手术和整体看诊的比率显著更高。

SAN RAFAEL, Calif.

加利福尼亚州圣拉斐尔

，

May 12, 2025

2025年5月12日

/PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:

/PRNewswire/ -- BioMarin制药公司（纳斯达克：

BMRN

) today announced new data from studies of VOXZOGO

）今天宣布了VOXZOGO研究的新数据

(vosoritide), demonstrating meaningful impact on tibial bowing in children with achondroplasia and investigational early efficacy results in other skeletal conditions. The data were presented at the 2025 Joint Congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) in .

（vosoritide），在患有软骨发育不全的儿童中显示出对胫骨弯曲的显著影响，并在其他骨骼疾病中展示了早期疗效的研究结果。这些数据在2025年欧洲儿科内分泌学会（ESPE）和欧洲内分泌学会（ESE）联合大会上发布。

Copenhagen, Denmark

丹麦哥本哈根

and will be shared at the Pediatric Endocrine Society Annual Meeting (PES) in National Harbor,

并将在国家港口举行的儿科内分泌学会年会（PES）上分享，

Maryland

马里兰州

。

VOXZOGO Demonstrated Positive Impact on Tibial Bowing

VOXZOGO对胫骨弯曲显示出积极影响

An analysis of data presented at PES from the VOXZOGO Phase 2 CANOPY clinical studies in younger children assessed the impact of treatment on tibial bowing, an orthopedic complication and significant cause of pain in children with achondroplasia. Children who received VOXZOGO had a significant reduction in the magnitude of tibial bowing compared to children who received placebo.

对在PES上展示的VOXZOGO 2期CANOPY临床研究数据的分析评估了治疗对胫骨弯曲的影响，胫骨弯曲是导致软骨发育不全儿童出现疼痛的一种骨科并发症和重要原因。接受VOXZOGO治疗的儿童与接受安慰剂的儿童相比，胫骨弯曲程度显著减少。

Furthermore, the researchers found that this improvement was sustained in children who received treatment for several years..

此外，研究人员发现，这种改善在那些接受治疗多年的儿童中得以持续。

'Outcomes beyond height have long been prioritized by the dwarfism community,' said

“长期以来，侏儒症群体一直优先考虑身高以外的结果，”

Michael Hughes

迈克尔·休斯

, Chair, Little People of America's (LPA) Biotech Industry Liaison Committee. 'Tibial bowing can be a source of pain and surgical intervention for some children with achondroplasia, and this research reflects an encouraging shift toward addressing a meaningful outcome to the community.'

，主席，美国小人国（LPA）生物技术行业联络委员会。“胫骨弯曲可能是一些软骨发育不全儿童疼痛和手术干预的来源，这项研究反映了一个令人鼓舞的转变，即针对对社区有意义的结果进行处理。”

'In children with achondroplasia, tibial bowing can be painful and often requires surgical intervention,' said

“在患有软骨发育不全的儿童中，胫骨弯曲可能会引起疼痛，并且通常需要手术干预，”

Klane White

克兰·怀特

, M.D., pediatric orthopedic surgeon and Rose Brown Endowed Chair of Pediatric Orthopedics at Children's Hospital Colorado. 'Through my clinical practice, I have observed meaningful improvements that early treatment initiation with VOXZOGO has offered children with achondroplasia, including in proportionality, quality of life and now tibial bowing, which have the potential to improve physical well-being for these children into adulthood.'.

医学博士，儿童医院科罗拉多州小儿骨科医生及Rose Brown捐赠设立的小儿骨科主席表示：“通过我的临床实践，我观察到早期使用VOXZOGO治疗对软骨发育不全的儿童带来了显著改善，包括身体比例、生活质量，现在还包括胫骨弯曲，这些改善有潜力提升这些儿童直至成年期的身体健康。”

The latest results from the VOXZOGO Phase 3 clinical trial reporting the longest follow-up to date demonstrated that continuous and early treatment was associated with sustained increases in annualized growth velocity (AGV). For the first time, it was possible to model potential final height gain for children with achondroplasia if they were treated early and continuously from 6 months until final adult height (FAH).

VOXZOGO第三期临床试验的最新结果报告了迄今为止最长的随访数据，显示持续且早期的治疗与年生长速度（AGV）的持续增加相关。首次有可能模拟患有软骨发育不全症的儿童在从6个月开始持续到最终成人身高（FAH）接受治疗的情况下可能获得的最终身高增长。

Modeling indicated that VOXZOGO treatment could result in a height increase of 21.7 centimeters (cm) (95% Cl, 18.7-24.6) in girls and 26.4 cm (95% Cl, 22.9-29.8) in boys compared to untreated children. These data were presented today at ESPE as an oral presentation and add to the totality of evidence demonstrating that early and continuous treatment with VOXZOGO maximizes clinical benefits for children with achondroplasia..

模型显示，与未接受治疗的儿童相比，使用VOXZOGO治疗可使女孩身高增加21.7厘米（95%置信区间，18.7-24.6），男孩身高增加26.4厘米（95%置信区间，22.9-29.8）。这些数据今天在欧洲儿科内分泌学会（ESPE）上以口头报告形式展示，并进一步补充了证明早期和持续使用VOXZOGO治疗能够最大化软骨发育不全患儿临床获益的全部证据。

Hypochondroplasia Associated with Significant Increase in Comorbidities

与显著增加的合并症相关的软骨发育不全症

Researchers also presented data shedding light on the comorbidities associated with hypochondroplasia, a genetically defined form of skeletal dysplasia for which VOXZOGO is currently being evaluated in clinical trials. In more than 600 adults and children with hypochondroplasia who were studied using real-world electronic health primary care medical record data in .

研究人员还提供了有关软骨发育不全相关合并症的数据，软骨发育不全是一种基因定义的骨骼发育不良形式，目前VOXZOGO正在临床试验中进行评估。研究使用了来自真实世界电子健康初级保健医疗记录的数据，对600多名成人和儿童患者进行了研究。

England

英格兰

, comorbidity event rates were more common compared to people without hypochondroplasia. The condition was also associated with an increase in doctor visits, surgeries and orthopedic interventions.

，与没有软骨发育不全症的人相比，并发症发生率更常见。这种情况还伴随着看医生次数、手术以及骨科干预的增加。

'This research is critical to shaping a deeper understanding of hypochondroplasia and underscores the importance of early diagnosis, which allows physicians to begin working with children and their families as soon as possible,' said

“这项研究对于加深对软骨发育不全症的理解至关重要，并强调了早期诊断的重要性，早期诊断使医生能够尽快开始与儿童及其家人合作，”

Greg Friberg

格雷格·弗里伯格

, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. 'We remain committed to developing new options for skeletal conditions, such as hypochondroplasia and Turner syndrome, and these early data continue to inform our clinical trials.'

，医学博士，BioMarin执行副总裁兼首席研发官。“我们仍然致力于开发针对骨骼疾病（如软骨发育不全和特纳综合征）的新疗法，这些早期数据继续为我们的临床试验提供指导。”

In a prospective, single-center Phase 2 open-label clinical study in girls with Turner syndrome, researchers found that treatment with VOXZOGO was associated with a promising increase in AGV at 6 months compared to their baseline AGV. Importantly, AGV was increased even in children who were previously treated with human growth hormone and discontinued due to suboptimal results prior to enrollment in this study.

在一项针对特纳综合征女孩的前瞻性、单中心2期开放标签临床研究中，研究人员发现，与基线AGV相比，使用VOXZOGO治疗在6个月时AGV有显著提升。重要的是，即使是在入组本研究前曾因效果不佳而停止使用人类生长激素的儿童，其AGV也有所增加。

VOXZOGO was also well-tolerated with safety results consistent with its known profile in achondroplasia..

VOXZOGO 也具有良好的耐受性，其安全性结果与其在软骨发育不全中的已知特征一致。

BioMarin continues to advance development across its CANOPY clinical program for VOXZOGO in hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome and SHOX deficiency. In April, BioMarin completed enrollment in its pivotal Phase 3 study with VOXZOGO in hypochondroplasia, and the company is on track to share topline data in 2026, with potential launch in 2027..

BioMarin继续推进VOXZOGO在软骨发育不全、特发性矮小症、努南综合征、特纳综合征和SHOX缺陷中的CANOPY临床项目开发。今年4月，BioMarin完成了VOXZOGO针对软骨发育不全的关键性3期研究的受试者招募，公司预计将于2026年分享初步数据，并有望在2027年上市。

Below are key presentations for BioMarin and VOXZOGO at both meetings:

以下是BioMarin和VOXZOGO在两次会议上的重要报告：

ESPE and ESE

ESPE 和 ESE

Design of a Phase 2, Randomized, Controlled, Multicentre Study of Vosoritide Treatment in Children with Idiopathic Short Stature

一项关于Vosoritide治疗特发性矮小症儿童的二期、随机、对照、多中心研究设计

ePoster P647

电子海报 P647

Saturday, May 10

5月10日，星期六

Design of a Randomized, Multicentre, Phase 2 Study of Vosoritide in Children with Turner Syndrome, Noonan Syndrome, or Short Stature Homeobox-Containing Gene (SHOX) Deficiency

伏索利肽治疗特纳综合征、努南综合征或含短身高同源盒基因（SHOX）缺陷儿童的随机、多中心、2期研究设计

ePoster EP840

电子海报 EP840

Saturday, May 10

5月10日，星期六

The Medical Impact of Hypochondroplasia Among Children in England Between 1998 and 2019: A Matched Cohort Study Using Electronic Medical Records from the Clinical Practice Research Datalink

1998年至2019年间英格兰儿童中软骨发育不全的医学影响：一项使用临床实践研究数据链电子医疗记录的匹配队列研究

ePoster P617

电子海报 P617

Saturday, May 10

5月10日，星期六

The Medical Impact of Hypochondroplasia by Age Among Adults in

成人中按年龄划分的软骨发育不全的医学影响

England

英格兰

between 1998 and 2019: A Matched Cohort Study Using Electronic Medical Records from the Clinical Practice Research Datalink

1998年至2019年：使用临床实践研究数据链电子医疗记录的匹配队列研究

ePoster P603

电子海报 P603

Saturday, May 10

5月10日，星期六

The Achondroplasia Roadmap

软骨发育不全路线图

ePoster EP202

电子海报 EP202

Saturday, May 10

5月10日，星期六

Long-Term Height Gain and Maintenance of Treatment Effect in Children with Achondroplasia Receiving Vosoritide

接受伏索利肽治疗的软骨发育不全儿童的长期身高增长与治疗效果维持

Oral Presentation OC7.4

口头报告 OC7.4

Monday, May 12

5月12日，星期一

, 3:05 –

，3:05 –

3:15 p.m.

下午3点15分

Central European Summer Time (CEST)

中欧夏令时间 (CEST)

PES (all times in Eastern Daylight Time [EDT])

PES（所有时间均为东部夏令时间 [EDT]）

IGF-1 Values in Hypochondroplasia and Effects of Vosoritide

低矮软骨发育不全中的IGF-1值及Vosoritide的影响

Poster P9

海报 P9

Thursday, May 15

5月15日，星期四

, 6:30 –

，6:30 –

8:30 p.m.

晚上8点30分

Medical Impact of Hypochondroplasia Among Children and Adults in

假性软骨发育不全对儿童和成人的医学影响

England

英格兰

: A 22-Year Retrospective Study

：一项22年的回顾性研究

Poster P42

海报 P42

Friday, May 16

5月16日，星期五

, 12:00 –

，12:00 –

12:30 p.m.

中午12点30分

Vosoritide Improves Tibial Bowing in Infants and Toddlers with Achondroplasia

Vosoritide 改善了患有软骨发育不全的婴幼儿的胫骨弯曲问题。

Poster P28

海报 P28

Friday, May 16

5月16日，星期五

, 12:00 –

，12:00 –

12:30 p.m.

中午12点30分

Preliminary Assessment of Vosoritide for Short Stature in Turner Syndrome: 6-Month Data from a Clinical Trial

Vosoritide治疗特纳综合征矮小症的初步评估：来自临床试验的6个月数据

Poster P39

海报 P39

Saturday, May 17

5月17日，星期六

, 12:45 –

，12:45 –

2:30 p.m.

下午2点30分

About Achondroplasia

关于软骨发育不全

Achondroplasia, the most common form of skeletal dysplasia, is characterized by impaired bone growth caused by a change in the

软骨发育不全是最常见的骨骼发育不良形式，其特征是由于

FGFR3

gene. Bone growth is regulated by multiple biological processes including signaling pathways through fibroblast growth factor (FGF, which slows bone growth) and C-type natriuretic peptide (CNP, which increases bone growth). In achondroplasia, these signals are out of balance, resulting in a slowing of endochondral ossification, and causing disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull..

基因。骨骼生长受多种生物过程的调节，包括通过成纤维细胞生长因子（FGF，抑制骨骼生长）和C型利钠肽（CNP，促进骨骼生长）的信号通路。在软骨发育不全中，这些信号失衡，导致软骨内骨化减缓，并引起长骨、脊柱、面部和颅底的不成比例短小和结构紊乱。

More than 80% of children with achondroplasia have parents of average stature and have the condition as a result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being evaluated in children whose growth plates are still 'open,' typically those under 18 years of age.

超过80%的软骨发育不全儿童的父母身高正常，该疾病是由于自发基因突变引起的。软骨发育不全的全球发病率约为每25,000例活产婴儿中有一例。VOXZOGO正在针对生长板仍然“开放”的儿童进行评估，通常为18岁以下的儿童。

Approximately 25% of people with achondroplasia fall into this category..

大约25%的软骨发育不全患者属于这一类。

About VOXZOGO

关于VOXZOGO

In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in

在患有软骨发育不全的儿童中，由于功能获得性突变，软骨内骨生长这一创建骨组织的关键过程受到负向调控。

FGFR3

. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of

VOXZOGO 是一种 C 型利钠肽 (CNP) 类似物，作为下游信号通路的正向调节剂发挥作用。

FGFR3

to promote endochondral bone growth.

促进软骨内骨生长。

VOXZOGO is approved in the U.S.,

VOXZOGO已在美国获得批准，

Japan

日本

and

和

Australia

澳大利亚

to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity.

在所有年龄段的软骨发育不全且骨骺未闭合的儿童中增加线性生长，VOXZOGO在欧盟被批准用于治疗4个月及以上、经适当基因检测确认骨骺未闭合的软骨发育不全儿童。在美国，该适应症基于年生长速度的改善获得了加速批准。

Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history..

持续批准可能取决于在确证性试验中对临床益处的验证和描述。为了满足这一上市后要求，BioMarin 打算利用正在进行的开放标签扩展研究，并与可用的自然史数据进行比较。

Patient Support Accessing VOXZOGO

患者支持获取VOXZOGO

To reach a BioMarin RareConnections

要联系BioMarin RareConnections

Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail

案例经理，请拨打免费电话1-833-VOXZOGO（1-833-869-9646）或发送电子邮件

VOXZOGOSupport@biomarin-rareconnections.com

. For more information about VOXZOGO, please visit

如需更多关于VOXZOGO的信息，请访问

www.voxzogo.com

. For additional information regarding this product, please contact BioMarin Medical Information at

有关此产品的更多信息，请联系BioMarin医学信息部，联系方式为

medinfo@bmrn.com

。

VOXZOGO U.S. Important Safety Information

VOXZOGO 美国重要安全信息

What is VOXZOGO used for?

VOXZOGO 是用来做什么的？

VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses).

VOXZOGO 是一种用于增加软骨发育不全且生长板（骨骺）开放的儿童线性生长的处方药。

VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

VOXZOGO 是基于年生长速度的提高而获得加速批准的。持续的批准可能取决于在确证性试验中对临床益处的验证和描述。

What is the most important safety information about VOXZOGO?

关于VOXZOGO最重要的安全信息是什么？

VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO..

VOXZOGO 可能会引起严重的副作用，包括部分患者血压暂时下降。为了降低血压下降及相关症状（头晕、疲倦或恶心）的风险，患者应在接受 VOXZOGO 治疗前 1 小时内进食并饮用 8 至 10 盎司的液体。

What are the most common side effects of VOXZOGO?

VOXZOGO 最常见的副作用是什么？

The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO.

VOXZOGO最常见的副作用包括注射部位反应（包括发红、瘙痒、肿胀、瘀伤、皮疹、荨麻疹和注射部位疼痛）、血液碱性磷酸酶水平升高、呕吐、关节痛、血压下降和胃痛。这些并非VOXZOGO的所有可能副作用。

Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away..

请向您的医疗保健提供者咨询有关副作用的医疗建议，以及任何困扰患者或未消失的副作用。

How is VOXZOGO taken?

VOXZOGO是如何服用的？

VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO.

VOXZOGO 每日通过皮下注射给药，由护理人员在医疗保健提供者确认其有能力施用 VOXZOGO 后进行。在您的医疗保健提供者向您展示正确方法之前，请勿尝试注射 VOXZOGO。VOXZOGO 随附使用说明，描述了准备、注射和处理 VOXZOGO 的步骤。

Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made..

护理人员应查阅使用说明以获得指导，并在每次收到VOXZOGO补充装时检查是否有任何变更。

Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual.

每天大约在同一时间注射一次VOXZOGO。如果错过了一剂VOXZOGO，可以在错过剂量后的12小时内补上。超过12小时后，跳过错过的剂量，并按常规给予下一剂每日剂量。

The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups.

VOXZOGO的剂量基于体重。您的医疗保健提供者将根据定期检查后的体重变化调整剂量。

Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider.

您的医疗保健提供者将监测患者的生长情况，并在他们确定患者不再能够生长时告诉您何时停止服用VOXZOGO。如果您的医疗保健提供者指示，停止使用VOXZOGO。

What should you tell the doctor before or during taking VOXZOGO?

在服用VOXZOGO之前或期间，你应该告诉医生什么？

Tell your doctor about all of the patient's medical conditions including

告诉医生患者的所有医疗状况，包括

If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine).

如果患者患有心脏病（心脏或血管疾病），或者患者正在服用降压药（抗高血压药物）。

If the patient has kidney problems or renal impairment.

如果患者有肾脏问题或肾功能不全。

If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby.

如果患者怀孕或计划怀孕，尚不清楚VOXZOGO是否会伤害未出生的婴儿。

If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk.

如果患者正在哺乳或计划哺乳。尚不清楚VOXZOGO是否会进入母乳。

Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

告诉医生患者服用的所有药物，包括处方药和非处方药、维生素以及草药补充剂。

You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit

您可以拨打1-866-906-6100向BioMarin报告副作用。我们鼓励您向FDA报告处方药的负面副作用。访问

www.fda.gov/medwatch

, or call 1-800-FDA-1088.

，或者拨打1-800-FDA-1088。

Please see additional safety information in the full

请参阅完整内容中的更多安全信息

Prescribing Information

处方信息

and

和

Patient Information

患者信息

。

About BioMarin

关于BioMarin

BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The

BioMarin是一家全球生物技术公司，致力于将基因发现的承诺转化为对每位患者生活产生深远影响的药物。

San Rafael, California

加利福尼亚州圣拉斐尔

-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that offer new possibilities for people living with genetically defined conditions around the world.

总部位于-，成立于1997年，拥有八种商业疗法和强大的临床及临床前管线，创新成果显著。BioMarin采用独特的药物发现与开发方法，致力于通过开发定义类别的药物，充分释放基因科学的潜力，为全球患有基因定义疾病的患者提供新的可能性。

To learn more, please visit .

要了解更多信息，请访问。

www.biomarin.com

。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: data presented at the 2025 Joint Congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) and data to be shared at the Pediatric Endocrine Society Annual Meeting (PES), including the oral and poster presentations; VOXZOGO's efficacy, safety and impact on children with achondroplasia, including the potential benefits of early treatment with VOXZOGO on height increase, proportionality, quality of life and tibial bowing and potential to improve physical well-being for children into adulthood; VOXZOGO's potential benefits, safety and impact on children with hypochondroplasia and Turner syndrome, including potential increase in annualized growth velocity (AGV); BioMarin's commitment to develop new options for skeletal conditions; and BioMarin's CANOPY clinical development program for achondroplasia, hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome and SHOX deficiency, including BioMarin's plans and expectations to share topline data for its pivotal Phase 3 study with VOXZOGO in hypochondroplasia in 2026, with potential launch in 2027.

本新闻稿包含关于BioMarin Pharmaceutical Inc.（BioMarin）业务前景的前瞻性声明，包括但不限于以下内容的陈述：在2025年欧洲儿科内分泌学会（ESPE）和欧洲内分泌学会（ESE）联合大会上展示的数据，以及将在儿科内分泌学会年会（PES）上分享的数据，包括口头和海报展示；VOXZOGO对软骨发育不全儿童的疗效、安全性和影响，包括早期使用VOXZOGO治疗可能带来的身高增长、比例协调、生活质量改善、胫骨弯曲减少以及改善儿童直至成年期的身体健康状况的潜在益处；VOXZOGO对患有软骨发育低下和特纳综合症儿童的潜在益处、安全性及影响，包括年度生长速度（AGV）的潜在提升；BioMarin致力于开发骨骼疾病的新疗法；以及BioMarin针对软骨发育不全、软骨发育低下、特发性矮小症、努南综合症、特纳综合症和SHOX缺陷的CANOPY临床开发计划，包括BioMarin计划于2026年分享其针对软骨发育低下进行的VOXZOGO关键3期研究的顶线数据，并预计在2027年推出。

These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned pre-clinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S.

这些前瞻性陈述为预测性声明，涉及风险和不确定性，可能导致实际结果与这些陈述存在重大差异。这些风险和不确定性包括但不限于：VOXZOGO当前及计划中的临床前研究和临床试验的结果与时间安排；在临床试验中对患者持续监测所观察到的任何潜在不良事件；美国相关部门的决策内容与时机；

Food and Drug Administration, t.

食品药品监督管理局，t。

March 31, 2025

2025年3月31日

, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise..

，因为此类因素可能会因后续报告而更新。股东们被敦促不要过分依赖前瞻性声明，这些声明仅截至本日期有效。BioMarin 没有义务，也明确否认任何更新或更改任何前瞻性声明的义务，无论是由于新信息、未来事件或其他原因。

BioMarin

, BioMarin RareConnections

，BioMarin RareConnections

and VOXZOGO

和 VOXZOGO

are registered trademarks of BioMarin Pharmaceutical Inc.

是BioMarin Pharmaceutical Inc.的注册商标。

Contacts:

联系人：

Investors Media

投资者媒体