BioMarin在美国骨与矿物研究学会年会上发布新数据，进一步深化对软骨发育不全和软骨发育不良治疗的理解-动脉网

BioMarin Presents New Data Advancing Understanding of Achondroplasia and Hypochondroplasia Treatment at the American Society for Bone and Mineral Research Annual Meeting

CISION 等信源发布 2026-10-09 21:05

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New analysis showcases impact of VOXZOGO

新分析展示VOXZOGO的影响

(vosoritide) in foramen magnum and craniocervical junction growth in children with achondroplasia, including infants

（伏索利肽）对包括婴儿在内的软骨发育不全儿童枕骨大孔及颅颈交界区生长的影响

SAN RAFAEL, Calif.

加利福尼亚州圣拉斐尔

，

Oct. 9, 2026

2026年10月9日

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

/美通社/ -- BioMarin Pharmaceutical Inc.（纳斯达克：

BMRN

) today announced it will present new data from 13 studies at the American Society for Bone and Mineral Research 2026 Annual Meeting (ASBMR) in Boston, including results from studies of VOXZOGO

）今日宣布，将在波士顿举行的2026年美国骨与矿物研究学会（ASBMR）年会上公布来自13项研究的新数据，其中包括VOXZOGO相关研究的结果。

(vosoritide) in children with achondroplasia and hypochondroplasia. The company will also share data from studies of the investigational long-acting medicine BMN 333.

（伏索利肽）用于患有软骨发育不全和软骨发育不良的儿童。该公司还将分享关于在研长效药物 BMN 333 的研究数据。

The data include encouraging new imaging results from a post-hoc analysis of a randomized, double-blind, placebo-controlled trial evaluating the effect of VOXZOGO on the growth of the foramen magnum and craniocervical junction in children with achondroplasia, including infants.

这些数据包括来自一项随机、双盲、安慰剂对照试验的事后分析中令人鼓舞的新影像学结果，该试验评估了VOXZOGO对软骨发育不全儿童（包括婴儿）枕骨大孔和颅颈交界区生长的影响。

'These findings provide important new insight into the potential effect of VOXZOGO on growth-related dimensions of the foramen magnum and craniocervical junction, regions of the skull implicated in cervicomedullary compression and serious complications in children with achondroplasia,' said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin.

“这些发现为VOXZOGO对枕骨大孔和颅颈交界区生长相关维度的潜在影响提供了重要的新见解，而颅骨的这些区域与软骨发育不全儿童的延髓受压及严重并发症密切相关。”BioMarin执行副总裁兼首席研发官Greg Friberg博士表示。

'The results also underscore the potential VOXZOGO treatment may offer when started as early as possible, when it can have the greatest impact. Together with the additional data we're presenting on bone strength, limb alignment, and our emerging work in hypochondroplasia and next-generation medicines such as BMN 333, these findings reflect the depth and breadth of our commitment to understanding and improving outcomes for children living with skeletal dysplasias.'.

“研究结果还强调，VOXZOGO 治疗在尽早启动时可能带来的潜力，因为此时其影响最大。结合我们此次展示的关于骨强度、肢体对线方面的额外数据，以及我们在软骨发育不全和下一代药物（如 BMN 333）方面的新兴研究成果，这些发现体现了我们致力于深入理解并改善骨骼发育不良患儿预后的坚定承诺。”

BioMarin recently submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for VOXZOGO for the treatment of hypochondroplasia. The company is also on track with its submissions to the European Medicines Agency (EMA) and other regional health authorities. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch..

BioMarin 最近向美国食品药品监督管理局（FDA）提交了 VOXZOGO 用于治疗软骨发育不全的补充新药申请（sNDA）。该公司向欧洲药品管理局（EMA）及其他地区卫生监管机构的提交工作也正按计划推进。如果获得批准，VOXZOGO 将成为首个针对软骨发育不全的靶向疗法，并有望于 2027 年上市。

Additional data to be presented at the meeting include research in hypophosphatasia, as well as results from the Phase 3 ENERGY 3 study of BMN 401 in children ages 1 to 12 years with ENPP1 deficiency.

将在会议上呈现的额外数据包括关于低磷酸酶症的研究，以及BMN 401在治疗1至12岁ENPP1缺乏症儿童的3期ENERGY 3研究中的结果。

Below are key presentations at ASBMR, with all times listed in Eastern Daylight Time:

以下是ASBMR会议的关键报告，所有时间均按东部夏令时（EDT）列出：

C-Type Natriuretic Peptide (CNP) Agonist CNP38 Promotes Longitudinal Bone Growth by Growth Plate Expansion in the Hyp Mouse Model of XLH

C型利钠肽（CNP）激动剂CNP38通过生长板扩张促进XLH的Hyp小鼠模型中的纵向骨生长

Oral Presentation #1138

口头报告 #1138

Monday, Oct. 12, 11:30 – 11:42 a.m.

10月12日，星期一，上午11:30 – 11:42

BMN 333, a Long-Acting Vosoritide Investigational Product, Promotes Endochondral Bone Growth and Skeletal Microarchitecture in Juvenile Mice and Nonhuman Primates

BMN 333，一种长效伏索利肽 investigational product，促进幼年小鼠和非人灵长类动物的软骨内骨生长和骨骼微结构

Poster Presentation #092

海报展示 #092

Friday, Oct. 9, 5:30 – 7 p.m.

10月9日，星期五，下午5:30至7:00

Sunday, Oct. 11, 2 – 3:30 p.m.

10月11日，星期日，下午2:00 – 3:30

Examining the Effect of Vosoritide Treatment on Bone Strength in Children With Achondroplasia

评估伏索利肽治疗对软骨发育不全儿童骨强度的影响

Poster Presentation #486

海报展示 #486

Saturday, Oct. 10, 2 – 3:30 p.m.

10月10日（星期六）下午2:00 – 3:30

Efficacy and Safety of BMN 401 in Children With ENPP1 Deficiency: 52-Week Results

BMN 401在ENPP1缺乏症儿童中的有效性和安全性：52周结果

From

从

the Pivotal ENERGY 3 Trial

Pivotal ENERGY 3 试验

Poster Presentation #554

海报展示 #554

Saturday, Oct. 10, 2 – 3:30 p.m.

10月10日，星期六，下午2点至3点30分

Economic Burden of Hypophosphatasia: A Systematic Literature Review

低磷酸酯酶症的经济负担：一项系统文献综述

Poster Presentation #473

海报展示 #473

Saturday, Oct. 10, 2 – 3:30 p.m.

10月10日，星期六，下午2点至3点30分

Development of BMN 333, a Long-Acting Version of Vosoritide for FGFR3-Related Dwarfism and Other Skeletal Dysplasias

BMN 333（一种长效型Vosoritide）的开发，用于治疗FGFR3相关侏儒症及其他骨骼发育不良

Poster Presentation #115

海报展示 #115

Sunday, Oct. 11, 2 – 3:30 p.m.

10月11日，星期日，下午2:00 – 3:30

Comparison of Quantitative Computed Tomography Bone Mineral Density Measurements of Vosoritide-Treated Children With Achondroplasia to an Untreated Achondroplasia Reference Population

接受伏索利肽治疗的软骨发育不全儿童与未治疗软骨发育不全参照人群的定量计算机断层扫描骨密度测量比较

Poster Presentation #474

海报展示 #474

Sunday, Oct. 11, 2 – 3:30 p.m.

10月11日，星期日，下午2:00 – 3:30

Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial Results

伏索利肽提高软骨发育不全儿童的生长速度：三期临床试验结果

Poster Presentation #552

海报展示 #552

Sunday, Oct. 11, 2 – 3:30 p.m.

10月11日（星期日）下午2:00 – 3:30

The Effect of Vosoritide on Growth of the Foramen Magnum and Craniocervical Junction in Children With Achondroplasia

Vosoritide对软骨发育不全儿童枕骨大孔及颅颈交界区生长的影响

Poster Presentation #483

海报展示 #483

Sunday, Oct. 11, 2 – 3:30 p.m.

10月11日，星期日，下午2点至3点30分

Molecular Insights into Vosoritide's Role in Enhancing Bone Growth and Mineralization

分子层面解析Vosoritide在促进骨骼生长与矿化中的作用

Poster Presentation #362

海报展示 #362

Sunday, Oct. 11, 2 – 3:30 p.m.

10月11日，星期日，下午2点至3点30分

Understanding the Impact of Hypophosphatasia (HPP) on Health-Related Quality-of-Life and Activities of Daily Living: A Patient-Based Conceptual Model

理解低磷酸酯酶症（HPP）对健康相关生活质量和日常活动的影响：一个基于患者的概念模型

Poster Presentation #011

海报展示 #011

Sunday, Oct. 11, 2 – 3:30 p.m.

10月11日，星期日，下午2:00 – 3:30

Prospective, Observational Study of a Cohort of Hypophosphatasia Patients' Phenotypes Over Time and the Utility of Digital Outcomes Measures in This Population

低磷酸酯酶症患者表型随时间变化的前瞻性观察性研究及数字结局指标在该人群中的应用价值

Poster Presentation #521

海报展示 #521

Sunday, Oct. 11, 2 – 3:30 p.m.

10月11日，星期日，下午2点至3点30分

ENPP1 Inhibition Improves the Bone Phenotype, Mobility, Muscle Strength and Endurance in Later-Onset HPP Mice

ENPP1抑制改善迟发型低磷酸酯酶症小鼠的骨表型、活动能力、肌肉力量和耐力

Oral Presentation #1139

口头报告 #1139

Monday, Oct. 12, 11:42 – 11:54 a.m.

10月12日，星期一，上午11:42 – 11:54

About Achondroplasia

关于软骨发育不全

Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the .

软骨发育不全是人类中最常见的骨骼发育不良形式，导致不成比例的矮小身材，其特征是软骨内骨化减缓，从而导致长骨、脊柱、面部和颅底的不成比例矮小和结构紊乱。这种情况是由...的变化引起的。

FGFR3

成纤维细胞生长因子受体3

gene, a negative regulator of bone growth.

基因，一种骨生长的负调控因子。

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

超过80%的软骨发育不全患儿父母身高正常，其患病原因是自发性基因突变。全球软骨发育不全的发病率约为每25,000名活产婴儿中有1例。VOXZOGO正在骨骺仍“开放”的儿童中进行测试，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别。

For more information about our clinical trials in achondroplasia, hypochondroplasia and other skeletal conditions, please visit

如需了解更多关于我们针对软骨发育不全、低软骨发育不全及其他骨骼疾病的临床试验信息，请访问

clinicaltrials.biomarin.com

。

About Hypochondroplasia

关于软骨发育不全

Hypochondroplasia is a rare, genetic skeletal dysplasia characterized by impaired bone growth, leading to disproportionate short stature and skeletal differences that can affect the long bones, spine and other parts of the skeleton and may impact physical functioning and overall quality of life. The condition presents with a broad and variable clinical spectrum and may include otolaryngologic (related to the ears, nose and throat) and neurological complications and is often diagnosed in toddlerhood or early school age based on clinical and radiological findings.

软骨发育不全是一种罕见的遗传性骨骼发育不良，其特征为骨骼生长受损，导致不成比例的矮小身材以及骨骼差异，可影响长骨、脊柱及其他骨骼部位，并可能影响身体功能和整体生活质量。该病的临床表现广泛且多变，可能包括耳鼻喉科（与耳、鼻、喉相关）及神经系统并发症，通常根据临床和影像学表现，在幼儿期或学龄早期确诊。

BioMarin estimates that roughly 14,000 children with hypochondroplasia within the company's global footprint may be eligible for treatment with VOXZOGO. .

BioMarin 估计，在其全球业务覆盖范围内，约有 14,000 名患有软骨发育不全的儿童可能符合使用 VOXZOGO 进行治疗的条件。

There are currently no medicines approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia.

目前，美国食品药品监督管理局或欧洲药品管理局尚未批准任何用于治疗软骨发育不全的药物。

For more information about our clinical trials in hypochondroplasia, achondroplasia and other skeletal conditions, please visit

如需了解更多关于我们针对软骨发育不全、侏儒症及其他骨骼疾病的临床试验信息，请访问

clinicaltrials.biomarin.com

。

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（一种生成骨组织的关键过程）受到负向调控

FGFR3

成纤维细胞生长因子受体3

. 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

成纤维细胞生长因子受体3

to promote endochondral bone growth.

以促进软骨内成骨生长。

VOXZOGO is the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries.

VOXZOGO 是唯一获批用于支持从出生起患有软骨发育不全的儿童生长的药物，国际共识指南建议尽早开始使用 VOXZOGO。自2021年首次获批以来，VOXZOGO 已帮助全球50多个国家的5000多名婴幼儿和儿童。

Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures. .

通过我们持续进行的研究，BioMarin 继续评估 VOXZOGO 在与软骨发育不全患者相关的关键临床终点上的效果，例如臂展、胫骨弯曲（腿弯曲）、身体比例、脊柱形态（包括椎管狭窄）以及生活质量指标。

VOXZOGO is approved in the U.S., Japan and Australia 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.

VOXZOGO 已在美国、日本和澳大利亚获批，用于增加所有年龄阶段且骨骺未闭合的软骨发育不全儿童的线性生长；在欧盟，VOXZOGO 的适应症为治疗经适当基因检测确认骨骺未闭合的 4 个月及以上软骨发育不全儿童。

In the U.S., this indication 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 trial(s). An sNDA with long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth such as body proportionality and arm span, is under review with an FDA Prescription Drug User Fee Act (PDUFA) target action date of Feb.

在美国，该适应症基于年化生长速度的改善，通过加速批准途径获得批准。继续获批可能取决于在确证性试验中对临床获益的验证和描述。一项包含三项进行中研究的长期安全性和有效性数据（包括成年身高以及除线性生长外的其他临床结局，如身体比例和臂展）的补充新药申请（sNDA）正在审评中，美国食品药品监督管理局（FDA）根据《处方药用户付费法案》（PDUFA）设定的目标行动日期为2月。

28, 2027..

2027年28日..

The use of VOXZOGO to treat hypochondroplasia has not yet been approved by any regulatory agency.

VOXZOGO 用于治疗软骨发育不全尚未获得任何监管机构的批准。

Patient Support Accessing VOXZOGO

患者支持：获取VOXZOGO

BioMarin's robust support services have ensured a seamless treatment experience, spearheaded by Clinical Coordinators, who have conducted hundreds of trainings for families with achondroplasia since approval. BioMarin provides resources to support families navigating achondroplasia, including a caregiver mentorship program that connects parents with other caregivers, and a U.S.

BioMarin 强有力的支持服务确保了无缝的治疗体验，由临床协调员牵头，自获批以来，他们已为患有软骨发育不全的家庭开展了数百次培训。BioMarin 提供资源以支持应对软骨发育不全的家庭，包括一个将父母与其他照护者联系起来的照护者导师计划，以及在美国的

doctor directory that helps families and healthcare professionals identify clinicians experienced in achondroplasia care..

帮助家庭和医疗保健专业人员识别在软骨发育不全护理方面经验丰富的临床医生的医生名录。

To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or email

如需联系 BioMarin RareConnections® 案例经理，请拨打免费电话 1-833-VOXZOGO（1-833-869-9646）或发送电子邮件至

[email protected]

. For more information about VOXZOGO, please visit

有关 VOXZOGO 的更多信息，请访问

www.voxzogo.com

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

如需获取有关本产品的更多信息，请联系 BioMarin 医学信息部，联系方式如下：

[email protected]

。

VOXZOGO U.S. Important Safety Information

VOXZOGO 美国重要安全信息

What is VOXZOGO used for?

VOXZOGO 的用途是什么？

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

VOXZOGO 是一种处方药，用于促进患有软骨发育不全且骨骺（生长板）尚未闭合的儿童的身高增长。

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 基于年化生长速度的改善而获得加速批准。持续批准可能取决于在确证性试验中对临床获益的验证和描述。

What is the most important safety information about VOXZOGO?

关于 VOXZOGO 的最重要安全信息是什么？

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 可能导致严重的副作用，包括部分患者出现暂时性血压下降。为降低血压下降及相关症状（头晕、乏力或恶心）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 最常见的副作用有哪些？

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）、血液检查显示碱性磷酸酶水平升高、呕吐、关节痛、血压降低和胃痛。这些并非 VOXZOGO 所有可能的副作用。

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. .

请咨询您的医疗保健提供者，获取关于副作用的医学建议，特别是针对那些令患者困扰或持续不退的副作用。

How is VOXZOGO taken?

如何服用VOXZOGO？

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 需每日作为皮下注射给药，由照护者在医疗保健提供者确定其具备给药能力后执行。在医疗保健提供者展示正确的注射方法之前，请勿尝试自行注射 VOXZOGO。VOXZOGO 随附《使用说明》，其中详述了准备、注射及处置 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书以获取指导，并在每次收到VOXZOGO补充配药时进行查阅，以防说明书有任何变更。

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.

每日注射一次VOXZOGO，尽量在每天相同的时间进行。如果漏打了一剂VOXZOGO，可以在漏打后的12小时内补打。超过12小时后，跳过漏打的剂量，按常规时间注射下一剂。

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随访期间体重的变化调整剂量。

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，并在确定患者不再能够生长时告知您何时停止服用VOXZOGO。如果医疗保健提供者指示，请停止给药VOXZOGO。

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

在服用 VOXZOGO 之前或期间，您应该告知医生哪些信息？

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

告知医生患者的所有健康状况，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.

如果患者存在肾脏问题或肾功能损害。

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。目前尚不清楚VOXZOGO是否会对胎儿造成伤害。

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。目前尚不清楚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.

请告知医生患者正在服用的所有药物，包括处方药、非处方药、维生素和草本补充剂。

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

您可以致电 1-866-906-6100 向 BioMarin 报告副作用。我们鼓励您向美国食品药品监督管理局（FDA）报告处方药的不良反应。请访问

www.fda.gov/medwatch

, or call 1-800-FDA-1088.

，或拨打 1-800-FDA-1088。

Please see additional safety information in the full

请参阅完整文档中的其他安全信息

Prescribing Information

处方信息

and

和

Patient Information

患者信息

。

About BioMarin

关于BioMarin

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline.

BioMarin 是一家领先的全球罕见病生物技术公司，专注于为患有遗传性疾病的患者提供药物。该公司成立于 1997 年，总部位于加利福尼亚州圣拉斐尔，拥有经过验证的创新记录，目前已推出九种商业化疗法，并拥有强大的临床和临床前研发管线。

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 have a profound impact on patients. To learn more, please visit .

BioMarin 采用独特的药物研发方法，致力于通过开发具有深远患者影响力的开创性药物，充分释放遗传科学的潜力。欲了解更多信息，请访问。

www.biomarin.com

。

Forward-Looking Statements

前瞻性陈述

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the data to be presented at the American Society for Bone and Mineral Research 2026 Annual Meeting (ASBMR), including the potential impact and safety of VOXZOGO in children with achondroplasia and hypochondroplasia; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including plans for submissions to the European Medicines Agency (EMA) and other regional health authorities as well as the expectation that, if approved by the U.S.

本新闻稿包含有关BioMarin Pharmaceutical Inc.（BioMarin）业务前景的前瞻性陈述，包括但不限于以下陈述：将在2026年美国骨与矿物研究学会（ASBMR）年会上公布的数据，包括VOXZOGO在软骨发育不全和软骨发育低下儿童中的潜在影响和安全性；BioMarin针对软骨发育低下儿童开发VOXZOGO的计划和预期，包括向欧洲药品管理局（EMA）及其他地区卫生监管机构提交申请的计划，以及如果获得美国批准

Food and Drug Administration (FDA), VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia with an anticipated launch in 2027; BioMarin's expectations regarding its supplemental New Drug Application (sNDA) for VOXZOGO for full approval in children with achondroplasia, including expectations regarding the Prescription Drug User Fee Act (PDUFA) target action date; BioMarin's plans and expectations for the development of BMN 333; and BioMarin's estimate regarding incidence and total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products, including achondroplasia and hypochondroplasia.

食品药品监督管理局（FDA），VOXZOGO 将成为首个用于治疗软骨发育不全的靶向疗法，预计于2027年上市；BioMarin 关于 VOXZOGO 补充新药申请（sNDA）以获得在软骨发育不全儿童中全面批准的预期，包括对《处方药用户费用法案》（PDUFA）目标行动日期的预期；BioMarin 对于 BMN 333 开发的计划和预期；以及 BioMarin 对其产品候选物和商业化产品所针对的疾病（包括软骨发育不全和软骨发育不良）的发病率和总可治疗患者人群（TAPP）的估计。

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 preclinical studies and clinical trials and the release of data from those trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the.

这些前瞻性陈述属于预测性质，涉及风险和不确定性，因此实际结果可能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：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，以及上述试验数据的发布；在临床试验患者持续监测过程中观察到的任何潜在不良事件；

BioMarin

, BioMarin RareConnections

，BioMarin RareConnections

and VOXZOGO

以及 VOXZOGO

are registered trademarks of BioMarin Pharmaceutical Inc.

是 BioMarin Pharmaceutical Inc. 的注册商标。

Contacts:

联系人：

Investors

投资者