Investigator-Sponsored Phase 2 Study Demonstrated Positive Results for VOXZOGO in Children with Hypochondroplasia

研究者赞助的2期研究显示，在患有软骨发育不良的儿童中，VOXZOGO的阳性结果

New Analyses Highlight VOXZOGO 7-year Data and Improvement on Quality of Life in Children with Achondroplasia

新的分析突出了VOXZOGO 7年的数据和软骨发育不全儿童生活质量的改善

SAN RAFAEL, Calif., March 12, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that positive early results from an investigator-sponsored Phase 2 study of VOXZOGO® (vosoritide) in children with hypochondroplasia, will be presented at the 2024 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Toronto, Canada, March 12-16, 2024.

加利福尼亚州圣拉斐尔，2024年3月12日/PRNewswire/--BioMarin Pharmaceutical Inc.（纳斯达克：BMRN）宣布，由研究者赞助的VOXZOGO®（vosoritide）对软骨发育不良儿童的2期研究的早期阳性结果将于2024年3月12日至16日在加拿大多伦多举行的2024年美国医学遗传学和基因组学学院（ACMG）年度临床遗传学会议上公布。

Researchers will also present data from Phase 2 and Phase 3 studies of the medicine in children with achondroplasia, including results that demonstrate VOXZOGO's positive impact on quality of life. .

研究人员还将介绍该药物对软骨发育不全儿童的2期和3期研究数据，包括证明VOXZOGO对生活质量产生积极影响的结果。。

Results in Hypochondroplasia

软骨发育不良的结果

Andrew Dauber, M.D., will present positive results from his investigator-sponsored Phase 2 study of VOXZOGO in children with hypochondroplasia. The annualized growth velocity (AGV) increased from 5.12 cm/year during the observation period to 6.93 cm/year during the treatment period (mean difference: 1.81 cm/year, p<0.0001).

医学博士安德鲁·道伯（AndrewDauber）将从他的研究者赞助的关于软骨发育不良儿童VOXZOGO的2期研究中得出积极的结果。年化生长速度（AGV）从观察期的5.12 cm/年增加到治疗期的6.93 cm/年（平均差异：1.81 cm/年，p＜0.0001）。

No new safety signals were reported. .

没有报告新的安全信号。。

'We are encouraged by preliminary results from the first study evaluating VOXZOGO in children with hypochondroplasia, suggesting the medicine has the potential to impact growth outcomes for this group of patients,' said Dr. Dauber, who is the lead study author and Chief of Endocrinology at Children's National Hospital in Washington, D.C.

Dauber博士是该研究的主要作者兼华盛顿儿童国家医院内分泌科主任，他说：“我们对第一项评估VOXZOGO治疗软骨发育不良儿童的初步结果感到鼓舞，这表明该药物有可能影响这组患者的生长结果。”。

'There are currently no approved medicines for children with hypochondroplasia, so we look forward to gathering additional data to deepen our understanding of VOXZOGO in hypochondroplasia and other growth-related conditions.'.

“目前还没有批准用于软骨发育不良儿童的药物，因此我们期待着收集更多数据，以加深我们对软骨发育不良和其他生长相关疾病中VOXZOGO的理解。”。

In late 2023, BioMarin launched the pivotal clinical trial program studying the safety and efficacy of VOXZOGO in children with hypochondroplasia. A multinational observational study in children with hypochondroplasia (111-902) is currently recruiting participants, and the company plans to enter the treatment phase (Phase 3 trial) later this year. .

2023年末，BioMarin启动了关键的临床试验计划，研究VOXZOGO在软骨发育不良儿童中的安全性和有效性。一项针对软骨发育不良儿童（111-902）的多国观察性研究目前正在招募参与者，该公司计划在今年晚些时候进入治疗阶段（3期试验）。。

New Data Highlights VOXZOGO Long-Term Efficacy, Safety and Quality of Life in Achondroplasia

新数据突出了VOXZOGO在软骨发育不全中的长期疗效、安全性和生活质量

7-year Phase 2 Results in Achondroplasia

7年2期导致软骨发育不全

Data from two studies of VOXZOGO in children ≥5 years of age with achondroplasia demonstrated a sustained improvement in AGV for each year that the participants were treated. In the Phase 2 study with more than 7 years of follow up, the mean increase in growth across each year of age up to 16 years compared with untreated participants was 1.63 cm/year for boys and 1.33 cm/year for girls.

来自两项针对软骨发育不全≥5岁儿童的VOXZOGO研究的数据表明，参与者接受治疗的每年AGV持续改善。在为期7年以上的第二阶段研究中，与未经治疗的参与者相比，16岁以下的参与者每年的平均增长率为男孩1.63厘米/年，女孩1.33厘米/年。

Separate cross-sectional comparative analysis assessing increase in height over a 7-year period showed an additional height gain of 11.03 cm with use of VOXZOGO compared to matched untreated children over the same period..

评估7年期间身高增长的单独横断面比较分析显示，与同期未经治疗的匹配儿童相比，使用VOXZOGO可额外增加11.03厘米的身高。。

4-year Phase 3 Results in Achondroplasia

4年3期导致软骨发育不全

The same analyses were produced for the Phase 3 trial in which children with achondroplasia have a mean treatment follow-up of 4 years. The mean increase in growth across each year of age up to 17 years was 1.73 cm/year for boys and 1.46 cm/year for girls. A separate cross-sectional comparative analysis assessing increase in height over a 3-year period showed an additional height gain of 5.75 cm with use of VOXZOGO compared to matched untreated children over the same period..

对于软骨发育不全的儿童平均治疗随访4年的3期试验也进行了相同的分析。17岁以下的男孩每年的平均增长率为1.73厘米/年，女孩为1.46厘米/年。一项单独的横断面比较分析评估了3年内身高的增加情况，结果显示，与同期未经治疗的匹配儿童相比，使用VOXZOGO后，身高增加了5.75厘米。。

3-year Phase 3 Quality of Life Results in Achondroplasia

软骨发育不全的3年3期生活质量结果

Results from a separate analysis of the Phase 3 study showed that VOXZOGO improved health-related quality of life (HRQoL) among children with achondroplasia, particularly those associated with physical activities. After 3 years, the mean increase in Quality of Life in Short Stature Youth (QoLISSY) physical domain score was 6.0 as reported by caregivers and 6.3 as reported by children.

对第三阶段研究的单独分析结果表明，VOXZOGO改善了软骨发育不全儿童的健康相关生活质量（HRQoL），尤其是与体育活动相关的儿童。3年后，照顾者报告的矮身材青年（QoLISSY）身体领域得分的平均生活质量提高为6.0，儿童报告为6.3。

These improvements were even more pronounced in children who grew more (for those with ≥ 1 SD increase in height z-score, the mean increase in physical domain score was 11.4 as reported by caregivers and 8.5 as reported by children)..

这些改善在生长速度更快的儿童中更为明显（对于身高z评分增加≥1 SD的儿童，照顾者报告的身体领域评分平均增加11.4，儿童报告的平均增加8.5）。。

'The clinical results at ACMG reinforce the long-term benefit we are seeing in children treated with VOXZOGO, with a meaningful median height gain of more than 11 cm in children with achondroplasia following 7 years of treatment,' said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin.

BioMarin全球研发总裁汉克·福斯（HankFuchs）医学博士说：“ACMG的临床结果加强了我们在接受VOXZOGO治疗的儿童中看到的长期益处，经过7年的治疗，软骨发育不全儿童的中位身高增加超过11厘米。”。

'We are particularly encouraged by new data demonstrating the potential impact height gains from VOXZOGO can have on quality of life, an outcome of incredible importance for children and families impacted by achondroplasia.'.

“我们特别受到鼓舞的是，新数据表明，VOXZOGO的身高增长可能会对生活质量产生潜在影响，这一结果对受软骨发育不全影响的儿童和家庭具有难以置信的重要性。”。

'Achondroplasia can have a negative effect on a child's quality of life, including physical, social and emotional aspects,' said Melita Irving, M.D., study investigator and consultant clinical geneticist at Guy's and St. Thomas' NHS Foundation Trust in London. 'Our studies showed that increasing height with long-term administration of VOXZOGO can result in meaningful improvements in quality-of-life measures for children with achondroplasia.'.

伦敦盖伊和圣托马斯NHS基金会信托基金会（Guy's and St.Thomas'Foundation Trust）的研究研究员兼临床遗传学家顾问梅利塔·欧文（MelitaIrving）医学博士说，软骨发育不全会对孩子的生活质量产生负面影响，包括身体、社会和情感方面我们的研究表明，长期服用VOXZOGO可以增加身高，从而有意义地改善软骨发育不全儿童的生活质量。

BioMarin's full list of presentations at ACMG includes:

BioMarin在ACMG的完整演讲列表包括：

BioMarin Poster Presentations

BioMarin海报展示

Persistence of Growth-Promoting Effects in Children with Achondroplasia Up to 7 Years: Update from a Phase 2 Extension Study with Vosoritide Poster #: P144Friday, March 15, 2024, 10:30 a.m. – 12 p.m. ET

软骨发育不全7岁以下儿童的生长促进作用持续存在：Vosoritide海报第二阶段扩展研究的最新进展：P144 2024年3月15日星期五，美国东部时间上午10:30–下午12:00

Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia for Up to 4 Years: Update from Phase 3 Extension StudyPoster #: P139Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET

Vosoritide对软骨发育不全儿童的持续生长促进作用长达4年：第三阶段扩展研究的更新Poster#：P139 2024年3月14日星期四，美国东部时间上午10:30–下午12:00

Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia is Accompanied by Improvement in Physical Aspects of Quality of Life Poster #: P141Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET

Vosoritide对软骨发育不全儿童的持续生长促进作用伴随着生活质量身体方面的改善海报#：P141 2024年3月14日星期四，美国东部时间上午10:30–下午12:00

Persistence of Growth-Promoting Effects in Infants and Toddlers with Achondroplasia: Results from a Phase 2 Extension Study with Vosoritide Poster #: P131Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET

软骨发育不全婴幼儿生长促进作用的持续存在：Vosoritide海报第二阶段扩展研究的结果#：P131 2024年3月14日星期四，美国东部时间上午10:30–下午12:00

Exploring the Landscape of Phenylketonuria Education and Learning Needs of Genetics Trainees Poster #: P049Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET

探索苯丙酮尿症教育的前景和遗传学受训者的学习需求海报#：P049 2024年3月14日星期四，美国东部时间上午10:30–下午12:00

Reduction of Blood Phenylalanine in Participants Enrolled in OPAL, an Observational Study, Mirror Findings from the U.S.-based PRISM Population Poster #: P034Friday, March 15, 2024, 10:30 a.m. – 12 p.m. ET

OPAL观察性研究参与者血液苯丙氨酸的减少，反映了美国PRISM人口海报的发现#：P034 2024年3月15日星期五，美国东部时间上午10:30–下午12:00

Investigator-Sponsored Oral Presentation

调查员赞助的口头陈述

Vosoritide Increases Growth Velocity in Hypochondroplasia: Phase 2 Trial Results Oral Session: O03 Thursday, March 14, 2024, 1:30 – 3 p.m. ET

Vosoritide增加软骨发育不良的生长速度：第二阶段试验结果口头会议：O03，2024年3月14日，星期四，美国东部时间下午1:30–3:00

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 FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth..

在软骨发育不全的儿童中，由于FGFR3的功能获得性突变，软骨内骨生长（骨组织产生的基本过程）受到负调控。VOXZOGO是一种C型利钠肽（CNP）类似物，可作为FGFR3下游信号通路的正调节剂，促进软骨内骨生长。。

VOXZOGO is approved in the U.S. and indicated to increase linear growth in children with achondroplasia with open epiphyses. 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).

VOXZOGO在美国获得批准，并被证明可以增加骨骺开放的软骨发育不全儿童的线性生长。基于年化增长速度的改善，该适应症在加速批准下获得批准。继续批准可能取决于验证性试验中临床益处的验证和描述。

To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history..

为了满足这一上市后的要求，BioMarin打算使用正在进行的开放标签扩展研究，与现有的自然史进行比较。。

Patient Support Accessing VOXZOGO

患者支持访问VOXZOGO

To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail VOXZOGOSupport@biomarin-rareconnections.com. For more information about VOXZOGO, please visit www.voxzogo.com. For additional information regarding this product, please contact BioMarin Medical Information at medinfo@bmrn.com. .

要联系BioMarin RareConnections®案例经理，请拨打免费电话1-833-VOXZOGO（1-833-869-9646）或电子邮件VOXZOGOSupport@biomarin-rareconnections.com.有关VOXZOGO的更多信息，请访问www.VOXZOGO.com。有关此产品的更多信息，请联系BioMarin Medical informationmedinfo@bmrn.com.。

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.

软骨发育不全是导致人类身材矮小的骨骼发育不良的最常见形式，其特征是软骨内骨化减慢，导致长骨，脊柱，面部和颅底的身材矮小和结构紊乱。

This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth..

这种情况是由FGFR3基因的变化引起的，FGFR3基因是骨骼生长的负调节剂。。

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%的软骨发育不全儿童的父母身高平均，并且由于自发的基因突变而患病。全世界软骨发育不全的发病率约为25000例活产中的一例。VOXZOGO正在对生长板仍然“开放”的儿童进行测试，这些儿童通常是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。。

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，用于增加软骨发育不全和开放生长板（骨骺）儿童的线性生长。

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基于年化增长速度的提高而获得加速批准。继续批准可能取决于验证性试验中临床益处的验证和描述。

What is the most important safety information about VOXZOGO?

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，包括一些患者的血压暂时下降。为了降低血压下降和相关症状（头晕、疲倦或恶心）的风险，患者应在服用VOXZOGO前1小时内进食并饮用8至10盎司液体。。

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），血液检查中显示的高水平血液碱性磷酸酶，呕吐，关节疼痛，血压下降和胃痛。这些并不是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..

询问您的医疗保健提供者有关副作用的医疗建议，以及任何困扰患者或不会消失的副作用。。

How is VOXZOGO taken?

VOXZOGO是如何拍摄的？

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每天在皮肤下注射，在医疗保健提供者确定护理人员能够服用VOXZOGO后，由护理人员进行管理。在你的医疗保健提供者向你展示正确的方法之前，不要尝试注射VOXZOGO。VOXZOGO提供了使用说明，描述了准备，注射和处置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..

护理人员应审查指导使用说明，并在任何时候收到VOXZOGO补液，以防发生任何变化。。

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VOXZOGO 1次。如果错过了一剂VOXZOGO，可以在错过剂量的12小时内给予。12小时后，跳过错过的剂量，照常服用下一次每日剂量。

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诉您何时停止服用VOXZOGO。如果您的医疗保健提供者指示，请停止管理VOXZOGO。

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

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）。

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。目前尚不清楚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.

如果患者正在母乳喂养或计划母乳喂养。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. 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 www.fda.gov/medwatch, or call 1-800-FDA-1088..

告诉你的医生病人服用的所有药物，包括处方药和非处方药、维生素和草药补充剂。您可以通过1-866-906-6100向BioMarin报告副作用。鼓励您向FDA报告处方药的负面副作用。访问www.fda.gov/medwatch，或致电1-800-fda-1088。。

Please see additional safety information in the full Prescribing Information and Patient Information.

请参阅完整处方信息和患者信息中的其他安全信息。

About BioMarin

关于BioMarin

Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of the genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders.

BioMarin成立于1997年，是一家全球生物技术公司，致力于通过基因发现改变生命。该公司开发并商业化了针对遗传病根源的靶向疗法。BioMarin无与伦比的研究和开发能力为罕见遗传疾病患者带来了八种转化性商业疗法。

The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options.

该公司独特的药物发现方法已经产生了多种商业，临床和临床前候选药物，这些候选药物可以解决严重未满足的医疗需求，对生物学有很好的理解，并提供了一个机会，可以率先上市或提供比现有治疗方案更大的益处。

For additional information, please visit www.biomarin.com..

有关更多信息，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 to be presented at the 2024 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting, including the six poster presentations and the investor-sponsored oral presentation; the development of BioMarin's VOXZOGO program generally; the potential benefits of VOXZOGO for children with achondroplasia, including the duration of such benefits, potential improvement in annualized growth velocity (AGV), and potential improvement in health-related quality of life; the potential efficacy of VOXZOGO for children with hypochondroplasia, including potential to impact growth outcomes; and the continued clinical development of VOXZOGO, including BioMarin's plans for launching the treatment phase of the Phase 3 trial studying the safety and efficacy of VOXZOGO in children with hypochondroplasia.

本新闻稿包含有关BioMarin Pharmaceutical Inc.（BioMarin）业务前景的前瞻性声明，包括但不限于以下声明：将在2024年美国医学遗传学和基因组学学院年度临床遗传学会议上提交的数据，包括六海报演示和投资者赞助的口头演示；BioMarin的VOXZOGO程序的开发；VOXZOGO对软骨发育不全儿童的潜在益处，包括这种益处的持续时间，年化生长速度（AGV）的潜在改善以及与健康相关的生活质量的潜在改善；VOXZOGO对软骨发育不良儿童的潜在疗效，包括影响生长结果的潜力；以及VOXZOGO的持续临床开发，包括BioMarin计划启动3期临床试验的治疗阶段，研究VOXZOGO在软骨发育不良儿童中的安全性和有效性。

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 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.

这些前瞻性陈述是预测，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：VOXZOGO当前和计划的临床前研究和临床试验的结果和时间安排；在临床试验中持续监测患者时观察到的任何潜在不良事件；美国决策的内容和时机。

Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption 'Risk Factors' in BioMarin's Annual Report on Form 10-K for the .

食品和药物管理局、欧洲药品管理局、欧盟委员会和其他监管机构；以及BioMarin向美国证券交易委员会提交的文件中详细列出的因素，包括但不限于BioMarin年度报告10-K表格中“风险因素”标题下包含的因素。

Contacts:

联系人：

Investors

投资者

Media

媒体

Traci McCarty

Traci McCarty。

Andrew Villani

Andrew Continuous Villani

BioMarin Pharmaceutical Inc.

BioMarin制药公司。

BioMarin Pharmaceutical Inc.

BioMarin制药公司。

(415) 455-7558

(415) 455-7558

(628) 269-7393

(628) 269-7393

SOURCE BioMarin Pharmaceutical Inc.

来源BioMarin Pharmaceutical Inc。