基因疗法开发商BioMarin完成对Amicus Therapeutics的收购-动脉网

BioMarin Completes Acquisition of Amicus Therapeutics

CISION 等信源发布 2026-04-27 20:44

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Acquisition Adds Galafold

收购增加Galafold

(migalastat) for Fabry Disease and Pombiliti

（米加拉司特）用于法布里病和庞比立提

(cipaglucosidase alfa-atga) + Opfolda

（cipaglucosidase alfa-atga）+ Opfolda

(miglustat) for Pompe Disease to BioMarin's Commercial Portfolio

将（米格鲁司他）用于庞贝病纳入BioMarin的商业产品组合

BioMarin Expects to Provide Updated FY 2026 Guidance During its First Quarter Earnings Call, May 4, 2026

BioMarin预计将在其第一季度财报电话会议（2026年5月4日）上提供更新的2026财年指导。

SAN RAFAEL, Calif.

加利福尼亚州圣拉斐尔

，

April 27, 2026

2026年4月27日

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

/PRNewswire/ -- BioMarin制药公司（纳斯达克：

BMRN

) said today that it completed the previously announced agreement to acquire Amicus Therapeutics for $14.50 per share in an all-cash transaction for a total equity value of approximately $4.8 billion. The acquisition will strengthen BioMarin's commercial portfolio, adding two new treatments to the company's existing portfolio of medicines that target lysosomal storage diseases: Galafold.

）今天表示，已完成此前宣布的以每股 14.50 美元全现金交易收购 Amicus Therapeutics 的协议，总股权价值约为 48 亿美元。此次收购将加强 BioMarin 的商业产品组合，为其现有的针对溶酶体贮积症的药物组合增加两种新疗法：Galafold。

(migalastat), the first oral treatment for Fabry disease, and Pombiliti

（米加拉斯塔）是法布里病的首个口服治疗药物，以及庞比立瑅。

(cipaglucosidase alfa-atga) + Opfolda

（cipaglucosidase alfa-atga）+ Opfolda

(miglustat), a two-component therapy for Pompe disease. BioMarin also now has U.S. rights to DMX-200, a potential first-in-class investigational small molecule for the treatment of focal segmental glomerulosclerosis (FSGS), a rare and fatal kidney disease in Phase 3 development.

（米格鲁司他）是庞贝氏病的双组分疗法。BioMarin现在还拥有DMX-200的美国权利，DMX-200是一种潜在的首创小分子研究性药物，用于治疗局灶节段性肾小球硬化症（FSGS），这是一种罕见且致命的肾脏疾病，目前处于第三阶段开发中。

'The completion of the Amicus acquisition advances BioMarin's strategy to strengthen and diversify our growth profile while furthering our mission to deliver medicines for people living with rare diseases,' said Alexander Hardy, President and Chief Executive Officer of BioMarin. 'BioMarin's global scale, established commercial infrastructure, and advanced in‑house manufacturing capabilities build on Amicus' legacy and position us to bring Galafold and Pombiliti + Opfolda to more patients around the world.'.

“完成对Amicus的收购推进了BioMarin加强和多元化我们增长战略的目标，同时进一步履行我们为罕见病患者提供药物的使命，”BioMarin总裁兼首席执行官Alexander Hardy表示。“BioMarin的全球规模、成熟的商业基础设施以及先进的内部生产能力，建立在Amicus的传承之上，使我们能够将Galafold和Pombiliti + Opfolda带给世界各地更多的患者。”

About Galafold

关于Galafold

Galafold

(migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body's own dysfunctional enzyme so that it can clear the accumulation of disease substrate.

(migalastat) 123毫克胶囊是一种口服的α-半乳糖苷酶A（alpha-Gal A）药理伴侣，用于治疗携带可应对的半乳糖苷酶α基因（GLA）变异的成年法布里病患者。在这些患者中，Galafold通过稳定身体自身功能失调的酶，使其能够清除疾病底物的积累。

Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of people living with Fabry disease may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan..

全球范围内，Amicus Therapeutics 估计大约 35% 到 50% 的法布里病患者可能携带可治疗的 GLA 基因变异，但该范围内的可治疗率因地区而异。Galafold 已在全球 40 多个国家获得批准，包括美国、欧盟、英国和日本。

U.S. INDICATIONS AND USAGE

美国适应症和用法

Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable GLA variant based on in vitro assay data.

Galafold 适用于治疗经体外检测数据确认诊断为法布里病且具有适合的 GLA 变异的成人患者。

This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

该适应症是基于减少肾间质毛细血管细胞球三糖神经酰胺（KIC GL-3）底物而通过加速审批批准的。此适应症的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。

U.S. IMPORTANT SAFETY INFORMATION

美国重要安全信息

ADVERSE REACTIONS

不良反应

: The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.

Galafold最常见的不良反应（≥10%）包括头痛、鼻咽炎、尿路感染、恶心和发热。

USE IN SPECIFIC POPULATIONS

特定人群中的使用

: There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus. It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.

：关于孕妇使用Galafold的临床数据不足，无法评估该药物与重大出生缺陷和流产之间的相关风险。应告知女性该药物可能对胎儿造成的潜在风险。目前尚不清楚Galafold是否会出现在母乳中。因此，在考虑母乳喂养对婴儿发育和健康的益处时，还需权衡母亲对Galafold的临床需求以及Galafold或母亲的基础健康状况可能对哺乳婴儿造成的任何潜在不良影响。

Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis. The safety and effectiveness of Galafold have not been established in pediatric patients. To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or .

Galafold 不建议用于严重肾功能损害或需要透析的终末期肾病患者。Galafold 在儿科患者中的安全性和有效性尚未确定。如需报告疑似不良反应，请联系 Amicus Therapeutics，电话：1-877-4AMICUS，或 FDA，电话：1-800-FDA-1088。

www.fda.gov/medwatch

。

For additional information about Galafold, including the full U.S. Prescribing Information, please visit

有关Galafold的更多信息，包括完整的美国处方信息，请访问

https://www.amicusrx.com/pi/Galafold.pdf.

https://www.amicusrx.com/pi/Galafold.pdf

About Pombiliti + Opfolda

关于Pombiliti + Opfolda

Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that's designed to reduce loss of enzyme activity in the blood..

Pombiliti + Opfolda 是一种双组分疗法，包含cipaglucosidase alfa-atga（一种富含双M6P的重组人酸性α-葡萄糖苷酶），通过M6P受体促进高亲和力摄取，同时保留其转化为酶最活跃形式的能力；以及口服酶稳定剂miglustat，旨在减少血液中酶活性的损失。

U.S. INDICATIONS AND USAGE

美国适应症和用法

POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase (GAA) deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).

POMBILITI与OPFOLDA联合使用，适用于治疗体重≥40公斤且当前酶替代疗法（ERT）效果不佳的晚发型庞贝病（溶酶体酸性α-葡萄糖苷酶（GAA）缺乏症）成年患者。

SAFETY INFORMATION

安全信息

HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated.

超敏反应，包括过敏反应：应随时提供适当的医疗支持措施，包括心肺复苏设备。如果发生严重超敏反应，应立即停止使用POMBILITI，并启动适当的医疗处理。

INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion.

输注相关反应 (IAR)：如果发生严重 IAR，立即停止使用 POMBILITI 并启动适当的医疗处理。易感患者急性心肺功能衰竭的风险：易受液体容量超负荷影响的患者，或那些患有急性基础呼吸系统疾病或心脏或呼吸功能受损的患者，在 POMBILITI 输注期间可能会面临其心脏或呼吸状态严重恶化风险。

See the full U.S. Prescribing Information for complete Boxed Warning. CONTRAINDICATION:.

请参阅完整的美国处方信息以获取完整的黑框警告。禁忌症：。

POMBILITI in combination with Opfolda is contraindicated in pregnancy.

POMBILITI 与 Opfolda 联合使用时，孕妇禁用。

EMBRYO-FETAL TOXICITY:

胚胎-胎儿毒性：

May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose.

可能造成胚胎-胎儿伤害。告知有生殖潜力的女性对胎儿的潜在风险，并在治疗期间及最后一剂后至少60天内使用有效避孕措施。

Adverse Reactions:

不良反应：

Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.

最常见的不良反应（≥5%）包括头痛、腹泻、疲劳、恶心、腹痛和发热。

Please see U.S.

请参见美国。

full PRESCRIBING INFORMATION

完整的处方信息

, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) and

，包括加框警告，适用于POMBILITI（cipaglucosidase alfa-atga）和

full PRESCRIBING INFORMATION

完整的处方信息

for OPFOLDA (miglustat).

用于OPFOLDA（米格鲁司他）。

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 a portfolio of 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，专注于为患有基因定义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，拥有一系列商业治疗药物以及强大的临床和临床前管线。

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 .

通过采用独特的药物发现和开发方法，BioMarin 致力于通过研发对患者产生深远影响的开创性药物，充分释放基因科学的潜力。欲了解更多信息，请访问。

www.biomarin.com

。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements about, among other things, the business prospects of Amicus Therapeutics (Amicus) and BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the prospective benefits of the acquisition; expectations regarding Amicus' products, Galafold and Pombiliti + Opfolda; expectations regarding Amicus' product candidate, DMX-200, and its ongoing development; BioMarin's capital allocation strategy to leverage its financial strength to diversify its pipeline and add innovative new therapies for patients; BioMarin's plans for external innovation, including BioMarin's ability to execute additional transactions in future quarters; statements about BioMarin's future performance; and other statements that are not historical facts.

本新闻稿包含关于Amicus Therapeutics（Amicus）和BioMarin Pharmaceutical Inc.（BioMarin）业务前景的前瞻性声明，其中包括但不限于以下内容：收购的预期效益；对Amicus产品Galafold和Pombiliti + Opfolda的预期；对Amicus产品候选物DMX-200及其持续开发的预期；BioMarin利用其财务实力进行资本分配的战略以实现管线多样化并为患者增加创新疗法；BioMarin的外部创新计划，包括BioMarin在未来季度执行更多交易的能力；关于BioMarin未来表现的声明；以及其他非历史事实的声明。

Actual results could differ materially from those anticipated in these forward-looking statements. Except as required by law, each of BioMarin and Amicus assume no obligation to update these forward-looking statements, whether as a result of new information, future events or otherwise. These statements, which represent each of BioMarin's and Amicus' current expectations or beliefs concerning various future events that are subject to significant risks and uncertainties, may contain words such as 'may,' 'will,' 'would,' 'could,' 'expect,' 'anticipate,' 'intend,' 'plan,' 'believe,' 'estimate,' 'project,' 'seek,' 'should,' 'strategy,' 'future,' 'opportunity,' 'potential' or other similar words and expressions indicating future results..

实际结果可能与这些前瞻性陈述中预期的结果有重大差异。除非法律要求，BioMarin和Amicus均不承担更新这些前瞻性陈述的义务，无论是由于新信息、未来事件或其他原因。这些陈述代表了BioMarin和Amicus对受重大风险和不确定性影响的各种未来事件的当前预期或信念，可能包含诸如“可能”、“将”、“会”、“能够”、“预计”、“预期”、“打算”、“计划”、“相信”、“估计”、“预测”、“寻求”、“应该”、“策略”、“未来”、“机会”、“潜力”等词语或其他类似表达未来结果的措辞。

These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. Forward-looking statements reflect current beliefs and expectations; however, these statements involve inherent risks and uncertainties, including, without limitation, with respect to: the effects of the acquisition on Amicus' or BioMarin's stock price and/or Amicus' or BioMarin's operating results; unknown or inestimable liabilities; the development, launch and commercialization of products and product candidates; the parties' ability to realize the anticipated benefits of the acquisition, including the possibility that the expected benefits from the acquisition will not be realized or will not be realized within the expected time period and that BioMarin and Amicus will not be integrated successfully or that such integration may be more difficult, time-consuming or costly than expected; obtaining and maintaining adequate coverage and reimbursement for BioMarin's or Amicus' products; the time-consuming and uncertain regulatory approval process; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients, including with respect to current and planned future clinical trials; global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to BioMarin's or Amicus' business operations and financial results; the sufficiency of BioMarin's or Amicus' cash flows and capital resources; BioMarin's evaluation of the potential impact of the transaction on its financial results and financial guidance; Bi.

这些前瞻性陈述是预测性的，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。前瞻性陈述反映了当前的信念和期望；然而，这些陈述涉及固有的风险和不确定性，包括但不限于：收购对Amicus或BioMarin股价和/或Amicus或BioMarin经营结果的影响；未知或无法估量的负债；产品及候选产品的开发、上市和商业化；各方实现收购预期收益的能力，包括预期收益可能无法实现或无法在预期时间内实现的风险，以及BioMarin和Amicus可能无法成功整合或整合可能比预期更困难、更耗时或成本更高的风险；获得并维持对BioMarin或Amicus产品的充分覆盖和报销；耗时且不确定的监管审批流程；昂贵且耗时的医药产品开发过程以及临床成功的不确定性，包括与未能或延迟成功启动或完成临床试验及评估患者相关的风险，特别是针对当前和计划中的未来临床试验；全球经济、金融和医疗系统的中断以及当前和潜在未来对BioMarin或Amicus业务运营和财务结果的负面影响；BioMarin或Amicus现金流和资本资源的充足性；BioMarin对交易可能对其财务结果和财务指引影响的评估；Bi。

BioMarin

is a registered trademark of BioMarin Pharmaceutical Inc. or its affiliates.

是BioMarin制药公司或其附属公司的注册商标。

Contacts:

联系人：

Investors

投资者

Traci McCarty

特雷西·麦卡特里

BioMarin Pharmaceutical Inc.

BioMarin制药公司

(415) 455-7558