This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.

开始日期2023-11-10

申办/合作机构

Sanofi

NCT05843916

/ Completed临床3期

Phase III, Open-label, Switch Over Trial of the Efficacy and Safety of Agalsidase Beta Biosidus (AGA BETA BS) in Fabry Disease Patients Previously Stabilized With Fabrazyme®

BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and previously stabilized with Fabrazyme®.

开始日期2022-12-13

申办/合作机构

IQVIA RDS, Inc.

NCT05054387

/ Completed临床4期

A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replacement Therapy in Chinese Participants With Fabry Disease

This is a 54-week Phase 4, open label, single arm study to evaluate the safety and the efficacy of Fabrazyme (agalsidase beta) as enzyme replacement therapy (ERT) in Chinese participants with Fabry Disease.

开始日期2021-10-13

申办/合作机构

Genzyme Corp.

100 项与阿加糖酶β 相关的临床结果

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100 项与阿加糖酶β 相关的转化医学

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100 项与阿加糖酶β 相关的专利（医药）

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项与阿加糖酶β 相关的文献（医药）

2026-03-04·Expert Opinion On Drug Safety

A real-world pharmacovigilance analysis for agalsidase beta: findings from the FDA adverse event reporting database

Article

作者: Xie, Wen-Long ; Deng, Ai-Ping ; Li, Ju-Yi ; Li, Hou-Hong ; Li, Di

BACKGROUND:

Fabry disease (FD), an X-linked lysosomal disorder, is marked by a lack of alpha-galactosidase A (α-Gal A). Agalsidase beta, a recombinant form of α-Gal A, is fundamental to enzyme replacement therapy for FD but requires close monitoring for adverse events (AEs).

RESEARCH DESIGN AND METHODS:

This study retrospectively analyzed the Food and Drug Administration Adverse Event Reporting System (FAERS) database for agalsidase beta-related AEs. Disproportionality analysis was used for data analysis.

RESULTS:

A total of 7,611 AE reports for agalsidase beta were analyzed. The most common AEs included pyrexia, pain, chills, malaise, and nausea. Several system organ classes including Cardiac Disorders, General Disorders and Administration Site Conditions, and Vascular Disorders, showed positive signals. Subgroup analysis by gender revealed differences in AE reporting, with males exhibiting a higher reporting odds ratio for certain preferred terms such as Renal Transplant and Drug Specific Antibody Present.

CONCLUSION:

The FAERS database analysis of agalsidase beta AEs identified a significant number of cardiovascular, renal, and cerebrovascular system-related reports. While agalsidase beta is generally well-tolerated, the study underscores the necessity for gender-specific treatment approaches due to the higher incidence of certain AEs in males.

2025-11-01·Molecular Genetics & Genomic Medicine

Historical Control Analysis Demonstrates Greater Long‐Term Reduction in Plasma Globotriaosylceramide (Gb3) by Venglustat Compared With Placebo or Agalsidase Beta in Male Patients With Classic Fabry Disease

Article

作者: Ortiz, Alberto ; DasMahapatra, Pronabesh ; Liu, Shiguang ; Wilcox, William R. ; Deegan, Patrick ; Germain, Dominique P. ; Modur, Vijay

ABSTRACT:

Purpose:

To evaluate the disease biomarker response of venglustat in patients with Fabry disease (FD), utilizing data from a single‐arm phase 2 study of venglustat and a placebo‐controlled phase 3 study of agalsidase beta through historical control and case‐matched analyses.

Methods:

Eleven venglustat‐treated male patients with classic FD in the phase 2 study were matched with placebo‐ or agalsidase beta–treated patients from the phase 3 study based on propensity scores at baseline. Changes from baseline in plasma globotriaosylceramide (GL‐3 or Gb3) concentrations were analyzed at approximately 6–36 months.

Results:

Venglustat treatment resulted in greater significant reductions in plasma GL‐3 concentrations at 6 months from baseline vs. placebo (mean difference −2.56 μg/mL, p < 0.001), and at 24 and 36 months from baseline vs. agalsidase beta (mean difference −1.8 μg/mL, p < 0.05 and −2.35 μg/mL, p < 0.01, respectively). GL‐3 concentrations continued to decline with venglustat for up to 3 years without plateauing.

Conclusions:

Venglustat showed significantly greater reductions in plasma GL‐3 concentrations than placebo after 6 months and agalsidase beta after 24 and 36 months. These findings support the potential of long‐term venglustat treatment to reduce GL‐3 accumulation in patients with classic FD. Further studies are needed to confirm clinical benefit.

2025-07-01·NAUNYN-SCHMIEDEBERGS ARCHIVES OF PHARMACOLOGY

Efforts of the Pharmaceuticals and Medical Devices Agency of Japanese regulatory agency in supporting biosimilar development and disseminate information

Article

作者: Saino, Yuka ; Hariu, Aya ; Shinohara, Kayo ; Kuribayashi, Ryosuke

Information on biosimilars was rarely disseminated on the Pharmaceuticals and Medical Devices Agency (PMDA) website of the Japanese Regulatory Agency until the fiscal year (FY) 2022. Therefore, the PMDA website for biosimilars was created in FY 2023. This study confirmed the PMDA website for biosimilars and surveyed information about the approval fiscal year, brand name, indications at the initial approval, type of biopharmaceuticals, and disease area at the initial approval based on review reports and the "List of Approved Products." The PMDA website for biosimilars provides information on the definition of biosimilar, approved biosimilar products, related guidelines and notifications, learning videos, presentation material, and publication articles. As of March 2024, 35 biosimilars were approved in Japan based on the following 18 active pharmaceutical ingredients: somatropin, epoetin-alfa, filgrastim, infliximab, insulin glargine, rituximab, etanercept, trastuzumab, agalsidase beta, bevacizumab, teriparatide, darbepoetin-alfa, insulin lispro, adalimumab, insulin aspart, ranibizumab, pegfilgrastim, and ustekinumab. The disease area comprised nine patterns: rheumatology, oncology, hematology, gastroenterology, dermatology, endocrinology, ophthalmology, bone, and inborn errors represented in numbers and percentages for each pattern: 12 (23.1%), 9 (17.3%), 8 (15.4%), 7 (13.5%), 7 (13.5%), 5 (9.6%), 2 (3.8%), 1 (1.9%), and 1 (1.9%), respectively. This website provides various information regarding biosimilars in Japan. It will be more important to effort the understanding and education for the healthcare providers and patients on biosimilar regulations. Moreover, information should be disseminated through the PMDA website of biosimilars to accelerate and ensure transparency regarding regulatory approvals and biosimilar regulations.

项与阿加糖酶β 相关的新闻（医药）

2026-03-08

·生命科学领域猎头candice

本土药企多点开花，慢病与罕见病赛道迎利好

! 点击蓝字关注我们本土药企多点开花，慢病与罕见病赛道迎利好创新始终是医药行业不变的主旋律，而本周的多项动态，恰好为 2026 年的春天写下了一个精彩的注脚。刚刚过去的一周，中国医药行业迎来多项实打实的重要进展。复星医药斯鲁利单抗冲刺肝癌新适应症、石药集团全球首创新药获批临床、信达生物深耕慢病赛道再进一步、阿斯利康经典药跨界拓展适应症、赛诺菲罕见病药物成功纳入医保，五条重磅动态均来自企业官方公告与国家药监、医保部门公开信息，背后折射出当前中国医药市场管线深耕、慢病拓展、罕见病可及三大核心发展方向。透过这些真实落地的行业动态，我们既能看清头部药企的战略重心，更能窥见中国医药产业高质量发展的深层脉络。 1 事件速览：五条真实动态，踏准行业发展节奏复星医药：控股子公司复宏汉霖自主研发的斯鲁利单抗新适应症上市申请获国家药监局正式受理，拟用于治疗晚期肝细胞癌，这也是该产品在国内申报的第 5 项适应症。目前斯鲁利单抗已在全球多个国家和地区获批，覆盖鳞状非小细胞肺癌、广泛期小细胞肺癌、食管鳞状细胞癌等多个实体瘤大癌种，临床价值与商业化能力已获多方验证。石药集团：创新药 SYHX1901 片获国家药监局临床试验批准，拟用于治疗晚期实体瘤。该产品为全球首创的靶向 ERK1/2 的小分子抑制剂，是石药集团小分子新药筛选及研发平台的核心在研项目，瞄准 MAPK 信号通路异常激活的实体瘤临床未满足需求。信达生物：与礼来联合开发的 PCSK9 抑制剂托莱西单抗注射液，新增适应症申请获国家药监局受理，拟用于治疗原发性高胆固醇血症和混合型血脂异常。作为国内首个自主研发获批的 PCSK9 抑制剂，此次适应症拓展标志着信达生物在心血管代谢领域的布局持续深化，进一步覆盖更广阔的慢病患者人群。阿斯利康：糖尿病药物达格列净片新适应症获国家药监局批准，用于治疗射血分数保留的心力衰竭成人患者。这是达格列净在中国获批的又一项重要心血管适应症，此前其已获批 2 型糖尿病、射血分数降低的心力衰竭、慢性肾脏病适应症，进一步巩固了其作为 SGLT2 抑制剂类药物的 “跨界王者” 地位。 2 企业纵深：从单点突破到管线协同，真实布局见真章复星医药：PD-1 红海突围，靠差异化布局站稳脚跟在 PD-1 赛道竞争日趋白热化的背景下，斯鲁利单抗能持续拓展适应症，核心在于精准布局大癌种 + 打造联合疗法矩阵的差异化策略。截至目前，该产品已在国内获批 4 项适应症，此次冲刺肝癌适应症更是瞄准了国内高发的实体瘤领域。同时复宏汉霖以斯鲁利单抗为核心，联动自有管线构筑组合疗法，其近期获批临床的 HLX43（PD-L1 ADC）联合 HLX07（EGFR 单抗）及斯鲁利单抗的联合疗法，正成为提升产品竞争力的重要抓手，也让这款核心产品的生命周期价值持续提升。石药集团：技术平台驱动源头创新，原创实力获全球认可 SYHX1901 的获批临床，并非偶然突破，而是石药集团多年布局源头创新的必然结果。近年来石药集团已搭建起涵盖小分子、ADC、siRNA、mRNA 等前沿领域的八大创新技术平台，形成了从早期研发到商业化落地的完整创新体系。2026 年初，石药集团与阿斯利康达成总金额高达185 亿美元的长效多肽 / 代谢管线战略授权合作，这一真实落地的重磅交易，更是让市场看到了其技术平台的国际竞争力，印证了本土药企在原创创新领域的硬实力。信达生物：从肿瘤到慢病，打造均衡管线穿越行业周期托莱西单抗 2023 年获批上市，一举打破了进口产品在国内 PCSK9 抑制剂领域的垄断，此次新适应症申报，进一步扩大了其在降脂领域的覆盖范围。而信达生物在代谢疾病领域的布局远不止于此，其 GCG/GLP-1 双受体激动剂玛仕度肽已在减重和降糖两大适应症上进入上市倒计时。从深耕肿瘤赛道到布局心血管代谢等慢病领域，信达生物正构建肿瘤 + 慢病双轮驱动的产品组合，以均衡的管线布局抵御赛道波动，这也是其穿越行业周期的核心战略。阿斯利康：大品种生命周期管理，教科书级的价值挖掘达格列净堪称医药行业大品种生命周期管理的标杆。从最初的 2 型糖尿病治疗，到逐步拓展至心力衰竭、慢性肾脏病等领域，其适应症版图的持续扩张，背后是基于大量循证医学证据的临床价值挖掘。根据 DrugPatentWatch2026 年 2 月最新数据，达格列净 2022 年全球销售额约 45 亿美元，预计到 2028 年有望超过 170 亿美元。在中国市场，随着新适应症的不断获批和医保覆盖的持续扩大，这款经典药物的市场增长空间依然可观。赛诺菲：坚守罕见病赛道，以长期主义兑现社会价值戈谢病作为超罕见病，患者人群极小但治疗需求极为迫切，阿加糖酶 β 纳入医保，让众多患者实现了 “用得起好药” 的愿望。赛诺菲在中国罕见病领域布局多年，除阿加糖酶 β 外，还拥有注射用阿糖苷酶 α（庞贝病）、注射用拉罗尼酶浓溶液（黏多糖贮积症）等多款获批产品，形成了完善的罕见病产品矩阵。此次阿加糖酶 β 纳入医保，不仅体现了国家对罕见病患者的关怀，也为跨国药企持续深耕中国罕见病市场注入了信心，更印证了 “长期主义” 在罕见病赛道的价值。 3 趋势洞察：三大真实方向，定调 2026 医药行业发展 1. 慢病治疗的边界正在模糊，靶点机制成为核心逻辑达格列净从降糖药跨界成为心衰、肾病治疗药物，托莱西单抗持续拓展降脂适应症，这一系列真实动态背后，是慢病治疗逻辑的根本转变。过去以 “降糖”“降脂” 为核心的单一治疗思路，正逐步向以靶点机制为基础、覆盖多系统获益的综合治疗模式转变。一款药物不再局限于单一疾病领域，而是基于靶点的生理作用，挖掘其在多个系统的临床价值，这也对药企的临床设计、循证医学研究能力提出了更高要求。 2. 罕见病用药进入医保快车道，可及性持续提升赛诺菲阿加糖酶 β 纳入医保，是近年来国家医保目录向罕见病用药倾斜的又一真实案例。从 2022 年开始，罕见病用药就已成为医保目录调整的重点考虑对象，截至 2025 年底，已有超 50 种罕见病用药被纳入国家医保目录。随着多层次医疗保障体系的不断完善，医保、商保、医疗救助的协同作用持续发挥，罕见病患者的用药可及性正在稳步提升，这也为布局罕见病领域的企业提供了更清晰的市场预期，推动更多企业投入罕见病药物研发。 3. 本土药企的 “技术出海” 成为常态，原创实力获全球认可石药集团与阿斯利康 185 亿美元的授权合作，并非孤例。2026 年开年以来，瑞博生物 44 亿美元 siRNA 药物出海、信达生物与礼来 85 亿美元全球合作等重磅交易接连落地，这些真实的行业动态印证了一个事实：中国药企的研发能力已实现从 “仿制” 到 “模仿创新” 再到 “原始创新” 的质变，以技术平台为核心的创新资产，正获得全球同行的广泛认可。未来，本土药企的技术授权、全球联合开发将成为行业常态，“技术出海” 也将成为中国医药创新的新名片。结语这一周的五条医药行业动态，均源于企业官方公告、国家药监和医保部门的公开信息，无虚构内容，恰如一幅真实的行业发展拼图：既有 PD-1、PCSK9 等成熟靶点的持续深耕，也有 ERK1/2 这类全新靶点的勇敢探索；既有慢病大品种的价值深度挖掘，也有罕见病药物的政策落地突围。对于药企而言，管线的厚度决定生存的底线，创新的浓度决定发展的高度，从单点突破到管线协同，从本土布局到全球拓展，真实的研发实力和商业化能力才是核心竞争力。对于行业而言，政策引导与技术突破的双轮驱动，正在加速中国医药市场的高质量转型，本土创新与国际合作的深度融合，让中国医药产业逐步站上全球舞台。而对于最核心的患者群体，每一项新适应症的获批、每一次医保目录的更新、每一款创新药的落地，都是离 “用得上、用得起好药” 更近的一步。医药创新的终极意义，终究要落在患者身上。这五条真实发生的行业动态，正是这份意义最生动的注脚。参考资料：国家药品监督管理局、药品审评中心官网公开审评 / 获批信息复星医药、石药集团、信达生物、阿斯利康中国 2026 年 3 月官方公告国家医保局 2025 年《国家基本医疗保险、工伤保险和生育保险药品目录》 DrugPatentWatch 2026 年 2 月达格列净销售额预测报告上海证券报、中国医药报 2026 年一季度医药行业权威报道点击蓝字关注我们

申请上市临床申请引进/卖出

2026-02-02

·摩熵医药

全球罕见病药千亿赛道巅峰对决！赛诺菲、BI、BMS谁更胜一筹？

注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需要得到授权。在罕见病治疗的浩瀚星图中，每一份深耕与突破都意味着无数生命的转机。摩熵咨询撰写的《2025全球罕见病行业发展报告：政策演进、市场趋势与领先企业布局》不仅是一份行业概览，更是洞悉未来的战略蓝图。它系统性地剖析了全球罕见病政策演进、市场趋势与领先企业的深度布局，为读者勾勒出一幅充满机遇与挑战的产业全景。本文作为报告核心内容的延伸，将继续探访在罕见病领域中以差异化战略树立标杆的制药巨头：勃林格殷格翰、赛诺菲与百时美施贵宝。 01 勃林格殷格翰：纤维化疾病领域的深度开拓者勃林格殷格翰在罕见病领域展现出特定疾病赛道的深度专注，凭借其在纤维化相关罕见病领域的里程碑式治疗药物，建立了坚实的市场地位。 2024年，勃林格殷格翰的营收达到268亿欧元，其在罕见病领域长期聚焦于高未满足医疗需求的遗传性和进展性疾病，尤其在特发性肺纤维化（IPF）等罕见呼吸系统疾病方面建立了具有里程碑意义的成果，代表性药物尼达尼布显著改变了疾病治疗格局，推动了抗纤维化治疗从无到有的发展。核心药物：尼达尼布（维加特/Ofev）尼达尼布（Nintedanib），商品名维加特®/Ofev®，是一种小分子多靶点酪氨酸激酶抑制剂（TKI）。它是全球首个获批用于治疗特发性肺纤维化（IPF）的靶向药，具有抗纤维化和抗炎活性。 2024年勃林格殷格翰在人用药品业务业绩同比增长7.0%，达219亿欧元，主要得益于恩格列净和尼达尼布的强势表现。其中用于治疗特发性肺纤维化和特定类型的纤维化性间质性肺疾病的药物维加特®净销售额同比增长8.9%，达38亿欧元。图片来源：摩熵咨询《2025全球罕见病行业发展报告》尼达尼布在勃林格殷格翰2024年总体营收持续增长的前提下，其销售额占总体销售额比例保持不变，显示出尼达尼布在勃林格殷格翰所有产品中的重要与潜力。图片来源：摩熵咨询《2025全球罕见病行业发展报告》研发管线：前沿探索与早期布局创新不止于已上市产品，勃林格殷格翰的在研管线同样延续了“细分领域深耕”的战略特色，聚焦肾脏疾病（FSGS）、肺部纤维化疾病（IPF）及自身免疫病（系统性硬化症）等高需求方向。摩熵医药数据库显示，目前其罕见病相关项目均处于临床Ⅱ期阶段，以早期前沿探索为核心方向。勃林格殷格翰在研管线清单数据来源：摩熵医药数据库值得关注的是，管线所采用的TRPC6、SIRPα、溶血磷脂酶、PDE4B等药物作用机制，均突破了经典常见靶点的局限，彰显了企业在罕见病治疗领域对新生物学通路和创新靶点的勇敢探索，为未来治疗方案的迭代升级奠定了基础。 02 赛诺菲：系统化疗法的长期领导者赛诺菲是全球罕见病领域的长期领导者与系统化疗法先驱，凭借其多款突破性酶替代疗法药物，在罕见遗传代谢病、罕见血凝病等领域构建了全面且可持续的全球化治疗布局。作为法国跨国制药巨头，赛诺菲2024年营收410.8亿欧元。赛诺菲是全球罕见病领域的长期领导者之一，依托其罕见病事业部（原Genzyme），在罕见遗传代谢病、罕见血液病、神经肌肉疾病及免疫性罕见病等领域建立了系统性布局，率先实现多款突破性疗法的全球可及化，如针对戈谢病、庞贝病、法布雷病等的酶替代疗法以及治疗多发性硬化、脊髓性肌萎缩症等疾病的创新药物。赛诺菲罕见病领域畅销药物清单图片来源：摩熵咨询《2025全球罕见病行业发展报告》赛诺菲罕见病领域畅销药物2024年销售额图片来源：摩熵咨询《2025全球罕见病行业发展报告》在赛诺菲的罕见病产品矩阵中，法布赞®（针对法布雷病）是唯一在2024年销售收入超过10亿欧元的品种。整体而言，除美而赞®销售收入有所下滑外，其余罕见病品种均实现了稳步增长，显示出产品线良好的生命力与市场接纳度。研发管线：多元化与深度推进赛诺菲在罕见病领域的在研管线展现出多元化，且聚焦遗传代谢病、自身免疫病及血液系统疾病方向的深度布局，目前已从早期研发向中后期加速推进，多项靶向RNAi、酶替代及小分子抑制剂药物已进入注册或Ⅲ期临床阶段。图片来源：摩熵咨询《2025全球罕见病行业发展报告》摩熵医药数据库显示，目前赛诺菲有93个临床项目，其中有14个临床项目聚焦罕见病领域，覆盖遗传代谢病、自身免疫病、血液系统疾病等多个高未满足需求方向。赛诺菲罕见病药物领域在研管线清单数据来源：摩熵医药数据库从研发阶段看，其罕见病项目已从早期研发向中后期加速推进，多项基于RNAi、酶替代及小分子抑制剂技术的药物已进入注册或Ⅲ期临床阶段，管线成熟度较高。 03 百时美施贵宝：罕见病领域的创新合作者与其他企业不同，百时美施贵宝以“创新合作者”的定位，在罕见病领域走出了一条独具特色的发展路径——依托已上市重磅药物筑牢基础，通过战略合纵连横拓展基因编辑与细胞疗法等前沿管线，构建了以临床需求为导向、合作驱动为核心的业务模式。 2024年，百时美施贵宝实现营收483亿美元。其在罕见病领域持续投入研发与商业化力量，通过创新疗法改善多种高未满足需求的少见病患者预后，其畅销罕见病药物涵盖了罕见血液病、罕见肿瘤等多个方向。畅销罕见病药物：罗特西普、奥普杜拉格罗特西普（Reblozyl Luspatercept）是一种用于治疗β地中海贫血和骨髓增生异常综合征所致贫血的药物罗特西普在作为FDA认可的罕见病用药，在2024年百时美施贵宝的销售额榜单上排名第七，展现了良好的市场潜力。奥普杜拉格（Opdualag）是由百时美施贵宝（BMS）研发的全球首款抗LAG-3/PD-1双免疫联合复方制剂，用于治疗12岁及以上不可切除或转移性黑色素瘤患者。该药物由PD-1抑制剂纳武利尤单抗（nivolumab）与新型抗LAG-3抗体relatlimab组成，通过同时阻断LAG-3和PD-1两个免疫检查点，增强T细胞活性以抑制肿瘤生长。战略布局与合作模式百时美施贵宝的战略智慧体现在其积极的外部合作上。2023年，公司与SystImmune达成BL-B01D1全球独家合作协议，总潜在价值高达84亿美元，这充分展示了其通过许可合作方式快速扩展高未满足领域研发管线的决心。此外，与Prime Medicine建立战略合作，推动基因编辑技术与T细胞疗法的开发，Prime Medicine获得约1.1亿美元前期付款及数十亿美元级里程碑付款潜力，为公司在复杂遗传性疾病和免疫细胞治疗方向的布局奠定基础，尤其中长期可能涉及如囊性纤维化（CF）等罕见病相关适应症的潜在解决方案。罗特西普和奥普杜拉格2022-2024年销售额图片来源：摩熵咨询《2025全球罕见病行业发展报告》在研管线方面，摩熵医药数据库显示，截至2025年12月17日，百时美施贵宝正在开发的化合物有48个，涉及到的疾病领域超过40个，其中涉及到的罕见病包括了黑色素瘤、镰刀型贫血、多发性硬化、阿尔茨海默病、重症肌无力等。百时美施贵宝在研管线清单图片来源：摩熵咨询《2025全球罕见病行业发展报告》当前，百时美施贵宝的在研药物管线多个项目已进入临床后期或重要临床验证阶段，同时持续推进细胞疗法、蛋白降解、小分子靶向和免疫调节等多种创新治疗策略。结语从勃林格殷格翰的垂直深耕，到赛诺菲的生态构建，再到百时美施贵宝的开放会师，这三家企业以截然不同的战略路径，共同拓展着罕见病治疗的疆界。他们的实践表明，在罕见病这场需要毅力与智慧的征程中，无论是深度聚焦、系统布局还是生态合作，都能抵达拯救生命的彼岸。更多产业洞察与数据解码，详见摩熵咨询完整版行业报告。摩熵咨询深度报告 2025全球罕见病行业发展报告完整版领取方式识别下方二维码领取 END 本文为原创文章，转载请留言获取授权近期热门资源获取中国临床试验趋势与国际多中心临床展望-202505 2024年医药企业综合实力排行榜-202505 中国带状疱疹疫苗行业分析报告-202505 2023H2-2024H1中国药品分析报告-202504 2024年中国1类新药靶点白皮书-202503 中国AI医疗健康企业创新发展百强榜单-202502 2024年FDA批准上市的新药分析报告-202501 小分子化药白皮书（上）-202501 2024年中国医疗健康投融资全景洞察报告-202501 2024年医保谈判及市场分析报告-202501 近期更多摩熵咨询热门报告，识别下方二维码领取联系我们，体验摩熵医药更多专业服务会议合作园区服务数据库咨询定制服务媒体合作点击阅读原文，申请摩熵医药企业版免费试用！

申请上市临床2期优先审批引进/卖出财报

2026-02-02

·FierceBiotech

Sanofi\'s troubled GCS inhibitor fails phase 3 Fabry trial but scores in Gaucher disease

Venglustat was once considered as a potential \"pipeline in a pill.\"\n Sanofi’s pipeline features a number of once-hyped candidates that are struggling to deliver in the clinic, and the latest mixed phase 3 data from venglustat continue this trend.The French Big Pharma was studying venglustat, an oral, brain-penetrant glucosylceramide synthase (GCS) inhibitor, in a pair of late-stage studies of rare lysosomal storage disorders. One of these trials, called Peridot, assessed the pill in 122 patients with Fabry disease, while the Leap2Mono trial focused on 43 patients with type 3 Gaucher disease (GD3).The Peridot study missed its primary endpoint of demonstrating superiority in patient-reported improvement of symptoms—namely neuropathic pain in upper and lower extremities, as well as abdominal pain—compared to a placebo cohort. Sanofi explained this failure by pointing to a “reduction in neuropathic and abdominal pain … observed in both study arms.”It was better news in the Leap2Mono study, where the 21 patients who received venglustat saw statistically significant improvements in neurological symptoms at Week 52 as measured by scales for ataxia and for neuropsychological status. The improvement between the venglustat cohort and the 22 patients who received enzyme replacement therapy (ERT) worked out as a p-value of 0.007, Sanofi explained in its Feb. 2 release.When it came to non-neurological secondary endpoints like changes in spleen volume, liver volume and hemoglobin level, venglustat “performed as well as ERT,” according to the pharma.Venglustat was well tolerated, Sanofi said. The most common adverse events were headache, nausea, spleen enlargement and diarrhea—although reports of headache were higher in the ERT cohort.Based on the Leap2Mono data, Sanofi said it will seek regulatory approval for venglustat in GD3. The company already markets the ERT Cerezyme and the oral glucosylceramide synthase inhibitor Cerdelga for the disease.The failure of the Peridot study doesn’t mark the end of Sanofi’s Fabry ambitions, either. As well as its approved Fabry ERT Fabrazyme, Sanofi is still running a phase 3 study of venglustat on left cardiac ventricular mass index in patients with the disease.“These findings underscore Sanofi\'s commitment to rare disease research and the promise we aim to deliver for people living with these conditions,” Sanofi’s head of R&D Houman Ashrafian, Ph.D., said in this morning’s release.“What excites us most is the potential to address critical unmet medical needs,” Ashrafian added. “A daily pill could make a serious difference for Gaucher patients facing neurological challenges.”This morning’s phase 3 readouts continued a run of mixed data for venglustat, which had at one point been hailed as one of Sanofi\'s top prospects and a potential “pipeline in a pill” contender. Those ambitions were blunted as far back as 2021, when venglustat failed midstage studies in both Parkinson’s disease and autosomal dominant polycystic kidney disease. By 2024, venglustat’s options were narrowed down to just Fabry disease and GD3 after Sanofi gave up on GM2 gangliosidosis as an indication “based on the absence of positive trends on clinical endpoints.”While Sanofi remains hopeful of getting venglustat approved for GD3, the Fabry failure this morning also continues a longer-term trend of the drugmaker’s pipeline struggling to achieve its potential. The company’s R&D chief admitted to Fierce last year that clinical setbacks for its anti-OX40L-ligand antibody amlitelimab, its oral TNF inhibitor balinatunfib and its Regeneron-partnered IL-33 candidate itepekimab had resulted in a “mixed” 2025.On an earnings call with journalists last week, Sanofi CEO Paul Hudson directly acknowledged that it had been a “bumpy ride” for R&D.“I\'m well aware of the headlines—we had some challenges,” the CEO said at the time.

$Sanofi\'s troubled GCS inhibitor fails phase 3 Fabry trial but scores in Gaucher disease$

临床3期临床结果上市批准

100 项与阿加糖酶β 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D03228	阿加糖酶β	A16AB04	DB00103

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
法布里病	欧盟	Sanofi	2001-08-03
法布里病	冰岛	Sanofi	2001-08-03
法布里病	列支敦士登	Sanofi	2001-08-03
法布里病	挪威	Sanofi	2001-08-03

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
终末期肾脏病	临床2期	美国	Genzyme Corp.	2002-12-01

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分期

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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


NCT05843916 (SMILE, CTgov)	临床3期	法布里病	20	AGA BETA BS+Fabrazyme (Fabrazyme Then AGA BETA BS (Intention to Treat Population))	蓋壓積蓋鹹鏇網壓衊蓋(艱構膚廠膚艱觸憲鹹鹹) = 積壓蓋餘選築積繭壓壓製壓衊膚淵鹹繭衊範醖 (鑰憲顧壓憲選鏇鹹範窪, 3.10) 更多	-	2026-02-18
NCT05843916 (SMILE, CTgov)	临床3期	法布里病	20	AGA BETA BS+Fabrazyme (Fabrazyme Then AGA BETA BS (Per Protocol Population))	蓋壓積蓋鹹鏇網壓衊蓋(艱構膚廠膚艱觸憲鹹鹹) = 衊製遞繭鹽範憲窪艱願製壓衊膚淵鹹繭衊範醖 (鑰憲顧壓憲選鏇鹹範窪, 3.15) 更多	-	2026-02-18
NCT06019728 (SHORTEN, CTgov)	临床4期	法布里病	8	Fabrazyme	襯觸繭範顧蓋範範積繭(糧鹽顧襯顧艱鏇壓鏇鑰) = 網膚觸夢壓網製築製顧醖選遞壓積夢選襯網齋 (選願襯鏇築鏇衊艱觸蓋, 鏇鬱鹽鹽遞觸築選鹽淵 ~ 齋齋鏇糧艱築顧願選獵) 更多	-	2025-10-21
NCT06019728 (ASN2024)	临床4期	法布里病	4	Agalsidase beta (Fabry disease)	繭艱鏇壓艱淵鹹齋觸選(鏇獵餘蓋鹽窪繭製鹽糧) = 鬱製築範製窪願網積憲鏇觸鑰築積選齋鹽蓋蓋 (鬱範廠鹽獵夢鹹選憲獵 )	积极	2024-10-26
NCT00230607 (CTgov)	临床4期	法布里病	7	agalsidase beta	繭鬱淵獵醖遞範構廠繭(簾窪遞齋鑰衊廠範顧襯) = 鏇築衊憲積襯獵淵觸願獵願膚襯鬱衊蓋糧鹹製 (構鹹襯網醖構齋艱積糧, 願齋顧鬱遞餘鬱積繭範 ~ 艱鑰獵壓鏇製獵顧鏇積) 更多	-	2024-04-10
NCT00233870 (Pubmed \| Orphanet J Rare Dis)	N/A	法布里病	-	Agalsidase beta	構餘憲衊鑰壓壓淵網繭(窪糧餘範壓淵顧鑰觸製) = 鹽夢築齋餘憲壓齋觸願獵鏇選簾壓繭憲鬱鏇鏇 (製夢遞壓鹹鹹淵夢構獵 ) 更多	-	2023-07-24
Pubmed 人工标引	临床2期	法布里病	10	Agalsidase Beta	選蓋齋壓衊窪醖鬱襯鑰(壓鑰網淵製艱窪繭積艱) = decreased 4.01 ± 1.29 μg/mL 遞蓋願獵鏇遞餘繭觸廠 (願鬱鑰艱淵網鏇鏇網餘 ) 更多	积极	2022-09-16
ASN2021	临床2期	法布里病 globotriaosylceramide (GL3)	7	Agalsidase beta	獵鏇糧廠壓蓋鑰餘鬱窪(壓鹽範顧築築醖網衊構) = improved in all patients after ERT from that evaluated before ERT 壓構艱夢鏇顧艱選鬱鹹 (繭憲顧遞糧齋蓋淵窪觸 ) 更多	积极	2021-10-27
NCT00701415 (FIELD, CTgov)	临床3期	法布里病	31	Agalsidase beta (Fabrazyme 0.5 mg/kg)	鏇蓋顧蓋醖淵憲遞選製 = 鹹願鑰範簾膚襯網鬱獵遞構繭鏇繭窪鬱壓鹹範 (願廠鏇構蓋餘顧蓋鏇膚, 糧壓齋築廠簾憲艱範餘 ~ 齋艱齋遞蓋願製構顧遞) 更多	-	2016-06-29
NCT00701415 (FIELD, CTgov)	临床3期	法布里病	31	Agalsidase beta (Fabrazyme 1.0 mg/kg)	鏇蓋顧蓋醖淵憲遞選製 = 簾製膚艱積齋網齋鬱鹽遞構繭鏇繭窪鬱壓鹹範 (願廠鏇構蓋餘顧蓋鏇膚, 憲蓋顧衊膚蓋醖選積顧 ~ 糧蓋製衊糧廠淵顧糧艱) 更多	-	2016-06-29
NCT00074971 \| NCT00196742 (Pubmed \| Hum Genet \| J Med Genet) 人工标引	临床3期	法布里病	52	agalsidase beta	襯齋築顧鹹鑰鑰鏇蓋糧(淵蓋醖繭壓簾襯齋遞餘) = 簾衊鏇積鹹夢繭廠選憲獵餘鬱築築遞艱遞憲衊 (鬱醖夢鏇遞網廠簾襯選 ) 更多	积极	2015-05-01
NCT00140621 (CTgov)	临床4期	法布里病	6	Agalsidase beta	憲鑰繭積鬱廠鑰選廠繭(網蓋艱艱憲窪願糧醖製) = 艱齋願顧鹹築繭願製淵鏇夢範願鹽憲憲鑰願製 (糧夢觸窪構廠餘糧壓鏇, 積築膚積繭網鏇簾蓋糧 ~ 製窪艱繭繭鏇窪顧鏇築) 更多	-	2015-04-15