A Multicenter, Open-Label, Single-Arm, Two-Cohort, Phase 3, Clinical Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Eliglustat Sublingual Film in Patients with Gaucher Disease Type 1. - NIL

开始日期2024-09-02

申办/合作机构

Kashiv Biosciences Pvt Ltd.

NCT06558773

/ Not yet recruiting临床2期IIT

GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor and/or Regorafenib for Patients With Advanced/Metastatic pMMR/MSS Colorectal Cancer:an Open-Label, Randomized,Phase II Study

In this single-center,open-label, randomized, phase II study, the efficacy and feasibility of GSL synthetase inhibitor in combination with immune checkpoint inhibitor and/or regorafenib therapeutic regimen will be evaluated in patients with advanced/metastatic proficient mismatch repair/microsatellite stable (pMMR/MSS) colorectal cancer (CRC).In this clinical trial, a total of 120 eligible patients were stratified randomly (with/without liver metastases) assigned to the 3 arms in a 1:1:1 ratio: experimental group-arm A(Eliglustat+Immune checkpoint inhibitor+Regorafenib),experimental group-arm B(Eliglustat+Immune checkpoint inhibitor) and comparator group-arm C(Regorafenib+Immune checkpoint inhibitor).It aims to: 1).assess the antitumor effects of GSL synthetase inhibitor in combination with immune checkpoint inhibitor and/or regorafenib;2).evaluate the immunological or clinical predictive biomarkers for efficacy and toxicity; 3).detect the transformation of tumor microenvironment (TME) and dynamic changes of immune cells in peripheral blood after the treatment with GSL synthetase inhibitor in combination with immune checkpoint inhibitor and/or regorafenib.

开始日期2024-09-01

申办/合作机构

中国人民解放军总医院

NCT06523517

/ Not yet recruiting临床2期IIT

A Single-center, Single-arm, Prospective Clinical Study to Evaluate the Efficacy and Safety of Eliglustat in Chinese Pediatric Patients (≥12 to <18 Years Old) With Gaucher Disease Type 1 and Type 3

Primary Objective:
Evaluate the efficacy and safety of eliglustat in Chinese pediatric patients (≥12 to <18 years old) with Gaucher disease type 1 and type 3.
Secondary Objective:
Evaluate the quality of life in Chinese pediatric patients (≥12 to <18 years old) with Gaucher disease type 1 and type 3 treated with eliglustat.

开始日期2024-08-01

申办/合作机构

北京协和医院

100 项与依鲁司他相关的临床结果

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100 项与依鲁司他相关的转化医学

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100 项与依鲁司他相关的专利（医药）

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195

项与依鲁司他相关的文献（医药）

2025-06-01·PEDIATRIC NEPHROLOGY

Model-informed repurposing of eliglustat for treatment and prophylaxis of Shiga toxin-producing Escherichia coli hemolytic-uremic syndrome (STEC-HUS) in children

Article

作者: Wolthuis, David F G J ; van de Kar, Nicole C A J ; Kartha, Reena V ; Wijnsma, Kioa ; Ter Avest, Mendy ; Freriksen, Jolien J M ; de Wildt, Saskia N ; Ter Heine, Rob

Abstract:

Background:

Shiga toxin-producing Escherichia coli hemolytic-uremic syndrome (STEC-HUS) is a severe illness predominantly affecting young children, with limited treatment options beyond supportive care. Eliglustat, approved for Gaucher disease, shows potential in reducing Shiga toxin binding to target glomerular endothelial cells in vitro, prompting interest as a treatment for STEC-HUS. However, it remains unknown what dose is likely to be effective and safe for treatment of STEC-HUS in the pediatric population. We hypothesize that effective and safe levels of eliglustat can be reached in children.

Methods:

We identified pharmacokinetic targets of efficacy for treatment and prophylaxis of STEC-HUS based on a preclinical model and human cardiac safety data. Then, we developed oral and intravenous dosing regimens using population pharmacokinetic (popPK) simulations based on an existing model enriched to allow extrapolation to a simulated virtual pediatric population. These dosing regimens were then confirmed using a verified physiologically based pharmacokinetic (PBPK) model.

Results:

We simulated, using popPK data, oral and intravenous dosing regimens resulting in adequate target exposure in > 90% of all patients, with minimal expected risk for cardiotoxicity. Confirmation of these dosing regimens with PBPK modeling resulted in very similar exposure, with lower interindividual variability and minimal toxicity potential.

Conclusions:

Based on pharmacokinetic modeling, we developed oral and intravenous eliglustat dosing regimens that are likely safe and effective for treatment of STEC-HUS and prophylaxis in case of outbreaks of STEC infections. Clinical evaluation of these dosing regimens in children suspected of or diagnosed with STEC-HUS is required and should include assessment of pharmacokinetics, efficacy, and safety (e.g., ECG monitoring).

Graphical abstract:

2025-05-01·MOLECULAR GENETICS AND METABOLISM

Challenges in Gaucher disease: Perspectives from an expert panel

Review

作者: Prakalapakorn, S Grace ; Weinreb, Neal J ; Tuason, Dominick A ; Grabowski, Gregory A ; Rosenbloom, Barry E ; Alcalay, Roy N ; Kishnani, Priya S

This focused review concentrates on eight topics of high importance for Gaucher disease (GD) clinicians and researchers: 1) The consideration of GD as distinct types rather than a spectrum. A review of the literature clearly supports the view that there are distinct types of GD. Type 1 is characterized by the absence of primary neuronopathic involvement, while types 2 and 3 are characterized by progressive primary neuronopathic disease. 2) Neurologic and neuronopathic manifestations. A growing body of evidence indicates that the peripheral nervous system may be involved in GD type 1 and that there may also be signs and symptoms of central nervous system (CNS) disease in this group. However, GD type 1 is characterized by the absence of primary neuronopathic disease, whereas GD types 2 and 3 are characterized by progressive, albeit variable, primary neuronopathic disease. Abnormalities in saccadic eye movements have been suggested as being diagnostic for neuronopathic GD, but they may also occur in GD type 1 and in other inflammatory diseases. 3) The importance of whole GBA1 sequencing. This approach is superior to exome sequencing because of potential effects of deep intronic variants on gene expression. It also has the capacity to detect variant alleles that might be missed with gene panels. 4) Monoclonal gammopathy of undetermined significance (MGUS). The risks of MGUS, multiple myeloma, and non-Hodgkin's lymphoma are elevated in patients with GD compared to the general population and strong evidence indicates that lyso-Gb1 stimulates the formation of monoclonal immunoglobulins (M-protein) in patients with GD and MGUS. 5) Pulmonary involvement in GD. Pulmonary complications can be identified through spirometry in up to 45 % of patients with GD type 1 and 55 % of those with GD type 3. Limited evidence exists that enzyme replacement therapy (ERT) reduces the severity of these complications in patients with GD type 1. 6) Gaucheromas. These may occur in patients with GD types 1 or 3, but there is little detailed information about their inception, mechanisms underlying growth, cellular organization, and biochemical activities, and no definitive guidance for their management. Gaucheromas behave like benign (i.e. non-metastasizing) neoplasms, and it may be reasonable to classify them as such. 7) Bone and joint involvement. Dual-energy X-ray absorptiometry scans alone are insufficient for monitoring all changes in bone that may occur in patients with GD. Quantitative magnetic resonance imaging (MRI) techniques using Dixon quantitative chemical shift imaging have provided results that correlate with GD severity scores, bone complications, and biomarkers for GD bone involvement. Thoracic kyphosis is a common complication of GD types 1 and 3, and there is very limited information regarding the effects of ERT or substrate synthesis inhibition therapy (SSIT) on this condition. 8) Treatment initiation, selection, combination, and switching. Prompt initiation of treatment in pediatric patients is important as GD can lead to impaired growth, lower peak bone mass, and delayed puberty. These adverse outcomes can often be ameliorated or prevented with timely treatment. Either ERT or eliglustat, a SSIT agent, is suitable as first-line treatment of adults with GD. Studies of switching from ERT to eliglustat, or between different ERT products, have indicated that changing treatment is safe, although efficacy outcomes vary. A critical remaining issue is the lack of treatments capable of reaching the CNS to slow or halt the progression of neuronopathic disease in patients with GD type 2 or 3 and potentially reduce the risk of Parkinson's disease in GD type 1 patients and heterozygotes for GBA1 variants.

2025-02-01·CLINICAL PHARMACOLOGY & THERAPEUTICS

Building Confidence in Physiologically Based Pharmacokinetic Modeling of CYP3A Induction Mediated by Rifampin: An Industry Perspective

Review

作者: Liang, Guiqing ; Parrott, Neil ; Prakash, Chandra ; Hariparsad, Niresh ; Mukherjee, Dwaipayan ; Fu, Qiang ; Umehara, Kenichi ; Lewis, Gareth ; Taskar, Kunal S. ; Boulu, Laurent ; Kotsuma, Masakatsu ; Ramsden, Diane ; Pilla Reddy, Venkatesh ; Bolleddula, Jayaprakasam ; Badée, Justine ; Cabalu, Tamara D. ; de Zwart, Loeckie ; Dowty, Martin E. ; Reddy, Micaela B. ; Rehmel, Jessica ; Leblanc, Alix F. ; Shah, Kushal

Physiologically‐based pharmacokinetic (PBPK) modeling offers a viable approach to predict induction drug–drug interactions (DDIs) with the potential to streamline or reduce clinical trial burden if predictions can be made with sufficient confidence. In the current work, the ability to predict the effect of rifampin, a well‐characterized strong CYP3A4 inducer, on 20 CYP3A probes with publicly available PBPK models (often developed using a workflow with optimization following a strong inhibitor DDI study to gain confidence in fraction metabolized by CYP3A4, fm,CYP3A4, and fraction available after intestinal metabolism, Fg), was assessed. Substrates with a range of fm,CYP3A4 (0.086–1.0), Fg (0.11–1.0) and hepatic availability (0.09–0.96) were included. Predictions were most often accurate for compounds that are not P‐gp substrates or that are P‐gp substrates but that have high permeability. Case studies for three challenging DDI predictions (i.e., for eliglustat, tofacitinib, and ribociclib) are presented. Along with parameter sensitivity analysis to understand key parameters impacting DDI simulations, alternative model structures should be considered, for example, a mechanistic absorption model instead of a first‐order absorption model might be more appropriate for a P‐gp substrate with low permeability. Any mechanisms pertinent to the CYP3A substrate that rifampin might impact (e.g., induction of other enzymes or P‐gp) should be considered for inclusion in the model. PBPK modeling was shown to be an effective tool to predict induction DDIs with rifampin for CYP3A substrates with limited mechanistic complications, increasing confidence in the rifampin model. While this analysis focused on rifampin, the learnings may apply to other inducers.

项与依鲁司他相关的新闻（医药）

2025-03-01

·医药地理

不止罕见：罕见病用药也有春天

2025年2月28日（星期五）是第18个国际罕见病日，今年的主题是“More than you can imagine”，中文主题为“不止罕见”，旨在呼吁全球关注罕见病群体的多维需求，超越疾病本身的“罕见性”，聚焦患者全生命周期管理与社会支持体系的完善。罕见病，亦称孤儿病，是对一类患病率极低、患者总数少的疾病的统称。尽管单一病种发病率低，但是病种多，全球受罕见病影响患者人数达到2.6亿~4.5亿。公开数据显示，全球目前已知的罕见病超过7000种，中国已知的罕见病数量大约有1400余种，约71.9%的罕见病与基因有关，69.9%的罕见病在儿童期发病。由于罕见病常常确诊困难，有大量罕见病被当作普通疾病治疗，或并未发现，实际的病种数量可能更多。近年来，全球药企加速布局罕见病领域，2024年国家医保药品目录新增13种罕见病用药，覆盖戈谢病、肥厚型心肌病、法布雷病等疾病，目录内罕见病药物总数增至3159种。戈谢病（Gaucher Disease，GD）是一种常染色体隐性遗传代谢病，是比较具有代表性的罕见病之一，主要由于葡萄糖脑苷脂酶缺乏或功能异常，临床表现为肝脾肿大、骨骼病变及血液系统异常。据悉，GD在一般人群中的发病率约为0.39至5.80例/10万名新生儿。当前治疗以酶替代疗法（ERT）为主，如伊米苷酶（Imiglucerase）和艾格司他（Eliglustat），通过补充外源性酶改善患者代谢功能，提高患者的生活质量。通过使用Pharma ONE药物研发大数据平台检索，发现全球范围内很多药企都在积极开发戈谢病治疗药物，目前已有3款药物成功上市，更有超过10款药物进入了临床试验阶段，给戈谢病患者带来了更多的治疗选择。表：进入临床试验阶段的戈谢病治疗药物来源：Pharma ONE药物研发大数据平台不仅如此，其他药企也在积极推进罕见病用药向前一步走。当以戈谢病、重症肌无力、法布雷病、视神经脊髓炎谱系障碍、遗传性TTR淀粉样变性、血友病等罕见病为关键词进行检索时，检索结果显示目前已上市的罕见病药物达十余种，覆盖溶酶体贮积症、神经肌肉疾病等细分领域，各大药企的研发效率呈现指数级增长。其中比较具有代表性的两个药物就是萨特利珠单抗和艾加莫德α。萨特利珠单抗（Satralizumab）是一种人源化抗白介素-6受体（IL- 6R）单克隆抗体，由 Genentech 公司研发，可通过特异性结合 IL-6R，阻断IL-6信号通路，从而抑制炎症反应。萨特利珠单抗于 2020年4月获得美国食品药品监督管理局（FDA）批准上市，用于治疗抗水通道蛋白4（AQP4）抗体阳性的视神经脊髓炎谱系障碍（NMOSD）该药物为NMOSD患者提供了首个长效维持治疗方案。艾加莫德α作为全球首创的新生儿Fc受体拮抗剂，是一种人IgG1抗体片段，可与新生儿Fc受体（FcRn）结合，竞争性阻断IgG与FcRn结合，促进IgG被溶酶体降解，从而减少IgG的再循环，显著降低致病性乙酰胆碱受体抗体在内的血清Ig G水平。该药物由Argenx公司研发，2021年7月获得美国食品药品监督管理局（FDA）批准上市，用于治疗成人全身型重症肌无力（gMG），其创新机制为自身免疫性罕见病提供了新的干预路径。表：目前已经上市的特定病种的罕见病治疗药物来源：Pharma ONE药物研发大数据平台同时，以上述疾病为关键词进行检索，也发现了9款进入3期临床试验的罕见病治疗药物，涉及基因疗法、小分子抑制剂及单克隆抗体等多技术路径，相信未来会有更多的罕见病治疗药物出现，为患者带来福音。表：目前进入3期临床的特定病种的罕见病治疗药物来源：Pharma ONE药物研发大数据平台而今，基因编辑（如CRISPR）、RNA疗法及人工智能辅助药物设计等技术正重塑罕见病研发格局，同时，各国“孤儿药”激励政策（如优先审评、市场独占期）也将为企业诸如创新活力，持续推动企业投入“孤儿药”研发。罕见病药物研发的进展，不仅体现了科技的进步和药企的责任，也反映了社会对罕见病患者的关注和关爱，是医学进步与社会公平的重要标尺。随着技术创新、政策优化与社会意识的提升，罕见病领域正逐步突破“无药可医”困境。未来需持续构建“研-产-用”协同生态，真正实现“病虽罕见，关爱不罕至”的愿景。 END 如需获取更多数据洞察信息或公众号内容合作，请联系医药地理小助手微信号：pharmadl001

孤儿药上市批准临床研究

2025-02-06

·化学经纬

书籍分享||新药化学全合成路线手册

内容介绍本书归纳和综述了新药的化学合成路线。覆盖范围为近5年美国FDA批准上市的新分子实体药共109个。针对每一个药物，本书给出了药物简介，药物化学结构信息，产品上市信息，药品专利保护和市场独占权保护信息，化学全合成路线和参考文献等。这些合成路线大多是目前制药工业中正在使用的化学合成工艺，有较高的实用性和学术价值。对参与反应的起始原料、中间体、反应产物都给出了详细的化学结构。每一步化学合成反应都给出了重要的化学试剂、催化剂、所使用的溶剂、合成反应条件及文献出处等。本书的特点是：作者从浩如烟海的科技文献中，精心挑选出最新、最有实用价值的合成方法，汇编成化学合成路线图，内容简洁，深入浅出，逻辑性强，容易理解，以最少的篇幅表达最多的技术信息，可帮助读者很快找到他们需要的药物合成方法及最可靠的原始科学论文。读者对象： 1.任何从事与化学合成、药物合成、精细化工相关专业的科研人员、企业管理人员。 2.有机化学专业、药学专业、生物制药专业等大专院校的高年级学生、研究生、教师及科研人员。 3.从事客户委托的药物原料与药物中间体合成的科研人员、企业管理人员。编辑推荐对于制药企业而言,一个好的药物全合成路线, 可以大大缩短生产周期,简化生产工艺,降低生产成本,节约能源,减少或消除对环境的化学污染与排放。本书为了顺应我国近年来生物制药及医药工业飞速发展的潮流编写的，详细介绍了近五年美国FDA批准上市的所有（109个）新分子实体药的化学全合成路线。这些全合成路线多数是国外制药厂目前正在使用的工艺，其经济效益及社会效益都是极为显著，因此本书既有较高的科学价值又有很大的实用性。作者简介陈清奇，旅美药学家和化学家，担任美国MedKoo生物医药科研公司总裁，同时兼任美国数家生物制药公司的技术顾问，中国国内数个大学的客座教授。研究领域为药物化学，纳米医学体外诊断技术。书籍目录 Abiraterone Acetate（醋酸阿比特龙）001 Aclidinium Bromide（阿克利溴铵）006 Afatinib（阿法替尼）009 Alcaftadine（阿卡他定）014 Alogliptin（阿格列汀）017 Apixaban（阿哌沙班）022 Apremilast（阿普斯特）028 Avanafil（阿伐那非）033 Axitinib（阿西替尼）038 Azilsartan Kamedoxomil（阿齐沙坦酯钾盐）044 Bazedoxifene（巴多昔芬）048 Bedaquiline（贝达喹啉）053 Belinostat（贝利司他）060 Boceprevir（波西普韦）064 Bosutinib（博舒替尼）071 Cabozantinib（卡博替尼）075 Canagliflozin（卡格列净）078 Carfilzomib（卡非佐米）083 Ceftaroline Fosamil（头孢洛林酯）087 Ceftolozane（噻呋特啰嗪）094 Ceritinib（色瑞替尼）098 Clobazam（氯巴占）101 Cobicistat（可比司他）104 Crizotinib（克里唑蒂尼）112 Dabigatran Etexilate Mesylate（甲磺酸达比加群酯）118 Dabrafenib（达拉菲尼）123 Dapagliflozin（达格列净）127 Dasabuvir（达沙布韦）132 Deferiprone（去铁酮）136 Dienogest（地诺孕素）138 Dolutegravir（度鲁特韦）141 Droxidopa（屈昔多巴）145 Efinaconazole（艾氟康唑）149 Eliglustat（依利格鲁司特）152 Elvitegravir（埃替格韦）155 Empagliflozin（依帕列净）161 Enzalutamide（恩杂鲁胺）166 Eribulin Mesylate（甲磺酸艾日布林）168 Eslicarbazepine Acetate（艾利西平醋酸酯）175 Estradiol Valerate（雌二醇戊酸酯）180 Ezogabine（依佐加滨）185 Finafloxacin（非那沙星）193 Fingolimod Hydrochloride（盐酸芬戈莫德）197 Florbetapir F 18（氟比他匹）205 Flutemetamol F 18（富特米他）211 Gabapentin Enacarbil（加巴喷丁恩那卡比）214 Gadobutrol（钆布醇）218 Gadoterate Meglumine（钆特酸葡甲胺）224 Ibrutinib（依鲁替尼）227 Icatibant（艾替班特）232 Idelalisib（艾代拉里斯）234 Indacaterol （茚达特罗）236 Ingenol Mebutate（巨大戟醇甲基丁烯酸酯）240 Ioflupane 123I（碘[123I]氟潘）242 Ivacaftor（依瓦卡特）247 Ledipasvir（雷迪帕韦）250 Linaclotide（利那洛肽）259 Linagliptin（利拉利汀）261 Lomitapide（洛美他派）267 Lorcaserin（氯卡色林）270 Lucinactant（芦西纳坦）274 Luliconazole（卢立康唑）276 Lurasidone（鲁拉西酮）279 Macitentan（马西替坦）283 Miltefosine（米替福新）286 Mipomersen Sodium（米泊美生钠）290 Mirabegron（米拉贝隆）292 Naloxegol（纳罗克格）296 Netupitant（奈妥吡坦）299 Nintedanib（尼达尼布）303 Olaparib（奥拉帕尼）307 Olodaterol（奥达特罗）311 Omacetaxine Mepesuccinate（高三尖杉酯碱）315 Ombitasvir（艾姆伯韦）319 Ospemifene（奥培米芬）323 Paritaprevir（帕瑞它韦）327 Pasireotide（帕瑞肽）331 Peramivir（帕拉米韦）333 Perampanel（吡仑帕奈）337 Pirfenidone（吡非尼酮）343 Pomalidomide（泊马度胺）345 Ponatinib（泊那替尼）351 Regorafenib（瑞戈非尼）355 Rilpivirine（利匹韦林）357 Riociguat（利奥西呱）366 Rivaroxaban（利伐沙班）370 Roflumilast（罗氟司特）378 Ruxolitinib（鲁索替尼）384 Simeprevir（司美匹韦）388 Sofosbuvir（索非布韦）396 Suvorexant（苏沃雷生）401 Tafluprost（他氟前列腺素）405 Tasimelteon（他司美琼）409 Tavaborole（它瓦波罗）413 Tedizolid Phosphate（磷酸特地唑胺）415 Telaprevir（替拉瑞韦）418 Teriflunomide（特立氟胺）426 Ticagrelor（替格瑞洛）430 Tofacitinib（托法替尼）438 Trametinib（曲美替尼）443 Umeclidinium（芜地溴铵）447 Vandetanib（凡德他尼）450 Vemurafenib（威罗菲尼）456 Vilanterol（维兰特罗）460 Vilazodone Hydrochloride（盐酸维拉佐酮）463 Vismodegib（维莫德吉）468 Vorapaxar（沃拉帕沙）470 Vortioxetine（沃替西汀）473 附录1本书使用的期刊名称缩写表477 附录2专利文献代码481 附录3美国药品专利中的用途代码定义484 附录4美国药品市场独占权代码定义530 附录5美国FDA于2010—2014批准上市的药物名单563 英文索引607 中文索引611 CAS登记号索引613 下载方式现在，这份资料分享给大家。小编在公众号设置了自动回复功能，在公众号后台回复关键词：新药全合成 ●书籍分享||有机氧化反应原理与应用 ●书籍分享||有机卤化反应原理及应用 ●推荐合成书籍【有机缩合反应原理与应用】 ●推荐合成书籍【有机环合反应原理与应用】 ●书籍分享||有机还原反应原理与应用 ●ChemDraw使用让你从化学菜鸟变成老鸟 ●干货分享||有机合成: 策略与控制 ●干货分享||有机合成工艺研发-副反应抑制法 ●基础有机化学(邢其毅)第3、4版教材+习题解析 ●ChemDrawV20最新版详细安装图文教程 ●干货分享||核磁共振二维谱 ●书籍分享||全合成中的经典:更多的目标、战略和方法 ●书籍分享||实用药物化学 ●书籍分享||基于模仿创新的小分子药物发明 ●书籍分享||制药工艺开发-目前的化学与工程挑战 ●书籍分享||手性合成:不对称反应及其应用(第5版) ●书籍分享||新药研发案例研究--明星药物如何从实验室走向市场 ●书籍分享||英汉·汉英化学化工词汇 ●书籍分享||有机化学结构与功能（第四版） ●书籍分享||过渡金属有机化学(原著第6版） ●重磅书籍分享||不对称催化基础 ●书籍分享||药物设计学-唐赟

上市批准高管变更

2025-01-30

·GlobeNewswire-Health Care

Press Release: Q4 sales growth of 10.3%, 2024 business EPS guidance exceeded, and strong business EPS rebound expected in 2025

Sanofi: Q4 sales growth of 10.3%, 2024 business EPS guidance exceeded, and strong business EPS rebound expected in 2025 Paris, January 30, 2025 Q4: sales growth of 10.3% at CER1 and business EPS2 of €1.31 Pharma launches up 56.5%, reaching sales of €0.8 billion, 8% of total sales, led by ALTUVIIIODupixent sales up 16.0% to €3.5 billionVaccines sales up 10.8% to €2.2 billion, driven by Beyfortus sales in EuropeBusiness EPS of €1.31, -11.0% at CER and -14.9% reported; IFRS EPS of €0.54 FY: double-digit sales growth and business EPS guidance exceeded Sales totaled €41.1 billion, an increase of 11.3% at CERSales targets exceeded: Dupixent >€13 billion and Beyfortus blockbuster status (€1.7 billion) in its first full yearResearch and Development expenses reached €7.4 billion, up 14.6%, in line with commitmentsBusiness EPS of €7.12, +4.1% at CER, above guidance, and -1.8% reported; IFRS EPS of €4.59The Board of Directors met on January 29, 2025; proposes a dividend of €3.92 for 2024, 30th year of consecutive increases Pipeline: increased investment and progress Q4: four regulatory approvals: Dupixent EoE (children) (EU), Kevzara PMR (EU), Cerdelga GD1 (children) (EU), Efluelda flu (JP)3FY: 14 regulatory approvals of medicines and vaccines, 21 regulatory submission acceptances, and eight positive phase 3 readouts emphasize a positive and improving pipeline momentum Further progress towards a focused biopharma business Intention to sell a controlling stake in Opella consumer health at an attractive valuation; closing in Q2 2025 at the earliest4 Guidance In 2025, sales are anticipated to grow by a mid-to-high single-digit percentage at CER5. Sanofi confirms the expectation of a strong rebound in business EPS with growth at a low double-digit percentage at CER (before share buyback).6Sanofi intends to execute a share buyback program in 2025 of €5 billion. Shares will be purchased preferably through block trades and in the open market with the purpose of cancellation. Paul Hudson, Chief Executive Officer: “We achieved double-digit sales growth in 2024 while pursuing the transformation of the company. Innovation was a key driver of our growth as launches already contributed 11 percent of sales, with Beyfortus becoming a blockbuster in its first full year of sales. We exceeded our business EPS guidance. In 2024, we announced an intention to sell a controlling stake in Opella consumer health, which will make Sanofi a focused, science-driven biopharma company. We increased R&D investments and achieved significant progress with our pipeline in 2024, including positive phase 3 study results for new medicines such as rilzabrutinib in rare diseases and tolebrutinib in multiple sclerosis. As we enter 2025, we expect continued, solid growth in sales and a strong rebound in earnings. We are also confident in the mid to long-term growth prospects of Sanofi, supported by ongoing launches, Dupixent (currently expected to reach sales of around €22 billion in 20307, in line with the current ambition), and expected future launches from our pipeline.” Q4 2024 Change Change at CER FY 2024 Change Change at CER IFRS net sales reported €10,564m +9.1% +10.3% €41,081m +8.6% +11.3% IFRS net income reported €683m — — €5,744m +6.4% — IFRS EPS reported €0.54 — — €4.59 +6.5% — Free cash flow8 €2,340m -25.5% — €5,955m -19.6% — Business operating income €2,078m -11.8% -7.7 % €11,343m +1.5% +7.6% Business net income €1,642m -15.1% -11.2 % €8,912m -1.8% +4.1% Business EPS €1.31 -14.9% -11.0% €7.12 -1.8% +4.1% 1 Changes in net sales are at constant exchange rates (CER) unless stated otherwise (definition in Appendix 9).2 To facilitate an understanding of operational performance, Sanofi comments on the business net income statement which is a non-IFRS financial measure (definition in Appendix 9). The income statement is in Appendix 3 and a reconciliation of reported IFRS to business net income is in Appendix 4.3 For the definition of medical and scientific terms, please see the first use of the word in the Pipeline update section.4 Subject to finalization of definitive agreements and subject to obtaining regulatory approvals from the competent authorities.5 In 2025, sales growth will exclude any impact from hyperinflation. In 2024, it is estimated that sales growth benefited by 1.8 percentage points.6 Applying average January 2025 exchange rates, the currency impacts are estimated between +2% and +3% on sales and +2% and +3% on business EPS.7 At CER.8 Free cash flow is a non-IFRS financial measure (definition in Appendix 9). Attachment Press_Release

临床3期临床结果财报生物类似药上市批准

100 项与依鲁司他相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D09894	依鲁司他	A16AX10	DB09039

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
戈谢病	日本	Sanofi KK	2015-03-26
戈谢病	日本	Genzyme Corp.	2015-03-26
I型戈谢病	美国	Genzyme Corp.	2014-08-19

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
Ⅲ型戈谢病	临床3期	日本	Sanofi	2018-04-11
Ⅲ型戈谢病	临床3期	阿根廷	Sanofi	2018-04-11
Ⅲ型戈谢病	临床3期	加拿大	Sanofi	2018-04-11
Ⅲ型戈谢病	临床3期	法国	Sanofi	2018-04-11
Ⅲ型戈谢病	临床3期	意大利	Sanofi	2018-04-11
Ⅲ型戈谢病	临床3期	俄罗斯	Sanofi	2018-04-11
Ⅲ型戈谢病	临床3期	西班牙	Sanofi	2018-04-11
Ⅲ型戈谢病	临床3期	瑞典	Sanofi	2018-04-11
Ⅲ型戈谢病	临床3期	土耳其	Sanofi	2018-04-11
Ⅲ型戈谢病	临床3期	英国	Sanofi	2018-04-11

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临床结果

适应症

分期

评价

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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


NCT03485677 (ELIKIDS, SSIEM2023)	临床3期	戈谢病一线 CYP2D6 extensive metabolizers \| N370S variant	57	Eliglustat monotherapy	廠獵觸願顧艱簾鑰廠繭(遞餘範築網築廠構構廠) = 鹹壓遞願艱範淵願窪襯鏇糧壓選遞鏇衊鹹醖繭 (簾網鏇選鹽繭觸顧廠範 ) 更多	-	2023-08-30
				Eliglustat plus imiglucerase combination therapy	廠獵觸願顧艱簾鑰廠繭(遞餘範築網築廠構構廠) = 鬱醖膚獵窪築願淵襯觸鏇糧壓選遞鏇衊鹹醖繭 (簾網鏇選鹽繭觸顧廠範 ) 更多
ICGG Gaucher Registry (SSIEM2022)	N/A	一线 CYP2D6-metabolizer status	-	eliglustat	鹹襯襯壓鬱積觸壓繭鑰(蓋廠製窪遞齋顧顧窪夢) = 簾憲選範鑰願願構觸製選廠鹽餘憲簾構簾製鏇 (願夢鬱淵顧憲顧淵淵積 ) 更多	-	2022-08-30
				eliglustat	鹹襯襯壓鬱積觸壓繭鑰(蓋廠製窪遞齋顧顧窪夢) = 艱鬱顧憲壓夢願齋襯顧選廠鹽餘憲簾構簾製鏇 (願夢鬱淵顧憲顧淵淵積 ) 更多
NCT02536755 (EXOSKEL, CTgov)	临床3期	戈谢病	31	Eliglustat, GZ385660	襯淵鹹繭廠襯醖選艱築 = 醖襯淵觸鏇憲壓餘窪觸壓鑰範窪齋遞餘鏇齋艱 (積膚窪製壓齋壓鹹夢衊, 夢構鹽壓網壓築淵築艱 ~ 淵鬱鹹夢範築網鏇繭襯) 更多	-	2022-07-15
NCT00891202 (ENGAGE, Pubmed \| Am J Hematol)	临床3期	I型戈谢病	40	Eliglustat	遞鹹鏇餘獵網衊選夢壓(獵鑰網觸鬱鹽鑰醖憲積) = 醖憲鏇遞鹹積遞遞積淵夢衊顧繭顧窪願網衊願 (遞繭糧願齋衊繭遞顧鑰 ) 更多	积极	2021-06-23
				Placebo	鏇壓襯窪鏇築簾構夢糧(構觸顧壓鑰遞齋觸醖憲) = 繭壓膚齋鹹鏇蓋壓構壓廠鹹衊製艱鑰襯築襯廠 (簾築鏇衊醖鏇齋齋壓簾 ) 更多
EHA2019	N/A	I型戈谢病一线	-	Eliglustat	鑰構築淵簾選憲衊鏇製(觸選繭艱憲顧鬱憲顧襯) = 網網願願窪齋製鬱憲繭獵壓鏇壓獵築鏇蓋蓋蓋 (製網鑰鬱糧廠繭廠範艱 ) 更多	-	2019-06-14
NCT00358150 (Pubmed \| Am J Hematol) 人工标引	临床2期	I型戈谢病一线	26	eliglustat	憲淵醖膚選夢壓醖網網(鹽餘襯網網夢夢憲鹹繭) = 餘觸醖範範憲鑰鑰網艱鹽築鏇蓋蓋繭蓋齋範壓 (膚獵蓋選衊壓築壓範鏇 ) 更多	积极	2019-01-01
NCT01074944 (EDGE, Pubmed) 人工标引	临床3期	I型戈谢病	170	eliglustat (once-daily)	網顧艱餘憲網醖獵淵壓(製膚夢鹹觸鬱廠選繭構) = 窪醖繭觸齋顧鏇鬱築鏇網製襯積衊襯積鹹壓構 (憲蓋膚鏇繭積襯襯構鹽, 67.6 ~ 89.8)	积极	2018-03-01
				eliglustat (twice-daily)	網顧艱餘憲網醖獵淵壓(製膚夢鹹觸鬱廠選繭構) = 願觸選夢鏇鹽醖鬱繭餘網製襯積衊襯積鹹壓構 (憲蓋膚鏇繭積襯襯構鹽, 71.0 ~ 91.6)
NCT01074944 (EDGE, CTgov)	临床3期	I型戈谢病	170	PAP+Eliglustat (PAP, Eliglustat: Once Daily)	鏇製構築衊糧鑰廠繭觸 = 夢艱蓋積憲餘壓齋蓋憲醖鏇鏇鹽鹹積鑰醖憲醖 (範衊積淵鹽窪鏇選選窪, 艱餘觸願選齋淵遞壓製 ~ 壓衊構襯鹽夢觸醖遞壓) 更多	-	2016-12-02
				PAP+Eliglustat (PAP, Eliglustat: Twice Daily)	鏇製構築衊糧鑰廠繭觸 = 顧積廠淵鏇積憲築鏇鬱醖鏇鏇鹽鹹積鑰醖憲醖 (範衊積淵鹽窪鏇選選窪, 窪積製簾餘鏇積築艱窪 ~ 遞獵鹽網衊膚繭遞鬱範) 更多
NCT00943111 (ENCORE, Pubmed \| Lancet) 人工标引	临床3期	I型戈谢病	160	Eliglustat	窪膚齋簾鬱鬱廠積醖網(餘壓餘願遞範鑰淵築夢) = 壓簾築憲憲構淵鬱簾簾鹽壓獵鹽築壓夢襯獵衊 (願壓膚築網鏇廠構製壓 ) 更多	非优	2015-06-13
				imiglucerase	窪膚齋簾鬱鬱廠積醖網(餘壓餘願遞範鑰淵築夢) = 壓鹽衊繭積網觸艱範鑰鹽壓獵鹽築壓夢襯獵衊 (願壓膚築網鏇廠構製壓 ) 更多
NCT00891202 (ENGAGE, Pubmed \| J Coll Physicians Surg Pak \| JAMA) 人工标引	临床3期	脾肿大 \| I型戈谢病	40	eliglustat	製淵廠壓衊鬱構繭衊範(鏇顧製簾廠構夢構窪醖) = 顧廠顧鬱鹽觸齋範簾糧鏇鹽壓網艱鹹壓鑰襯醖 (積糧願膚積襯憲窪積衊, -32.57 ~ -22.97)	积极	2015-02-17
				Placebo	製淵廠壓衊鬱構繭衊範(鏇顧製簾廠構夢構窪醖) = 鑰觸夢廠鹹齋餘鹽齋範鏇鹽壓網艱鹹壓鑰襯醖 (積糧願膚積襯憲窪積衊, -2.54 ~ 7.06)