A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)

Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age.
Amyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN.
Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs.
This study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.

开始日期2025-05-01

申办/合作机构

Eidos Therapeutics, Inc.

ISRCTN11628974

/ Completed临床1期

A multiple dose study to evaluate the pharmacokinetics of an acoramidis (AG10) modified release tablet formulation in healthy subjects

开始日期2022-08-30

申办/合作机构

Eidos Therapeutics, Inc.

NCT04988386

/ Active, not recruiting临床3期

An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

开始日期2021-10-27

申办/合作机构

Eidos Therapeutics, Inc.

100 项与 Acoramidis 相关的临床结果

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100 项与 Acoramidis 相关的转化医学

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100 项与 Acoramidis 相关的专利（医药）

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项与 Acoramidis 相关的文献（医药）

2025-12-01·Current heart failure reports

Etiological Treatment of Cardiac Amyloidosis: Standard of Care and Future Directions

Review

作者: Morfino, Paolo ; Emdin, Michele ; Vergaro, Giuseppe ; Chubuchna, Olena ; Aimo, Alberto ; Castiglione, Vincenzo ; Buda, Gabriele ; Ferrari Chen, Yu Fu ; Fabiani, Iacopo

Abstract:

Purpose of Review:

Cardiac amyloidosis (CA) is a condition caused by interstitial infiltration of misfolded proteins structured into amyloid fibrils. Transthyretin (ATTR) and immunoglobulin light chain (AL) amyloidosis represent the most common forms of CA. CA was traditionally perceived as a rare and incurable disease, but diagnostic and therapeutic advances have undermined the conventional paradigm.

Recent Findings:

The standard of care for ATTR-CA include agents capable of selectively stabilizing the precursor protein (e.g., tafamidis), whereas the plasma cell clone is the main target of chemotherapy for AL-CA. For long, tafamidis represented the only drug approved for patients with ATTR-CA. Recent data from ATTRibute-CM led to the approval of acoramidis, whereas patisiran received refusal based on the APOLLO-B trial. Novel CRISPR-Cas9-based drugs (i.e., NTLA-2001) hold great potential in the setting of ATTR-CA. Several hematological regimens are available to treat AL-CA. The main limit of current therapies is their inability to trigger removal of amyloid from tissues. However, the investigation of monoclonal antibodies targeting misfolded ATTR (e.g., PRX004, NI301A) or AL (e.g., birtamimab, anselamimab) has led to encouraging results.

Summary:

Various cutting-edge strategies are being tested for treatment of CA and may change the prognostic landscape of this condition in the next years.

2025-12-01·MOLECULAR BIOLOGY REPORTS

Transthyretin as a therapeutic target: the future of disease-modifying therapies for Alzheimer’s disease

Review

作者: Shaw, Swetaleena ; Aran, Khadga Raj ; Porel, Pratyush

BACKGROUND:

Alzheimer's disease (AD) is the most common neurodegenerative disease for causing memory deficits and primarily characterized by extracellular deposition of amyloid-β (Aβ) plaques, intracellular neurofibrillary tangles (NFTs), and hyperphosphorylation of tau protein, all are pathological hallmarks for AD. Transthyretin (TTR) is a highly conserved homo-tetrameric protein, primarily synthesized in liver and choroid plexus, and most importantly involved in transport of T₃-T₄ hormones and retinol.

OBJECTIVES:

This review explores the dual role of TTR, with a greater emphasis on its neuroprotective action, particularly in AD.

METHODS:

Based on the available literature, TTR's potential as a biomarker in the central nervous system (CNS), focusing its role in stabilizing Aβ aggregation and the senile plaque formation during neurodegeneration. Additionally, TTR's dual roles, in neurodegeneration and neuroprotection are studied, emphasizing its potential for improving AD diagnosis and treatment strategies.

RESULTS:

Recent research has revealed that TTR is gradually showcasing its promise in neuroprotection and neuronal viability in AD by binding with Aβ and mitigating its neurotoxic effects. Current preclinical and clinical studies also support that TTR is actively involved in maintaining the blood-brain barrier (BBB) integrity and maintain neurotransmitter balance, all of which offer significant therapeutic promise through TTR stabilizers, such as Tafamidis, Acoramidis, and Vutrisiran, highlighting their potential in AD treatment CONCLUSION: This review concludes that TTR plays bidirectional role and gaining interest as a potential biomarker, though several challenges must be addressed before it can be established a novel therapeutic target in AD management in the modern era of drug discovery.

2025-04-16·AMERICAN JOURNAL OF HEALTH-SYSTEM PHARMACY

Acoramidis Hydrochloride

Article

250

项与 Acoramidis 相关的新闻（医药）

2025-05-08

·微信

RNAi疗法获批后的新格局

近日（3月20日），FDA已经批准了Alnylam Pharmaceuticals的RNAi疗法Amvuttra（vutrisiran），这次是用于治疗转甲状腺素蛋白淀粉样心肌病（ATTR-CM）。这是Amvuttra继2022年6月获批治疗遗传性转甲状腺素蛋白介导的淀粉样变性多发性神经病（hATTR-PN）后的第二项适应症。此次批准标志着ATTR-CM治疗领域首次引入RNA干扰（RNAi）技术，并与现有的两种转甲状腺素蛋白（TTR）稳定剂——辉瑞（Pfizer）的tafamidis（Vyndaqel/Vyndamax）和BridgeBio的Attruby（acoramidis）——形成三足鼎立的竞争格局。为什么两项适应症的获批差了近3年？相比而言，ATTR-CM的适应症开发要比PN更为艰难。hATTR-PN以周围神经病变为主，患者主体是年轻人，可通过神经功能评分（如mNIS+7）在较短时间内评估疗效。尤其是支持PN获批的III期临床HELIOS-A，仅需9个月即显示显著改善，达到了主要终点和次要终点，能支持快速审批。而相比而言，ATTR-CM适应症的开发就困难多了，在ATTR-CM中，由于肝脏产生有缺陷的TTR蛋白。这些畸形的蛋白质团块随后在心脏的主泵腔中积聚，导致心肌病，使心脏更难将血液泵入身体，涉及心脏需要考虑的就多了。需评估对生存率的影响（如全因死亡率、心血管事件复发），此类终点需长期随访（CM的III期临床HELIOS-B试验长达42个月）以验证疗效。例如，Amvuttra需证明降低36%全因死亡率，这对试验设计和数据收集时间要求更高。更关键的是，CM的患者平均年龄76.5岁，合并症多（如心力衰竭），试验入组和随访难度大。临床设计上，HELIOS-B试验允许患者同时使用TTR稳定剂（如辉瑞的tafamidis），tafamidis自2019年获批以来就是该领域的长期霸主，在市场地位举足轻重，Amvuttra想要获批，还需验证Amvuttra的独立疗效及联合治疗的额外获益，增加了数据分析复杂度。竞争态势越发激烈为了加速批准，Alnylam特别使用了一张优先审查凭证，将原本标准10个月评估期缩短4个月。从中不难看出，ATTR-CM的竞争格局正在呈现出白热化态势，而时间就是金钱。预计到2030年，ATTR-CM市场将达112亿美元，目前约80%患者未被确诊，增长空间显著。而与此同时竞争对手也不会放过这一市场。Amvuttra的优势在于给药便利性，机制。辉瑞的tafamidis（Vyndaqel/Vyndamax）和BridgeBio的Attruby（acoramidis）在机制上都是TTR稳定剂，通过稳定TTR蛋白的四聚体结构，减少错误折叠蛋白在心脏中的沉积。而Amvuttra则直接靶向沉默TTR基因的mRNA，从源头上减少TTR蛋白的生成，阻断淀粉样纤维的形成。这种机制上的优势，转化为临床数据就是更优秀的全因死亡率获益。在HELIOS-B试验中，Amvuttra相比安慰剂显著降低全因死亡风险和心血管事件复发率，而Attruby在III期ATTRibute-CM试验中未能达到这一次要终点。而从给药便捷性来看，Amvuttra每年仅需4次皮下注射，而Attruby需每日口服两次，辉瑞的tafamidis为每日一次。在这种情况下，可以期待Amvuttra在随后的商业化竞争中出现后来者居上的情况。对于Amvuttra来说，更值得担忧的可能是后来的竞争者。阿斯利康/Ionis的RNA沉默剂Wainua未来可能是强劲对手，目前正进行III期CARDIO-TTRansform试验评估其对ATTR-CM的疗效，预计2026年4月完成。Intellia的基因编辑疗法nex-z则可能更具有给药便捷性优势，作为一次性疗法，2024年11月公布的I期数据显示，单次给药后12个月内ATTR-CM患者血清TTR水平平均降低90%，且效果“快速、深度、持久”。参考来源：https://investors.alnylam.com/press-release?id=28831https://www.biospace.com/drug-development/alynlams-amvuttra-approved-as-first-rnai-silencer-for-rare-type-of-cardiomyopathy推文用于传递知识，如因版权等有疑问，请于本文刊发30日内联系医药速览。原创内容未经授权，禁止转载至其他平台。有问题可发邮件至yong_wang@pku.edu.cn获取更多信息。©2021 医药速览保留所有权利往期链接“小小疫苗”养成记 | 医药公司管线盘点人人学懂免疫学| 人人学懂免疫学（语音版）综述文章解读 | 文献略读 | 医学科普|医药前沿笔记PROTAC技术| 抗体药物| 抗体药物偶联-ADC核酸疫苗 | CAR技术| 化学生物学温馨提示医药速览公众号目前已经有近12个交流群（好学，有趣且奔波于医药圈人才聚集于此）。进群加作者微信（yiyaoxueshu666）或者扫描公众号二维码添加作者，备注“姓名/昵称-企业/高校-具体研究领域/专业”，此群仅为科研交流群，非诚勿扰。简单操作即可星标⭐️医药速览，第一时间收到我们的推送①点击标题下方“医药速览” ②至右上角“...” ③点击“设为星标·····

优先审批临床3期信使RNA临床2期

2025-04-30

·EndPoints

BridgeBio’s Attruby outperforms sales expectations, shares rise

When BridgeBio reported Attruby prescription numbers in February, the figures came in substantially above analyst projections. Now the company has pulled off a similar trick. The heart disease drug smashed the street’s first-quarter sales forecasts according to figures BridgeBio released late on Tuesday. Since its US market debut in November , Attruby was prescribed 2,072 times up to Apr. 25 — a jump of 1,044 since Feb. 17. The pill’s revenues for the first quarter of 2025 came in at $36.7 million, and BridgeBio’s shares $BBIO climbed 8% premarket Wednesday. These figures do not include sales or prescriptions of the drug in Europe. It was approved in the EU under the name Beyonttra in February, and is sold there by Bayer. Attruby treats a progressive, fatal condition called transthyretin amyloidosis with cardiomyopathy (ATTR-CM), in which a misfolded protein accumulates in the heart. Attruby works by stabilizing the protein’s shape. Analysts from Cowen wrote in an Apr. 29 note that the drug “came screaming out of the gate,” handily beating their forecast Q1 sales of just $17 million. Other bankers had pegged the Q1 numbers at around $19-20 million. The US Attruby prescriptions were written by 756 healthcare workers, and the Cowen analysts pointed out that as around 1,300 doctors write 80% of ATTR-CM prescriptions, BridgeBio has reached nearly 60% of them in the first five months of Attruby’s availability. The analysts upped their 2025 forecasts for the drug to $275 million. Hello, Isaiah Sanders You’re an essential part of the biopharma world. Share your voice on this important question to help shape our understanding of the industry. Can you help us out? Hello, Isaiah Sanders You’re an essential part of the biopharma world. Share your voice on this important question to help shape our understanding of the industry. Can you help us out? On a conference call to discuss the company’s Q1 results, BridgeBio CEO Neil Kumar credited Attruby’s commercial success to its clinical data . He added that around 80% of ATTR-CM patients are yet to be diagnosed, providing a tailwind for future growth. The challenge here is the launch of Alnylam’s rival, Amvuttra, which was approved in ATTR-CM last month with a broader label than Attruby. Overall, the company’s first-quarter sales were $116.6 million, a 45% drop from the same period in the prior year. This fall was primarily due to a $131.2 million decrease in license and services revenue, the company said. BridgeBio ended the first quarter with $541 million in cash and equivalents and expects a further $105 million in milestone payments in the second quarter for approvals of Beyonttra outside the US.

上市批准财报生物类似药加速审批

2025-04-29

·蒲公英Ouryao

一周 | 国际法规药事(04.21-04.27)

◆ ◆ ◆ ◆Paul's Insight2025年4月21日-4月27日◆ ◆ ◆ ◆过去一周（4月21日至4月27日），全球药事监管和行业机构齐齐发声，犹如一场精彩的药品政策与科技盛宴。从FDA的合成色素退市行动，到EMA的罕见病新疗法推荐；从EDQM对生物制品检测标准的升级，到MHRA多款创新疗法的批准；再到行业协会PDA和ISPE在指南与实践层面的多重发力，这一连串新闻不仅彰显了各大机构的监管力度，更映射出制药行业在创新、合规与可持续性间的不断平衡。美国FDA从食药安全到创新药物的“双轨”发力1. 合成食用色素“退场”行动4月22日，美国FDA宣布，计划逐步淘汰美国食品供应中的合成色素（Petroleum-Based Synthetic Dyes），为儿童健康撑起防护伞。FDA表示，这一举措，是“让美国再次健康”（Make America Healthy Again）计划的重要里程碑。此前，1月15日赤藓红（FD&C Red No. 3）禁用已拉开了帷幕，后续这一更广泛的色素退市，将持续推动产业寻求天然、低风险的替代方案。图：赤藓红禁用后，FDA再推新规，八大合成食用色素将退场https://www.fda.gov/news-events/press-announcements/hhs-fda-phase-out-petroleum-based-synthetic-dyes-nations-food-supply2. 药品安全敲响警钟：外用非那雄胺安全红线4月22日，FDA对市场上未经批准的外用非那雄胺配药制剂发出警示，提醒医疗机构、药房及消费者：目前无任何局部非那雄胺产品拥有FDA批准的标签和安全、有效性验证，配制药剂可能隐藏严重风险，包括与米诺地尔等混合制剂的混用场景。FDA直指部分处方药店和远程医疗平台的药品安全风险行为，呼吁行业加强自律与审查。图：就非那雄胺外用安全问题，FDA发布脱发常用药警示https://www.fda.gov/drugs/human-drug-compounding/fda-alerts-health-care-providers-compounders-and-consumers-potential-risks-associated-compounded3. 中国创新药的海外“登台”——派安普利单抗获批4月24日，FDA官网放出重磅消息：康方生物自主研发的PD1单抗派安普利单抗（Penpulimabkcqx）获批，用于复发或转移性非角化型鼻咽癌（NPC）治疗。该产品采用IgG1亚型Fc段改造设计，依托突破性疗法认定、孤儿药资格和快速通道三重加速，联袂正大天晴药业集团布局后续商业化。此举不仅为全球鼻咽癌患者带来曙光，也标志着中国创新药在国际舞台上稳步迈进。（点击此处查看相关阅读）图：中国制药行业又一里程碑，派安普利单抗获FDA批准进入美国市场https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-penpulimab-kcqx-non-keratinizing-nasopharyngeal-carcinoma美国PDA发布新闻季刊4月24日，美国注射剂协会（PDA）发布新闻季刊，总结其在过去一个季度的工作，要点如下。图：PDA发布新闻季刊https://www.pda.org/pda-letter-portal/home/full-article/community-news-quarterly-april-20251）2024年PDA出版奖揭晓PDA颁发奖项给Novartis&Ogilvy团队，表彰其用于无菌性检测的创新方法，该论文名为《一种利用固相流式细胞术进行细胞制备、培养基和缓冲液的快速无菌方法》。年度PDA Letter文章奖项授予武田制药的《理解日本品质》。2）区域分会动态：德国、奥地利和瑞士分会（DACH）亮点纷呈DACH分会“目视检测与颗粒挑战”网络研讨会吸引400余名专家，聚焦AI时代的风险基础方法与质量设计。PDA表示，此次网络研讨会超出预期，如此高的参与度凸显了业界对目视检测和颗粒物相关挑战的关注，同时也强化了DACH分会促进制药行业知识交流和最佳实践的使命。欧盟EMA欧盟罕见病与适应症扩展双管齐下1. DMD新疗法——组蛋白去乙酰化酶（HDAC）抑制剂获“有条件上市许可”4月25日，欧洲药品管理局（EMA）发布杜氏肌营养不良症（DMD）创新药物Duvyzat（givinostat）“有条件上市许可”建议。该组蛋白去乙酰化酶（HDAC）抑制剂在120例6岁以上患者的18个月研究中显著延缓台阶攀爬时间，虽次要终点未全部显著，但整体数据朝积极方向发展。获得“有条件上市许可”，这是欧盟的监管机制之一，旨在促进早期上市药物，以满足未满足医疗需求的。EMA要求申请人后续进行一项随机、安慰剂对照研究，并进行长期随访将补足盲点。上市许可人为Italfarmaco S.p.A.，是是一家总部位于意大利米兰的制药跨国公司。图：杜氏肌营养不良症（DMD）创新药物获“有条件上市许可”https://www.ema.europa.eu/en/news/new-treatment-against-duchenne-muscular-dystrophy2. 甲状腺眼病首创疗法Tepezza获积极审评意见4月25日，EMA对甲状腺眼病（TED）首款靶向IGF1受体的单抗Teprotumumab（Tepezza）给出积极评审意见。三项RCT临床显示活动期患者24周内眼球突出度平均减少2.0–2.3mm，临床活动评分大幅改善；慢性期亦有约1.5mm降低。基于肌肉痉挛、脱发等可管理风险，EMA将附加风险最小化措施并转交欧委会审批。申请人为Amgen Europe B.V.，是全球领先的生物技术公司安进在欧洲的子公司。图：甲状腺眼病首创疗法Tepezza获积极审评意见https://www.ema.europa.eu/en/news/first-treatment-against-severe-thyroid-eye-disease3. CHMP会议要点：新药、扩展、再审与撤回4月22–25日的EMA人药委员会（CHMP）会议，推荐16款新药（包含Vertex囊性纤维化三联疗法、差异化适应症的多款孤用药及九款生物类似药），推动10项适应症扩展，如武田制药的Adcetris多淋巴瘤适应等；同时，礼来的阿尔茨海默药物Kisunla申请再审，Biohaven和辉瑞的两款产品适应失利撤回。整体来看，EMA通过CMA平衡未满足需求与证据要求，也显示出对成熟产品生命周期管理策略的开放态度。图：2025年4月22日至25日人药委员会( CHMP )会议要点https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-april-2025欧洲EDQM高通量测序入典，提升生物制品安全4月23日，EDQM欧洲药典委员会（EPC）正式采纳通则“2.6.41病毒外源性因子检测的高通量测序（HTS）”，自2026年4月1日起生效。新章节涵盖非靶向与靶向HTS方法学、基因组/转录组数据分析和验证指南，驱动与ICH Q5A(R2)修订及WHO国际参考组协同，助力生物制品从传统动物实验迈向更高灵敏度的分子检测。图：高通量测序入欧洲药典https://www.edqm.eu/en/-/epc-adopts-cutting-edge-hts-chapter-to-enhance-viral-contaminant-detection-in-biological-products英国MHRA创新疗法齐发1. 4月拜耳新药Acoramidis获批，用于心肌病4月25日，MHRA批准拜耳制药的Acoramidis（Beyonttra），用于治疗因变异型或野生型转甲状腺素蛋白淀粉样变性（ATTR-CM）引起的心肌病（心肌损伤）成年患者。，632例国际多中心研究显示30个月心血管住院率和死亡率显著降低，6分钟步行距离改善。口服每日两次，通过IRP机制加速审评。图：拜耳新药Acoramidis获批，用于心肌病https://www.gov.uk/government/news/acoramidis-approved-to-treat-wild-type-or-variant-transthyretin-amyloidosis-in-adults-with-cardiomyopathy2. 白血病CAR-T疗法Obecabtagene autoleucel获有条件上市批准4月25日，obecabtagene autoleucel (Aucatzyl) 这一CART疗法获条件上市许可，用于治疗复发或难治性B 细胞前体急性淋巴细胞白血病 (ALL) 的成年患者。FELIX研究153例数据显示12个月总生存率81%、完全缓解55%，后续需提供更多数据满足全面上市要求。上市许可人为Autolus Therapeutics，是一家总部位于英国的生物制药公司，该公司于2014 年从伦敦大学学院（UCL）分拆成立。图：白血病CAR T疗法Obecabtagene autoleucel获有条件上市批准https://www.gov.uk/government/news/obecabtagene-autoleucel-conditionally-approved-to-treat-adults-with-relapsed-or-refractory-b-cell-precursor-acute-lymphoblastic-leukaemia3. 百健新药在英获批4月23日，MHRA批准了 omaveloxolone（Skyclarys），这是英国首个针对 16 岁及以上患者的治疗药物，用于治疗一种名为弗里德赖希共济失调的罕见神经退行性运动障碍。弗里德赖希共济失调是最常见的遗传性共济失调（由遗传基因引起）。据估计，每5万人中至少有1人患有此病。弗里德赖希共济失调的症状包括平衡和运动障碍，并会随着时间的推移逐渐恶化。该药物为口服胶囊剂。上市许可人为Biogen Netherlands B.V.，为全球领先的生物技术公司 Biogen Inc.（百健公司）在荷兰的全资子公司。图：百健新药在英获批https://www.gov.uk/government/news/mhra-approves-first-uk-treatment-for-friedreichs-ataxia-omaveloxolone4. 辉瑞血友病药物获批4月22日，MHRA批准辉瑞药物Marstacimab（Hympavzi），用于预防或减少12岁及以上、体重至少35公斤的血友病A和B患者的出血。该药物是同类药物中第一个通过针对血液凝固过程中的蛋白质来发挥作用的药物。Marstacimab每周一次皮下注射，使用预充式注射器或注射笔。患者或护理人员经过适当培训后即可自行注射药物。图：辉瑞血友病药物获批https://www.gov.uk/government/news/marstacimab-approved-to-treat-patients-aged-12-years-and-above-weighing-at-least-35-kg-with-haemophilia-a-or-b国际ISPE发布数字化验证良好实践指南国际制药工程协会（ISPE）发布了《数字化验证良好实践指南》（Good Practice Guide: Digital Validation），为制药企业提供从传统验证向数字化框架过渡提供了指导。这本90页的指导文件，旨在帮助制药企业采用数字化验证工具（Digital Validation Tools, DVT），提升数据可靠性、运营效率和监管合规性。该指南由全球专家团队开发，其核心成员来自于武田制药、葛兰素史克等全球药企和行业咨询公司。ISPE表示，指南结合真实案例和结构化方法，适用于从初创到优化现有系统的各类企业。（点击此处查看相关阅读）图：ISPE最新发布《数字化验证良好实践指南》https://ispe.org/publications/guidance-documents/good-practice-guide-digital-validation结语回望过去一周，FDA、EMA、EDQM、MHRA、PDA和ISPE等监管与行业机构的多项决策、指南与更新，彰显监管严谨，又鼓励技术革新。从食品安全到精准疗法，从分子检测到数字化治理，昭示出全球制药行业正朝着更健康、更高效、更可持续的未来协奏。END声明：本文仅代表作者个人观点，不代表任何组织及本公众号立场，如有不当之处，敬请指正。如需转载，请注明作者及来源：蒲公英Biopharma。活动推荐5月6日 | 线上 | 新法规和应用场景下的全球包材注册策略和最佳实践5月8日 | 线上 | 双维度解锁无菌药品生产污染防控策略

孤儿药突破性疗法上市批准医药出海申请上市

100 项与 Acoramidis 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11972	-	-	-

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
遗传性转甲状腺素蛋白淀粉样变性	欧盟	BridgeBio Pharma, Inc.	2025-02-10
遗传性转甲状腺素蛋白淀粉样变性	冰岛	BridgeBio Pharma, Inc.	2025-02-10
遗传性转甲状腺素蛋白淀粉样变性	列支敦士登	BridgeBio Pharma, Inc.	2025-02-10
遗传性转甲状腺素蛋白淀粉样变性	挪威	BridgeBio Pharma, Inc.	2025-02-10
转甲状腺素蛋白淀粉样变性心肌病	美国	BridgeBio Pharma, Inc.	2024-11-22

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
多发性神经病	临床3期	美国	Eidos Therapeutics, Inc.	2025-05-01
多发性神经病	临床3期	保加利亚	Eidos Therapeutics, Inc.	2025-05-01
多发性神经病	临床3期	意大利	Eidos Therapeutics, Inc.	2025-05-01
多发性神经病	临床3期	荷兰	Eidos Therapeutics, Inc.	2025-05-01
多发性神经病	临床3期	西班牙	Eidos Therapeutics, Inc.	2025-05-01
转甲状腺素蛋白相关家族性淀粉样多神经病变	临床3期	美国	Eidos Therapeutics, Inc.	2020-10-21
老年性心脏淀粉样变性	临床3期	美国	Eidos Therapeutics, Inc.	2019-03-19
老年性心脏淀粉样变性	临床3期	澳大利亚	Eidos Therapeutics, Inc.	2019-03-19
老年性心脏淀粉样变性	临床3期	比利时	Eidos Therapeutics, Inc.	2019-03-19
老年性心脏淀粉样变性	临床3期	巴西	Eidos Therapeutics, Inc.	2019-03-19

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临床结果

适应症

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


NCT03860935 (ATTRibute-CM, Pubmed \| J Am Coll Cardiol)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 \| 老年性心脏淀粉样变性 N-terminal pro-B-type natriuretic peptide level	632	Acoramidis hydrochloride 800 mg twice daily	鑰繭艱憲齋範網製憲製(壓艱廠窪齋壓鏇鑰網積): relative risk ratio = 50 (95% CI, 0.36 ~ 0.7), P-Value = < 0.0001 更多	积极	2025-03-01
				Placebo
NCT04622046 (CTgov)	临床3期	转甲状腺素蛋白淀粉样变性心肌病	25	ALXN2060	糧築築淵淵鏇繭艱築範(製積選範鑰壓築遞膚憲) = 憲遞齋糧蓋觸窪遞鹹蓋範蓋構顧襯淵願鬱積糧 (鏇選獵壓廠製齋遞鬱蓋, 9.182) 更多	-	2024-12-16
NCT03860935 (FDA_CDER) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	611	ATTRUBY 712 mg	膚觸醖艱鹹膚構壓夢夢(選餘鏇淵鏇餘艱醖鬱鑰): P-Value = 0.018 更多	积极	2024-11-22
				Placebo
NCT03860935 (ATTRibute-CM, Company_Website) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	389	Acoramidis	淵夢獵襯網鹽顧積壓膚(築餘襯觸範鑰構製憲醖) = 顧鏇鹹蓋獵簾夢範壓醖遞獵夢夢遞製餘醖簾蓋 (憲網蓋衊顧憲醖願築選 ) 更多	积极	2024-11-18
				Placebo	-
NCT03860935 (ATTRibute-CM, GlobeNewswire \| PipelineReview) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	-	Acoramidis	繭窪簾壓範齋蓋淵築膚(簾製鏇鹽範範憲窪願積) = 窪蓋膚築遞醖網窪顧艱鑰選顧願選憲範構淵網 (壓憲醖醖遞製齋衊夢衊 )	积极	2024-09-27
				Placebo	-
NCT03860935 (ATTRibute-CM, ESC2024)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 serum TTR levels	611	Acoramidis	夢齋願糧遞夢觸網製齋(簾網鬱淵簾鑰獵膚餘醖) = 觸鹽簾鬱獵鏇簾選顧夢憲築淵憲鑰築積襯糧鏇 (糧淵憲獵衊窪遞膚製網 )	积极	2024-08-30
				Tafamidis	夢齋願糧遞夢觸網製齋(簾網鬱淵簾鑰獵膚餘醖) = 鑰遞醖築餘鏇製選顧繭憲築淵憲鑰築積襯糧鏇 (糧淵憲獵衊窪遞膚製網, 0.4 (4.92))
NCT03860935 (ATTRibute-CM, CTgov)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 \| 老年性心脏淀粉样变性	632	acoramidis (Acoramidis HCl 800 mg)	簾繭糧憲衊積簾窪鹽襯 = 選艱願網鬱觸壓繭鑰鏇製選選齋網觸願餘蓋膚 (築築製鑰糧齋艱鑰襯糧, 願鹽遞網醖壓選鬱廠憲 ~ 醖鹽獵鑰鹽餘選艱憲鏇) 更多	-	2024-06-27
				Placebo Oral Tablet (Placebo)	簾繭糧憲衊積簾窪鹽襯 = 齋糧願齋獵憲範淵餘壓製選選齋網觸願餘蓋膚 (築築製鑰糧齋艱鑰襯糧, 齋齋壓淵壓範艱衊顧艱 ~ 衊觸網製廠簾願醖鹹構) 更多
NCT03860935 (ATTRibute-CM, Company_Website) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	-	Acoramidis	衊範憲構鹽衊廠醖範鑰(構餘觸獵構構壓糧願膚) = every 5mg/dL increase in serum TTR level at day 28 after treatment initiation, the risk of death through Month 30 was reduced by 30.9% (by the logistic model) and 26.1% (by the Cox proportional hazards model) 獵醖簾齋築淵顧觸選齋 (餘鹹範製築鬱廠顧範獵 ) 更多	积极	2024-05-29
				Placebo
ESC2024	N/A	转甲状腺素蛋白淀粉样变性心肌病	-	Acoramidis	網範膚壓顧構壓製鹹遞(遞鑰鏇積積衊壓膚蓋襯) = 糧選觸顧餘齋獵膚膚鏇鬱製範壓繭壓襯鹹遞鹽 (餘壓製觸製鏇願齋築築, 0.07 ~ 0.18)	-	2024-05-13
ESC2024	N/A	转甲状腺素蛋白淀粉样变性心肌病	-	Acoramidis	鬱淵鏇艱淵餘衊鬱願簾(膚積淵襯艱夢鏇選醖糧) = A statistically significant (p<0.0001) and clinically meaningful treatment benefit on KCCQ-OS was observed with acoramidis versus placebo 膚觸壓夢廠獵餘鹽餘積 (夢鹹願鏇遞獵糧淵積鬱 ) 更多	-	2024-05-13
				Placebo