A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients With Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)

The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).

开始日期2025-11-01

申办/合作机构

Eidos Therapeutics, Inc.

NCT06563895

/ Recruiting临床3期

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2025-05-12

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.

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.

2025-06-01·DRUGS

Acoramidis: First Approval

Review

作者: Lee, Arnold

Disassociation of transthyretin (TTR) has been identified as a key step in the pathology of TTR amyloid cardiomyopathy, which is characterised by the presence of amyloid fibrils in cardiac tissue. Acoramidis (ATTRUBY™) is a small molecule TTR stabiliser being developed by BridgeBio Pharma, Inc. and is the first drug to demonstrate near-complete (> 90%) stabilisation of TTR. This article summarizes the milestones in the development of acoramidis leading to its first approval in the USA for the treatment of the cardiomyopathy of wild-type or variant TTR-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization. In the EU, a positive opinion has been adopted for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy.

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项与 Acoramidis 相关的新闻（医药）

2025-08-07

·医药健闻

拜耳第二季度销售额为107.39亿欧元，处方药事业部诺倍戈与可申达实现显著增长

美通社消息，拜耳集团在2025年上半年进展良好。首席执行官比尔•安德森（Bill Anderson）在周三发布上半年财务报告时表示：“我们正步入2025下半年的关键阶段，这一阶段将全面推进各项战略重点领域、持续推出新产品，同时应对地缘政治与汇率带来的多重挑战。”他指出：“得益于处方药事业部从年初至今的良好表现，我们上调了集团2025年全年经汇率调整后的销售额与盈利预期。”该预期已综合考虑当前地缘政治新动向带来的财务影响。同时，公司预计汇率将出现大幅波动，可能会为销售额和利润带来负面影响，但将对金融债务净额产生积极影响。拜耳已于7月31日（周四）提前发布了第二季度关键财务数据，并上调了经汇率调整后的全年销售额与盈利预期。在本次披露的信息中，公司还报告了美国地区与诉讼相关的准备金与负债增加。安德森在周三进一步强调：“公司的目标是到2026年底显著降低诉讼风险。”拜耳正采取多管齐下的战略，全力推进应对措施，以降低公司在诉讼领域的风险。谈及公司正在推进的其他战略重点，安德森首先强调了处方药事业部的表现：“除了处方药业务的运营成效外，我们的研发管线及新上市产品持续取得进展。” 集团销售额（经汇率与资产组合调整）及不计特殊项目的息税折旧摊销前利润保持稳定拜耳集团2025年第二季度销售额为107.39亿欧元，增长0.9%（经汇率与资产组合调整）。汇率造成的负面影响为5.5亿欧元（2024年第二季度：2.4亿欧元）。不计特殊项目的息税折旧摊销前利润为21.05亿欧元（负0.3%），其中包括1.84亿欧元的汇率负面影响（2024年第二季度：1.29亿欧元）。扣除9.81亿欧元的净特殊费用后（2024年第二季度：4.9亿欧元），息税前利润为1300万欧元（2024年第二季度：5.25亿欧元）。特殊费用主要包括诉讼相关准备金的计提、作物科学事业部的减值损失转回及重组相关支出。净收益为负1.99亿欧元（2024年第二季度：负3400万欧元）。每股核心收益增长30.9%，至1.23欧元，主要得益于财务业绩改善及税费支出减少。自由现金流下降90.2%，至1.25亿欧元。此次下降主要由于经营现金流减少，而经营现金流减少主要归因于集团短期激励计划（STI）支出增加，以及去年同期作物科学事业部应收账款减少的季度性波动影响。截至2025年6月30日，金融债务净额为332.74亿欧元，较2025年3月31日下降2.9%，主要得益于汇率变动产生的积极影响。与2024年6月30日相比，金融债务净额下降9.5%。处方药事业部诺倍戈®与可申达®持续实现显著增长处方药业务（处方药事业部）的销售额增长0.6%（经汇率与资产组合调整），达到44.7亿欧元。该事业部诺倍戈®（用于治疗前列腺癌）和可申达®（用于治疗与2型糖尿病相关的慢性肾病）销售额再度实现强势增长，分别上升50.5%和67.1%（经汇率与资产组合调整）。影像诊断业务同样实现了大幅增长，主要是因为优维显®和CT高压注射系统产品线的销量提升。此外，眼科药物艾力雅®的销售额增长4.3%（经汇率与资产组合调整），特别是新上市的艾力雅® 8 mg能够实现更长的给药间隔，有效推动了业务增长。口服抗凝药物拜瑞妥®的销售额则由于专利到期下降了27.1%（经汇率与资产组合调整），这在一定程度上抵消了上述积极影响。处方药业务不计特殊项目的息税折旧摊销前利润下降了17.2%，至10.94亿欧元。利润受到了产品组合变动的影响，具体表现为拜瑞妥®销售额的下降和诺倍戈®及艾力雅®销售额的增长，以及相关许可费用的增加。销售和研发支出亦有所上升，其中包括心血管药物Beyonttra™（acoramidis）的上市支出以及更年期症状非激素治疗药物Lynkuet™（elinzanetant）的上市前准备工作相关支出。同时，对早期研究、细胞和基因疗法以及化学蛋白质组学技术的投资增加也导致盈利有所下降。此外，因业绩优于预期，该事业部的短期绩效奖金（STI）计划支出也有所增加。相比而言，增效计划带来的成本节约以及出售非核心业务带来的收入增加促进了盈利上涨。不计特殊项目的息税折旧摊销前利润率下降了4.2%，达到24.5%。健康消费品事业部销售额与去年同期持平（经汇率与资产组合调整）自我保健产品（健康消费品事业部）的销售额增长0.2%（经汇率与资产组合调整），达到14.27亿欧元。该事业部在美国和中国面临充满挑战的市场环境，环境影响包括消费信心、门店关闭以及市场整合等情况。其中，皮肤健康品类销售额增长4.4%（经汇率与资产组合调整），抗过敏与感冒品类则在去年同期较低基数的基础上增长5.7%（经汇率与资产组合调整）。相比之下，营养品类销售额下降7.0%（经汇率与资产组合调整），主要受中国市场需求以及美国地区去年终止Care/of品牌直销业务的影响。此外，消化道健康品类销售额同比下降3.8%（经汇率与资产组合调整），主要因去年同期该品类表现得益于供应情况的正常化。健康消费品事业部不计特殊项目的息税折旧摊销前利润增长5.4%，至3.31亿欧元。盈利增长主要受益于所售商品成本的下降以及销售费用的减少，后者得益于该事业部持续的成本管理措施带来的效率提升。不计特殊项目的息税折旧摊销前利润率上涨了1.7%，达到23.2%。联系美通社 +86-10-5953 9500 info@prnasia.com

财报孤儿药

2025-08-01

·氨基观察

认知颠覆战，当Alnylam逼近4000亿

氨基观察-创新药组原创出品作者 | 武月RNAi疗法的商业奇迹，正在颠覆制药巨头的商业化霸权逻辑。Alnylam的RNAi疗法Amvuttra在针对转甲状腺蛋白介导的淀粉样心肌病（ATTR-CM）的新适应症获批后的首个季度，销售额直接起飞，达到4.92亿美元，同比增长114%，更是远超过分析师约3.65亿美元的预期。原本在市场预期中，Amvuttra与辉瑞的tafamidis疗效相差并不大，前者年治疗费用高出1倍，加上面对的还是辉瑞这一巨头。大多数人对RNAi疗法能否打破辉瑞长期主导ATTR-CM市场现状，持怀疑态度。然而，获批适应症仅一个季度，Amvuttra的表现远超预期。并且，公司在二季度财报电话会上强调，这种强势增长绝不是昙花一现，未来Amvuttra将成为ATTR-CM一线领导者。在Amvuttra的加持下，Alnylam大幅提高了全年业绩指引，将总产品收入指引由此前的20.5亿-22.5亿美元，上调至26.5亿-28亿美元。并且，商业化多年后，Alnylam终于要见到盈利曙光了。公司首席执行官Yvonne Greenstreet表示，Amvuttra对ATTR-CM的适应症获批标志着公司进入增长新阶段，并有望在今年实现“可持续的非GAAP盈利”。这样的业绩发布后，Alnylam股价应声大涨15%，市值历史首次突破500亿美金。相比短期股价波动，或许更值得关注的是，在创新的加持下，生物科技公司与制药巨头的力量对比，究竟会发生哪些微妙变化。/ 01 /超预期的三个月ATTR-CM患者也被叫做“淀粉人”。这是一种罕见且致死的心肌病，平均而言，患者在确诊后只能存活2-5年。虽然ATTR-CM属于罕见病，但是市场规模也相当可观。海外分析师预测到2030年，ATTR-CM市场规模将突破112亿美元。而此前的治疗方案极为有限，存在极大的未满足的临床需求。2024年之前，只有辉瑞的TTR蛋白稳定剂Tafamidis获批用于治疗ATTR-CM，去年的销售额达到了55亿美元。数据显示，约1/3的患者使用Tafamidis仍会发生疾病进展。不过，美国大部分医生认为鉴于Tafamidis的口服易用性，原始安全性和积累的真实世界数据，仍有望保持ATTR-CM市场的最大份额。这也被视为Amvuttra最大、甚至难以逾越的存在。然而，似乎只用了三个月，Alnylam就扭转了市场预期。在3月获批治疗ATTR-CM后，Amvuttra在今年二季度的销售额达到了4.92亿美元，同比增长114%。Amvuttra的表现，远超分析师约3.65亿美元的预期，这也带动了公司整体收入的超预期增长。根据财报，截至6月30日，已有约1400名ATTR-CM患者正在接受Amvuttra治疗，贡献了1.5亿美元收入。尽管时间较短，但强劲的增长势头，让Alnylam信心满满，大幅上调了公司全年产品收入预期：从此前的20.5亿-22.5亿美元上调至26.5亿-28亿美元。其中，TTR产品组合（包括Amvuttra和另一款RNAi疗法Onpattro，二季度销售额0.5亿美元）的收入指引，则从16亿-17.3亿美元上调至21.8亿-22.8亿美元。以中值22.3亿美元计算，TTR组合将比2024年12亿美元的销售额，增长约10亿美元。Alnylam在电话会上表示，（Amvuttra）这样的增长不仅仅是昙花一现，预计将持续可持续增长，这也是公司上调业绩指导的核心原因。按照这个势头，仅ATTR-CM这一适应症，可能用不了两年时间，就将为Alnylam贡献一个重磅炸弹。更重要的是，在公司CEO看来，Amvuttra对ATTR-CM的适应症获批标志着公司进入增长新阶段，并有望在今年实现“可持续的非GAAP盈利”。这对Alnylam意义重大。自2002年成立以来，其已成功获批4款RNAi药物，但至今尚未盈利。而二季度的表现，让市场对公司的未来盈利能力信心倍增。/ 02 /起量三件套事实上，市场早有预期，Amvuttra会为ATTR-CM二线治疗带来一定改变，但没想到，改变来得如此迅猛和全面。Tafamidis此前独占市场多年，单看主要终点等疗效数据，Amvuttra与之相差也并不大：前者的三期试验显示，治疗30个月后，与安慰剂相比，Tafamidis显著降低全因死亡率和心血管相关住院率；主要终点的单个评价终点结果证明，与安慰剂组相比，Tafamidis组的全因死亡率和心血管相关住院率分别降低了30%和32%。后者的三期试验则显示，总体患者和接受单药治疗患者（基线时未接受Tafamidis治疗的患者）分别实现了全因死亡率和复发性心血管事件综合指标28%和33%的降低；Amvuttra组也达到了关键的次要终点，总体患者的全因死亡率降低了36%，单药治疗的患者降低了35%。而Amvuttra接近50万美元的年治疗费用，却比Tafamidis25万美元左右的标价，贵上1倍。加上注射不及小分子口服的可及性高，市场认为推广Amvuttra作为一线治疗将面临极大挑战。尤其是，医生端的固有认知。尽管医生都承认Amvuttra的作用机制比稳定剂具有优势，Tafamidis通过稳定转甲状腺素蛋白起作用，vutrisiran则充当RNA沉默剂以减少致病蛋白的产生，但他们也强调，心脏病学领域已经不再过度解释生物学原理，而是专注于临床数据。起初，Alnylam也是以吸收二线患者为重点。但很快，公司发现，一线治疗起量也很快。按照电话会上的说法，两者是均衡的。言下之意，目前1400名患者中约50%是一线使用Amvuttra。那么，Amvuttra是怎么做到的？简单来说，准入先行、支付提速、获得医生认可。首先是快速进入处方目录，超预期表现驱动快速增长。截至目前，约80%的患者已经可以通过保险获得处方，而这一进展原本预计要到2025年底才能完成。同时，公司建立了完善的给药网络，确保美国约90%的患者能够在距离居住地10英里内接受治疗。其次是大多数支付方（包括联邦医疗保险按服务收费计划、联邦医疗保险优惠计划和商业保险）已将Amvuttra纳入一线首选治疗范围，这也成为销量增长的关键驱动力之一。现实中患者对Amvuttra的获取非常便捷，很少遇到来自支付方阻力，包括“分步编辑”等限制。此前市场预期，商保可能要求患者先使用Tafamidis再使用Amvuttra，但按照公司的说法，很少有商保这样做。显然，海外支付方对这种罕见病的创新疗法仍大力支持。医生的快速接纳是最后一环。自上市以来，Amvuttra的处方医生基数季度环比增加了两倍，同时，社区医生和学术医生都在使用，新处方医生和重复处方医生也在使用。根据海外机构对于ATTR-CM领域KOL的调研，他们不仅看好Amvuttra在混合表型新诊断患者中的主导地位，更认为它将是Tafamidis进展患者的首选。一线、二线双优选，从结果来看也的确如此。过去3个月，Amvuttra起初主要是圈到了使用Tafamidis后疾病进展的患者，但很快不仅成功进入一线治疗市场，还吸引了部分患者从口服稳定剂转换到Amvuttra的治疗方案。也就是，Alnylam正在抢走辉瑞的蛋糕。/ 03 /颠覆常识制药行业长期存在一个“常识”：在相对成熟的领域，后来者单纯依靠疗效优势，无论微弱抑或强烈优势，都很难轻松突破先行者根深蒂固的商业壁垒，尤其当对手是商业化实力强大的MNC。这样的案例，很常见。而ATTR-CM领域的另一个后来者BridgeBio的选择，似乎也印证了这一点。其选择了在核心产品acoramidis正式上市之前，就卖出商业化权益，落袋为安。Alnylam则选择与巨头硬刚。在其运作下，Amvuttra不仅放量迅速，还将极大挑战辉瑞在该领域独霸多年的市场地位，改写ATTR-CM市场的竞争格局。在部分深耕TTR-CM领域的KOL眼中，Amvuttra将在未来的一线治疗中占据最大份额，约为50%，其次是Tafamidis，acoramidis则占据剩余的市场份额。一位波士顿的心脏病专家指出，对于新诊断的ATTR-CM患者，特别是占40-45%的混合表型或病情更严重的患者，Amvuttra将表现出色。来自西奈山医院的卡洛斯·桑托斯-加列戈医生更是表示，他希望将60-70%的新诊断ATTR-CM患者使用Amvuttra进行治疗。他偏爱Amvuttra的理由有四点：Amvuttra对心肌病和多发性神经病的全面疗效，3月注射一次的依从性，未来Tafamidis仿制药上市后联合用药的潜力，以及其所在机构先采购后报销的处方经济优势。显然，Alnylam让我们看到了中小药企也有“打败”MNC的可能。而这不仅要求企业自身产品实力够硬，也就是机制或者疗效优势，同时更加要求，药企必须提前补齐商业化短板，策略精准、执行力也不能掉队。从Amvuttra的超预期就能看出，在市场准入以及患者支付等层面，Alnylam的团队行动力十分高效。当然，你可能会说Alnylam此前已经有比较丰富商业化经验。那么，不久前被默沙东100亿美元收购的Verona可以提供另一个视角，即在核心产品商业化之前，找到合适的人、提前做好商业化准备的重要性。Verona不仅引入了有20年商业化经验的高管，还制定了周密的策略，并高效执行。按照公司的说法，其核心策略是聚焦于高处方量的医疗服务提供者（HCPs），并重点针对高处方量的HCPs。截至2024年底，公司已完成了约120场专家教育项目。销售团队还积极参加了重要的全国和区域性肺病学会议，以支持产品上市。在可及性方面，Ensifentrine通过独家认证的专业药房网络进行分销，确保了产品在上市后的迅速可及性。除此之外，Verona还推动Ensifentrine纳入治疗指南。与Alnylam类似，Verona的COPD新药Ensifentrine上市之初，市场还在担忧，一款小分子药，如何打得过赛诺菲的度普利尤单抗、GSK的美泊利珠单抗呢？上市之后，市场发现，Ensifentrine与前者几乎无竞争，加上自身使用、疗效优势及商业化布局，放量迅速。也正因此，Verona获得了默沙东的青睐。与之相比，Alnylam选择了一条更艰难的道路——自主研发并直接挑战行业巨头。整个行业的未来竞争版图，也正在这些勇敢者的挑战中，发生着或微妙或剧烈的变化。Alnylam的经历，对已经经过多年发展，迎来阶段成果检验期的国内创新药产业来说，无疑是最大启示。PS：欢迎扫描下方二维码，添加氨基君微信号交流。

财报临床3期siRNA

2025-08-01

·BioSpace

‘Very Impressive’ Amvuttra ATTR-CM Sales Send Alnylam Soaring

iStock, Deagreez In its first commercial quarter for ATTR-cardiomyopathy, Alnylam’s Amvuttra reached roughly 1,400 patients and made more than $490 million. In its first commercial quarter in the ATTR-CM market, Alnylam’s siRNA therapy Amvuttra carried the biotech’s revenues to a comfortable beat over consensus estimates and gave the company enough confidence to substantially raise its full-year 2025 guidance. Amvuttra made $491.9 million in the quarter, a 112% increase from the same period last year. Those sales are from both of its indications: polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTR), for which Amvuttra was initially approved in 2022 , and ATTR-cardiomyopathy (CM), its newest indication that was granted in March . Some 1,400 ATTR-CM patients were on Amvuttra at the end of its first quarter of availability for the condition, according to Alnylam’s second-quarter earnings report on Thursday. Writing to investors on Thursday, analysts at Stifel called Amvuttra’s Q2 performance “very impressive,” noting that its total revenues deliver a “significant beat versus Street expectations” of $342 million. Investors also seemed impressed by Alnylam’s execution of Amvuttra’s ATTR-CM launch. The company was up more than 15% at market close. “Even in the backdrop of high Street expectations, Amvuttra performance’s here clearly aligns with the bull case and makes us more optimistic on peak sales potential,” Stifel added. BMO Capital Markets wrote in their own note late Thursday that Amvuttra’s sales “reflect strong patient demand,” given a growing prescriber base, robust uptake without price adjustments and apparent penetration of the drug into first and second line settings.Given Amvuttra’s strong launch, Alnylam on Thursday lifted its full-year sales guidance from $2.05 billion to $2.25 billion, as previously forecasted, to $2.65 billion to $2.8 billion, a 27% increase. The company also now expects the growth of its net product sales to hit 59% to 68%, year-on-year at constant exchange rates, versus its prior guidance of 26% to 39%. The biotech made $773.7 million in Q2, representing a 17% year-on-year growth and a substantial beat over the consensus revenue forecast of $651 million. In the ATTR-CM market, Alnylam is facing off against Pfizer, which owns the transthyretin stabilizer tafamidis. That drug was approved in 2019 as Vyndaqel and Vyndamax and has used its first-to-market advantage well: In 2024, Pfizer logged $5.45 billion in sales for its tafamidis products, a hefty 60% year-on-year growth. Pfizer remains confident that it can protect its slice of the ATTR-CM market, with CEO Albert Bourla telling investors at a TD Cowen event in March that as long as treatment is going well, doctors “will never switch a patient to something different.” Pfizer “will maintain a lion’s market share” for new ATTR-CM prescriptions, Bourla added, noting the pharma’s extensive network and deep understanding of physician prescription patterns. Also competing with Alnylam is BrdigeBio, which owns Attruby, another transthyretin stabilizer. Since its approval in November 2024 , Attruby had delivered 1,028 unique prescriptions as of Feb. 17. As of April 25, more than 2,000 unique patients had been treated with Attruby, resulting in $36.7 million in sales during the drug’s first commercial quarter.

上市批准生物类似药临床结果

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	Bayer AG	2025-02-10
遗传性转甲状腺素蛋白淀粉样变性	冰岛	Bayer AG	2025-02-10
遗传性转甲状腺素蛋白淀粉样变性	列支敦士登	Bayer AG	2025-02-10
遗传性转甲状腺素蛋白淀粉样变性	挪威	Bayer AG	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.	2025-05-12
多发性神经病	临床3期	保加利亚	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	法国	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	德国	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	意大利	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	马来西亚	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	荷兰	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	葡萄牙	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	新加坡	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	韩国	Eidos Therapeutics, Inc.	2025-05-12

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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 sTTR levels	557	Acoramidis	構醖願蓋蓋窪夢憲淵遞(積憲築夢鑰製構選鏇壓) = was associated with reduced ACM in univariate analysis (HR: 0.96 per 1 mg/dL increase in early ΔTTR; 95% CI: 0.93-0.98; P = 0.002). In the multivariate analysis, after adjusting for TTR variant status, baseline New York Heart Association functional class, baseline National Amyloidosis Centre stage, and baseline sTTR level, early ΔTTR remained independently associated with reduced ACM (P < 0.001). 夢壓鬱鏇衊餘憲淵蓋觸 (網窪網鑰選積襯構範衊 ) 更多	积极	2025-05-01
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	鑰製齋鑰糧蓋構醖糧積(醖獵製鑰顧夢積鹽範選): 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	構顧繭蓋鑰齋鹽蓋繭蓋(選繭憲廠艱積憲鏇膚網) = 遞衊醖願窪築築製膚壓醖鹹夢顧製積製齋餘衊 (餘觸範蓋廠齋膚艱觸鏇, 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	顧鹹鏇遞鹹膚遞醖網餘(蓋窪顧窪遞構範夢製築): 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	遞鏇鹽糧網簾齋遞網顧(積艱鑰願顧壓齋壓鏇鹽) = 願醖選鬱積餘蓋範遞壓鬱製齋壓構繭選顧鏇壓 (鏇襯觸衊鑰觸網簾願艱 ) 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	鏇積繭壓蓋膚遞壓壓願(簾鹽繭觸網齋餘築廠遞) = 鬱衊淵簾糧醖襯糧糧衊鹹窪糧艱繭淵選顧觸遞 (襯憲齋衊夢築獵遞糧鑰 )	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	網蓋顧糧蓋廠糧鏇膚廠(膚餘選膚糧餘壓窪膚衊) = 範鏇蓋蓋網衊窪憲網膚簾獵壓鑰範憲艱範艱鏇 (範獵築夢餘選網積觸鹹 )	积极	2024-08-30
				Tafamidis	網蓋顧糧蓋廠糧鏇膚廠(膚餘選膚糧餘壓窪膚衊) = 遞廠衊淵構壓願廠鹽蓋簾獵壓鑰範憲艱範艱鏇 (範獵築夢餘選網積觸鹹, 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)	繭艱顧醖顧觸鬱範糧觸 = 構鏇艱窪選繭製憲糧觸膚鑰廠醖鹹簾衊衊積簾 (襯窪襯膚鹹蓋廠壓鑰鏇, 窪願鬱鑰憲觸鏇憲顧遞 ~ 鹹獵繭餘餘淵夢齋範繭) 更多	-	2024-06-27
				Placebo Oral Tablet (Placebo)	繭艱顧醖顧觸鬱範糧觸 = 鹹構糧製廠簾餘築願鑰膚鑰廠醖鹹簾衊衊積簾 (襯窪襯膚鹹蓋廠壓鑰鏇, 壓顧築廠襯廠淵構選襯 ~ 醖構築鬱遞鏇鬱壓窪網) 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	醖淵範襯糧鹹膚鏇窪醖(鑰選顧蓋鏇築齋構觸顧) = 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) 齋積築夢鑰鏇選範觸蓋 (繭獵餘蓋壓遞衊繭淵艱 ) 更多	积极	2024-05-29
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NCT03860935 (ATTRibute-CM, ESC2024)	临床3期	NT-proBNP	-	Acoramidis	築選積獵鹹構範艱願艱(鑰鹽鏇獵淵鬱願鹽鬱積) = 廠遞鏇餘獵醖遞製獵選鏇淵製鑰廠蓋簾艱艱遞 (鹹餘壓糧積衊製獵餘簾 )	积极	2024-05-13