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2025-02-01

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.

EUCTR2020-005643-22-BE

/ Not yet 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)

开始日期2022-07-07

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）

2025-03-04·CIRCULATION

Long-Term Efficacy and Safety of Acoramidis in ATTR-CM: Initial Report From the Open-Label Extension of the ATTRibute-CM Trial

Article

作者: Maurer, Mathew S. ; Sinha, Uma ; Shah, Keyur ; Hvitfeldt Poulsen, Steen ; Soman, Prem ; Cao, Xiaofan ; Pecoraro, Maria Lucia ; Nativi-Nicolau, Jose ; Obici, Laura ; Gillmore, Julian D. ; Grodin, Justin L. ; Tamby, Jean-François ; García-Pavía, Pablo ; Judge, Daniel P. ; Cappelli, Francesco ; Fontana, Marianna ; Katz, Leonid ; Wang, Kevin ; Sarswat, Nitasha ; Grogan, Martha ; Alexander, Kevin M. ; Ambardekar, Amrut V. ; Fox, Jonathan C. ; Hanna, Mazen ; Lystig, Ted ; Masri, Ahmad

BACKGROUND::

In the phase 3 randomized controlled study ATTRibute-CM (Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy), acoramidis, a transthyretin stabilizer, demonstrated significant efficacy on the primary end point. Participants with transthyretin amyloid cardiomyopathy who completed ATTRibute-CM were invited to enroll in an open-label extension study (OLE). We report the efficacy and safety data of acoramidis in participants who completed ATTRibute-CM and enrolled in the ongoing OLE.

METHODS::

Participants who previously received acoramidis through month 30 in ATTRibute-CM continued to receive it (continuous acoramidis), and those who received placebo through month 30 were switched to acoramidis (placebo to acoramidis). Participants who received concomitant tafamidis in ATTRibute-CM were required to discontinue it to be eligible to enroll in the OLE. Clinical efficacy outcomes analyzed through month 42 included time to event for all-cause mortality (ACM) or first cardiovascular-related hospitalization (CVH), ACM alone, first CVH alone, ACM or recurrent CVH, change from baseline in NT-proBNP (N-terminal pro-B-type natriuretic peptide), 6-minute walk distance, serum transthyretin, and Kansas City Cardiomyopathy Questionnaire Overall Summary score. Safety outcomes were analyzed through month 42.

RESULTS::

Overall, 438 of 632 participants in ATTRibute-CM completed treatment, and 389 enrolled in the ongoing OLE (263 continuous acoramidis and 126 placebo to acoramidis). The hazard ratio for ACM or first CVH was 0.57 (95% CI, 0.46–0.72) at month 42 based on a stratified Cox proportional hazards model ( P <0.0001) favoring continuous acoramidis. Similar analyses were performed on ACM alone and first CVH alone, with hazard ratios of 0.64 (95% CI, 0.47–0.88) and 0.53 (95% CI, 0.41–0.69), respectively, at month 42. Treatment effects for NT-proBNP and 6-minute walk distance also favored continuous acoramidis. On initiation of open-label acoramidis in the placebo-to-acoramidis arm, there was a prompt increase in serum transthyretin. Quality of life assessed by Kansas City Cardiomyopathy Questionnaire Overall Summary score was well preserved in continuous-acoramidis participants compared with the placebo-to-acoramidis participants. No new clinically important safety issues were identified in this long-term evaluation.

CONCLUSIONS::

Early initiation and continuous use of acoramidis in the ATTRibute-CM study through month 42 of the ongoing OLE study were associated with sustained clinical benefits in a contemporary transthyretin amyloid cardiomyopathy cohort, with no clinically important safety issues newly identified.

REGISTRATION::

URL: https://www.clinicaltrials.gov ; Unique identifier: NCT04988386.

2025-03-01·CURRENT OPINION IN CARDIOLOGY

New therapies to treat cardiac amyloidosis

Review

作者: Kamna, Daniel ; Nguyen, Olives ; Masri, Ahmad

Purpose of review:

Review advancements in therapies for transthyretin (ATTR-CM) and immunoglobulin light chain (AL-CM) cardiac amyloidosis.

Recent findings:

In ATTR-CM, tafamidis remains the cornerstone therapy, with Food and Drug Administration (FDA) approval for over 5 years. Acoramidis, another transthyretin stabilizer, has very recently been FDA-approved following positive results in the ATTRibute-CM trial. Vutrisiran, a transthyretin gene silencer, demonstrated efficacy in the HELIOS-B trial and awaits FDA review. Eplontersen's CARDIO-TTRansform trial, the largest ATTR-CM study to date, is expected to report by late 2025. Innovative approaches such as NTLA-2001 (a CRISPR-Cas9 therapy) and fibril depleters like ALXN2220 and coramitug are advancing in clinical trials. In AL-CM, daratumumab, cyclophosphamide, bortezomib, and dexamethasone (Dara-CyBorD) has established itself as the standard of care. Novel antiplasma cell therapies include CAR-T cells and bispecific antibodies (teclistimab) and fibril depleters. Birtamimab improved survival in advanced AL-CM during the VITAL trial and is under investigation in AFFIRM-AL. Anselamimab is in phase III CARES trials, whereas AT-02 undergoes early-phase testing for ATTR-CM and AL-CM.

Summary:

The therapeutic landscape for ATTR-CM and AL-CM is rapidly evolving, driven by novel therapies targeting diverse mechanisms. Ongoing clinical trials promise to further refine the standard of care and improve outcomes for patients with cardiac amyloidosis.

2025-02-01·PHARMACOTHERAPY

Disease‐modifying therapies for amyloid transthyretin cardiomyopathy: Current and emerging medications

Review

作者: DiDomenico, Robert J. ; Krishna, Hema ; Rodriguez‐Ziccardi, Mary C. ; Hellenbart, Erika L. ; Ipema, Heather J.

Abstract:

Transthyretin amyloidosis (ATTR) is a rare disease that results in amyloid fibril misfolding and deposition in multiple organs, including the heart, leading to the development of ATTR cardiomyopathy (ATTR‐CM), which is associated with poor outcomes. In the last decade, several disease‐modifying medications are in advanced stages of clinical development or have been approved to treat ATTR‐CM. The purpose of this review is to critically evaluate clinical trial data investigating the use of approved and investigational medications for the treatment of ATTR‐CM. We performed a comprehensive literature search via PubMed and EMBASE to identify randomized controlled trials evaluating medications for the treatment of ATTR‐CM published through August 2024. This narrative review describes the pathophysiology of ATTR‐CM, highlights important screening and diagnostic work‐up, and summarizes the existing clinical evidence resulting from our literature search. Several classes of disease‐modifying medications are in development for ATTR‐CM. The tetramer stabilizers and transthyretin silencers have proven to be the most effective therapies to date. Tafamidis and acoramidis are currently approved for ATTR‐CM while vutrisiran approval for ATTR‐CM may be forthcoming. Other disease‐modifying medication classes in development include antisense oligonucleotides, gene editing therapies, and monoclonal antibodies. However, several unmet needs exist including the lack of cost‐effectiveness due to the extremely high acquisition costs of these medications. Disease‐modifying medications approved and in development to treat ATTR‐CM offer hope for patients with this disease, but their lack of affordability is the biggest barrier to their use.

235

项与 Acoramidis 相关的新闻（医药）

2025-03-21

·EndPoints

Alnylam hopes its broad ATTR-CM label can give Amvuttra an edge in heart disease

Alnylam will likely face challenges in turning a profit this year, despite executives promising to do so. But the label for its newly-approved Amvuttra indication may give it an advantage over its rivals Pfizer and BridgeBio. That’s because Amvuttra’s label for transthyretin amyloid cardiomyopathy, or ATTR-CM, is broader than its competitors, saying it reduces the likelihood of hospitalization, death and urgent heart failure visits. Pfizer’s Vyndaqel/Vyndamax franchise, approved in 2019, and BridgeBio’s recently-approved Attruby are only indicated to reduce hospitalization and death. “The inclusion of urgent heart failure visits is unique and recognizes the effects that we’re also seeing on this important consequence of ATTR-cardiomyopathy,” Alnylam chief medical officer Pushkal Garg said on an investor call Thursday evening. Garg touted the broader label as key to getting Amvuttra in as many first-line patients as possible. Wall Street appeared to like what it saw as well after the FDA approved the drug for the rare heart disease. After a trading halt Thursday afternoon, Alnylam’s stock $ALNY was up more than 10% in Friday’s session. Most analysts expect getting Amvuttra into first-line patients will determine whether Alnylam is profitable this year, and they don’t think it will be easy. Both Vyndaqel/Vyndamax and Attruby are oral stabilizers, a class that’s been on the market for several years that works by stabilizing the TTR protein and preventing misfolding. Amvuttra, meanwhile, is the first therapy approved in its class for ATTR-CM, reducing the expression of the TTR gene through RNA interference. There’s also a wrinkle when it comes to second-line patients who could switch from the Pfizer and BridgeBio drugs to Amvuttra: There is no formal definition for ATTR-CM disease progression. The decisions that patients and their doctors make about which therapy to take — one of the Pfizer/BridgeBio stabilizers, Alnylam’s silencer or both mechanisms in combination — will be made on a case-by-case basis. In an interview before Thursday’s approval, CEO Yvonne Greenstreet told Endpoints News the company can support a successful launch in ATTR-CM by leveraging its existing physician network for doctors who prescribe Amvuttra in its other indication, hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy. She also said she believes Alnylam’s clinical trial data will make patients’ choices easier, regardless of whether they’ve taken a stabilizer before. “The simpler it is for physicians, the easier it is for them to come to a determination,” Greenstreet told Endpoints. “But I have to say that, even without specific, clear guidelines with, let’s say, one biomarker to assess, physicians are able to interpret whether their patients are progressing or not.” The company is forecasting revenues between $1.6 billion and $1.725 billion for its ATTR medicines (Amvuttra and Onpattro) this year after they made about $1.2 billion in 2024, giving them what Greenstreet has called a “path to profitability.” Alnylam lost $278 million last year. Alnylam is maintaining the same price of about $476,000 per year for Amvuttra in ATTR-CM as it had for hATTR. It’s nearly double what Pfizer and BridgeBio charge for their drugs, at around $250,000 and $244,500, respectively. Despite that, analysts largely saw the label as a win for Alnylam in notes out to investors Thursday evening and Friday morning. TD Cowen’s Ritu Baral wrote the inclusion of urgent heart failure visits will help in payer talks, “especially for front line use,” as ATTR-CM care has moved into the outpatient setting. With the pricing information confirmed, Baral estimates $254 million in ATTR-CM sales and peak sales of $2.3 billion by 2030. Stifel analyst Paul Matteis, meanwhile, wrote that he believes investor fixation on label language was overblown, questioning whether it would have an impact on patient uptake. But the fact that Alnylam was able to deliver on a differentiated label is still a success. “How much this will matter remains open for debate, but on the margin it could help with the marketing message and it also may be helpful with [Alnylam’s] ability to maintain premium pricing,” Matteis wrote. That high price may make some payers balk in insurance discussions, Leerink analyst Mani Foroohar noted. Most of the patient population is covered under Medicare Part B, however, giving doctors different prescribing incentives. Greenstreet said that about 70% of patients taking Amvuttra for hATTR pay no out-of-pocket costs, and she expects Alnylam to eventually get to about that level in ATTR-CM as well. That dynamic, when combined with the data and label, are all the pieces Alnylam needs to put together.

上市批准

2025-03-21

·医药魔方Info

首款RNAi疗法获FDA批准治疗ATTR淀粉样变性心肌病

3月20日，Alnylam Pharmaceuticals宣布其RNAi疗法Amvuttra（Vutrisiran）获批用于治疗ATTR淀粉样变性心肌病（ATTR-CM）。这是ATTR-CM领域首款RNAi疗法。 FDA此次批准主要是基于III期HELIOS-B研究的积极结果。该研究是一项多中心、随机、双盲、安慰剂对照临床试验（n=654），评估了Vutrisiran（25mg，每3个月1次，皮下注射）在ATTR-CM患者中的有效性和安全性。研究的主要终点是第36个月全人群的全因死亡和复发心血管（CV）事件（CV住院治疗和紧急心力衰竭就诊）的复合发生率。结果显示，在双盲期间，总人群的全因死亡和复发CV事件的复合发生率显著降低（HR=0.718，p=0.0118）；单药治疗人群（基线时未接受氯苯唑酸治疗，N=395）的全因死亡和复发CV事件的复合发生率亦显著降低（HR=0.672，p=0.0162）。此外，在总人群和单药治疗人群中，所有次要终点均实现了具有统计学意义的改善。包括疾病进展的关键测量结果：6分钟步行测试（6-MWT），堪萨斯城心肌病问卷调查（KCCQ）和第30个月纽约心功能分级（NYHA），p均小于0.025。重要的是，到第42个月，Vutrisiran还降低了总人群（HR=0.645，p<0.025）和单药治疗人群的全因死亡率（HR=0.655，p<0.05）。在所有关键亚组中，Vutrisiran对主要复合终点和所有次要终点均显示出一致的效果，包括基线Vutrisiran使用、ATTR疾病类型和疾病严重程度的测量。在HELIOS-B研究中，Vutrisiran表现出令人鼓舞的安全性和耐受性。Vutrisiran组和安慰剂组的不良事件（AE）、严重AE和因AE导致研究药物停药的发生率相似。 Vutrisiran是一种双链小干扰RNA（siRNA），靶向突变型和野生型转甲状腺素蛋白（TTR）信使RNA。Amvuttra使用Alnylam的增强稳定性化学（ESC）-GalNAc结合物递送平台，具有更高的效力和高代谢稳定性。2022年6月，Vutrisiran首次在美国获批上市，用于治疗成人ATTR淀粉样变性多发性神经病变。 ATTR-CM是一种毁灭性的、快速进展的、最终致命的疾病。据估计，美国和全球分别约有15万例和超过30万例ATTR-CM患者。目前，全球仅3款药物获批用于治疗该疾病，包括氯苯唑酸（辉瑞）、Acoramidis（拜耳/阿斯利康/BridgeBio Pharma）和Vutrisiran。 Copyright © 2025 PHARMCUBE. All Rights Reserved. 欢迎转发分享及合理引用，引用时请在显要位置标明文章来源；如需转载，请给微信公众号后台留言或发送消息，并注明公众号名称及ID。免责申明：本微信文章中的信息仅供一般参考之用，不可直接作为决策内容，医药魔方不对任何主体因使用本文内容而导致的任何损失承担责任。

临床3期siRNA临床结果上市批准

2025-03-21

·MedCity News

Alnylam Drug Approval Brings Cardiomyopathy Competition to Pfizer, BridgeBio Pharma - MedCity News

Alnylam Pharmaceuticals has landed a long-awaited FDA decision that expands use of its drug, Amvuttra, to the treatment of a type of cardiomyopathy that’s growing in prevalence. It’s the second FDA approval in this indication in four months and the third overall, introducing new competition to the treatment of a disease that leads to heart failure and frequent hospitalizations, often becoming fatal within five years of diagnosis. Amvuttra is now approved for treating adults who develop cardiomyopathy from transthyretin amyloidosis (ATTR), a disease in which abnormal versions of the liver protein transthyretin (TTR) lead to buildup of amyloid proteins in body tissue. The regulatory decision announced late Thursday covers ATTR cardiomyopathy that develops naturally as well as disease that is hereditary. Amvuttra is the third medication approved for ATTR cardiomyopathy, behind products from Pfizer that have become the standard of care and a BridgeBio Pharma drug approved last fall. But Amvuttra introduces a new mechanism of action to the disease. Cambridge, Massachusetts-based Alnylam specializes in RNA interference drugs, therapies that deliver small pieces of RNA to “silence” a gene, knocking down production of a disease-causing protein. Amvuttra is designed to lower levels of the TTR proteins that drive ATTR. The drug, administered as an injection every three months, was first approved in 2022 as a treatment for polyneuropathy caused by the disease. presented by Health Tech The 5 Best Software to Buy for Managing a Spa Franchise Choosing the right software translates directly into better client management, operational efficiency and growth for your spa franchise. Discover which tool is best for your business. By Meevo FDA approval of Amvuttra in ATTR cardiomyopathy is based on data from a placebo-controlled Phase 3 study, HELIOS-B, whose main goal was measuring death from all causes and recurrent cardiovascular events. The results on this composite endpoint showed Amvuttra achieved the statistically significant 28% reduction during the treatment period of up to 36 months. On a secondary goal measuring mortality, results showed a 36% reduction through 42 months. Results were published last August in the New England Journal of Medicine. “We believe Amvuttra has the potential to become a new standard of care in ATTR cardiomyopathy,” Alnylam CEO Yvonne Greenstreet said, speaking during a Thursday evening conference call. “Our confidence stems from the success of the HELIOS-B study, which demonstrated a therapeutic profile for Amvuttra that is supportive of first-line potential.” Pfizer won the first FDA approvals in ATTR cardiomyopathy in 2019. Its drug, tafamidis, marketed as Vyndaqel (a four-times-a-day capsule) and Vyndamax (a once-daily capsule), binds to mutant TTR protein, stabilizing it in order to prevent the protein buildup characteristic of ATTR. Though this Pfizer small molecule is approved in other countries for treating ATTR polyneuropathy, its U.S. approval only covers cardiomyopathy caused by the disease. Pfizer reported $5.5 billion in global sales for the Vyndaqel family of products in 2024, a 65% increase over the prior year. Most of the product’s revenue comes from the U.S. FDA approval of BridgeBio drug Attruby last November brings new cardiomyopathy competition. The twice-daily pill won European regulatory approval last month. Like the Pfizer drugs, Attruby is a TTR stabilizer. But BridgeBio contends its drug is the better stabilizer, with product label language stating the pill achieves “near-complete stabilization” of the target protein. presented by Artificial Intelligence Why Your Patients Are Leaving and How to Stop It Patient expectations continue to evolve, and healthcare practices that fail to adapt risk more than just revenue—they risk losing trust. By Bryan Sequeira, Director, Product Management Greenway Health Acknowledging the caveats that come with cross-trial comparisons, some differences could affect how prescribers and patients view these therapies. The reduction in cardiovascular hospitalizations and urgent heart failure visits with Amvuttra were well below what Attruby showed in its trial, Leerink Partners analyst Mani Foroohar wrote in a note sent to investors Friday. But William Blair’s Myles Minter highlights the Alnylam drug’s data on all-cause mortality as an edge over Attruby, which did not achieve statistical significance on this measure in its pivotal study. The label of Pfizer’s tafamidis includes a mortality benefit. “Given [all-cause mortality] claims and the totality of data in Amvuttra’s label, which derives from a patient population of whom some were on background tafamidis treatment, we see Amvuttra as a best-in-class option for patients and healthcare providers,” Minter said. Minter added that subgroup analyses show Amvuttra use was favored across patient populations, including younger and healthier patients that reflect the current disease population. William Blair believes the Alnylam drug is well positioned for first-line use in these patients. One headwind for Amvuttra is its price. For ATTR cardiomyopathy, Alnylam said Amvuttra will carry the same wholesale price as it does in ATTR polyneuropathy, which is about $119,000 per injection or $476,000 annually. In an investor presentation, the company said the product’s net price will decrease over time as uptake of the drug increases. But for now, patients will have less expensive options with Pfizer’s ATTR cardiomyopathy drugs, which carry a $267,987 annual list price, and BridgeBio’s Attruby priced at $244,500 a year. With a significantly higher list price, Leerink analysts expect challenges getting payers to cover Amvuttra in the new indication. But William Blair analysts see Amvuttra’s label as strong enough to secure broad payer coverage. The firm also sees quarterly injections as an advantage because this dosing regimen boosts compliance compared to needing to remember to take a pill every day. Amvuttra’s approval in cardiomyopathy offers the opportunity to address a much bigger market than polyneuropathy, one that Alnylam estimates is 300,000 patients globally. Furthermore, most of these patients undiagnosed, offering opportunity the company the opportunity to reach new patients. In 2024, Alnylam reported Amvuttra accounted for $970.5 million in revenue in the polyneuropathy indication. With the new approval, the company expects $1.6 billion to $1.725 billion in TTR product revenue this year. Though Amvuttra is already available for polyneuropathy, Alnylam is projecting a second-half 2025 launch for the drug as the company continues discussions with payers on value-based agreements for Amvuttra and secures formulary placement for the drug in its new indication. Amvuttra is still under review in other countries. Alnylam expects a regulatory decision in Japan in the second quarter of this year; additional decisions in Europe, Brazil, and Canada are expected later this year. In the nearer term, Alnylam is set to present additional Amvuttra data during the American Cardiology Conference in Chicago at the end of March. Photo by Alnylam Pharmaceuticals

临床结果上市批准临床3期

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

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10 条最早获批的记录,

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适应症	国家/地区	公司	日期
转甲状腺素运载蛋白淀粉样变性	欧盟	BridgeBio Pharma, Inc.	2025-02-10
转甲状腺素运载蛋白淀粉样变性	冰岛	BridgeBio Pharma, Inc.	2025-02-10
转甲状腺素运载蛋白淀粉样变性	列支敦士登	BridgeBio Pharma, Inc.	2025-02-10
转甲状腺素运载蛋白淀粉样变性	挪威	BridgeBio Pharma, Inc.	2025-02-10
甲状腺素运载蛋白淀粉样变心肌病	美国	BridgeBio Pharma, Inc.	2024-11-22

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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.	2025-02-01
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2期	美国	Eidos Therapeutics, Inc.	2018-04-27
淀粉样变性	临床1期	英国	Eidos Therapeutics, Inc.	2021-03-28

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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


NCT04622046 (CTgov)	临床3期	甲状腺素运载蛋白淀粉样变心肌病	25	ALXN2060	醖構積餘糧製範構糧鹽(衊積鬱齋窪淵簾鹽蓋醖) = 鏇憲蓋艱遞艱膚夢膚觸繭壓艱蓋觸夢遞鹽積築 (繭選網鏇鹽簾遞積餘糧, 9.182) 更多	-	2024-12-16
NCT03860935 (FDA_CDER) 人工标引	临床3期	甲状腺素运载蛋白淀粉样变心肌病	611	ATTRUBY 712 mg	構糧齋廠積齋齋築鑰鬱(餘廠糧齋繭鏇夢構壓繭): 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	甲状腺素运载蛋白淀粉样变心肌病	389	Acoramidis	積積繭願願艱艱夢艱網(蓋膚壓範鏇壓製網鑰網) = 鬱膚廠壓獵廠糧構艱遞繭鹹廠壓遞淵糧膚衊網 (簾觸構鏇鹹艱選鏇醖餘 ) 更多	积极	2024-11-18
				Placebo	-
NCT03860935 (ATTRibute-CM, GlobeNewswire \| PipelineReview) 人工标引	临床3期	甲状腺素运载蛋白淀粉样变心肌病	-	Acoramidis	夢繭餘構壓餘鏇齋鹽觸(獵觸鹹蓋製艱壓獵鹹壓) = 繭廠艱積繭鏇顧餘願淵糧遞鬱淵願壓壓鑰襯醖 (網艱蓋蓋憲醖鑰築選簾 )	积极	2024-09-27
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NCT03860935 (ATTRibute-CM, ESC2024)	临床3期	甲状腺素运载蛋白淀粉样变心肌病 serum TTR levels	611	Acoramidis	積製鑰顧鑰築餘鹹簾鹹(蓋衊鏇壓餘簾廠遞構醖) = 繭窪鹹鹽淵餘簾壓鬱鏇艱願鏇築網醖簾鏇鹽窪 (鬱廠鹽積襯願積獵鏇蓋 )	积极	2024-08-30
				Tafamidis	積製鑰顧鑰築餘鹹簾鹹(蓋衊鏇壓餘簾廠遞構醖) = 齋簾獵鏇壓鬱衊衊淵製艱願鏇築網醖簾鏇鹽窪 (鬱廠鹽積襯願積獵鏇蓋, 0.4 (4.92))
NCT03860935 (ATTRibute-CM, CTgov)	临床3期	转甲状腺素运载蛋白淀粉样变性 \| 甲状腺素运载蛋白淀粉样变心肌病	632	acoramidis (Acoramidis HCl 800 mg)	艱糧範範鏇遞糧遞繭齋 = 顧廠艱繭壓餘廠廠蓋艱製網鬱鬱衊觸鹽獵壓壓 (範衊鹹鬱鏇製網蓋廠鹽, 蓋糧艱簾餘願廠襯壓遞 ~ 鏇壓淵壓鏇夢糧憲蓋夢) 更多	-	2024-06-27
				Placebo Oral Tablet (Placebo)	艱糧範範鏇遞糧遞繭齋 = 鏇積積齋齋遞廠憲遞顧製網鬱鬱衊觸鹽獵壓壓 (範衊鹹鬱鏇製網蓋廠鹽, 鑰鏇襯醖衊艱廠範願鑰 ~ 餘鹽齋選鬱餘夢顧膚願) 更多
NCT03860935 (ATTRibute-CM, Company_Website) 人工标引	临床3期	甲状腺素运载蛋白淀粉样变心肌病	-	Acoramidis	廠鬱獵夢淵醖衊選窪衊(範膚衊窪獵廠壓廠壓膚) = 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) 範襯齋鹽製齋築鹽積壓 (糧壓獵製遞壓網遞願積 ) 更多	积极	2024-05-29
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ESC2024	N/A	甲状腺素运载蛋白淀粉样变心肌病	-	Acoramidis	築齋壓襯積鏇選艱網壓(積窪窪築積選淵壓餘鹽) = 選網鹽鹽觸壓顧願鑰遞簾選憲餘簾蓋觸選膚壓 (蓋膚網襯齋醖範築壓願, 0.07 ~ 0.18)	-	2024-05-13
ESC2024	N/A	甲状腺素运载蛋白淀粉样变心肌病	-	Acoramidis	網積襯窪製蓋遞憲襯繭(簾觸襯鏇選築蓋襯膚餘) = A statistically significant (p<0.0001) and clinically meaningful treatment benefit on KCCQ-OS was observed with acoramidis versus placebo 齋願築築簾製鹽鏇製鏇 (廠壓築鹹網獵蓋餘遞糧 ) 更多	-	2024-05-13
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NCT03860935 (ATTRibute-CM, ESC2024)	临床3期	NT-proBNP	-	Acoramidis	艱簾壓憲簾網糧窪齋鹹(範夢積築簾壓衊夢齋製) = 齋鑰遞糧範憲鬱鹽餘製鏇壓選醖製獵構製醖襯 (膚觸齋構遞夢蓋艱構製 )	积极	2024-05-13