A Prospective, Single-arm, Phase 4 Study to Evaluate the Course of Serum Transthyretin (TTR) Level With Acoramidis in Adult Patients With Variant or Wild-type Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM) Previously Treated With Tafamidis

Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid. TTR amyloid gets deposited in the heart wall and stops it from pumping blood properly, which may lead to heart failure. The breakage in TTR protein can be age-related (wild-type ATTR-CM), or genetic (variant ATTR-CM).
The study drug, acoramidis, works by attaching itself to the TTR protein, making TTR more stable and less likely to break down and form amyloid (clumps). This helps to slow down the progression of the disease, improve heart function, and increase the TTR levels in the blood. Acoramidis is an approved treatment for wild-type or variant ATTR-CM in Europe and the United States.
Tafamidis is another drug that stabilizes TTR and prevents it from breaking down.
In this study, acoramidis will be studied in participants with ATTR--CM who were previously treated with tafamidis. The main purpose of this study is to assess the change in blood TTR levels after participants are switched from tafamidis to acoramidis. This will be studied to understand if acoramidis causes an increase in blood TTR levels beyond the levels achieved with tafamidis.
For this, the researchers will measure the change in the levels of TTR protein in participants' blood after 6 months of the treatment with acoramidis, or earlier if a participant stops the treatment before reaching that six-month mark.
All participants will continue taking tafamidis during the screening period. In the treatment period of the study, participants will take acoramidis as two tablets twice daily by mouth, for up to 6 months.
At the start of this study, the study doctors will review each participant's medical history and check their overall health. The study doctors will perform electrocardiograms (ECG), and measure blood pressure and heart rate. Researchers will also take blood and urine samples from the participants to measure levels of TTr, NT-proBNP, hs-TnT, hs-CRP, RBP4, eGFR, creatinine, cystatin-C, UACR, and TSH at the start of the study, and at various time points thereafter (during the study) to assess heart, kidney and thyroid function.
There will be a total of 9 study check-ins. Participants will visit the study site twice: at screening and at the end of treatment period. A study nurse will visit the participant's home 6 times, at the start of treatment, Weeks 1, 2, 3 and 4, then again at 3 months. The final check-in will be done by phone.
The study doctors will monitor the health of the participants regularly for any medical problems during follow-up visits. Participants will know the treatment they will receive during the study. Each participant could be in the study for about 8 months.

开始日期2026-02-13

申办/合作机构

Bayer AG

NCT07116473

/ Not yet recruiting临床3期

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.

NCT07235462

/ RecruitingN/A

ACO-REAL - A Non-interventional Study Providing Insights Into the Use of Acoramidis in Patients With ATTR Amyloidosis With Cardiomyopathy (ATTR-CM) in Routine Clinical Practice

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a serious and life-threatening condition where a protein called transthyretin (TTR) misfolds and builds up as amyloid fibrils in the heart muscle. This buildup causes the heart to become stiff, leading to restrictive cardiomyopathy and progressive heart failure. There are two forms of ATTR-CM: a hereditary or 'variant' form (vATTR-CM) caused by a gene mutation, and a 'wild-type' form (wtATTR-CM) which is associated with aging. Because its symptoms can be similar to other heart conditions, ATTR-CM is often diagnosed late. However, recent advances in medical imaging are helping doctors to identify the disease earlier. Acoramidis is a new medication designed to treat ATTR-CM. It works by stabilizing the TTR protein, preventing it from misfolding and forming the harmful amyloid deposits. Acoramidis has been shown to be effective and safe in a major clinical trial (the ATTRibute-CM study), which led to its approval for use in both the United States and Europe. While clinical trials provide valuable information, data on how a new medicine performs in everyday clinical practice is also very important. This type of information is called real-world evidence. Currently, there is limited real-world information about the use of acoramidis. This study, called ACO-REAL, is an observational study, which means researchers will observe patients who are receiving acoramidis as part of their normal clinical care, without introducing any experimental interventions. The study will take place in approximately 20 European countries and aims to enroll up to 2,000 adults who have been diagnosed with either wild-type or variant ATTR-CM and are starting treatment with acoramidis. This includes patients who have not been treated for ATTR-CM before, as well as those who have been treated with other therapies. The main goals of the study are to understand the characteristics of patients being treated with acoramidis and to document how the treatment is used in routine medical practice. The study will also collect information on the safety of acoramidis. Furthermore, researchers will assess how the treatment affects patients' heart function, their functional capacity (such as their ability to walk), their overall health status, and their quality of life. The study will also track how often patients need to use healthcare resources like hospitals or emergency rooms. This information will help to improve the understanding and management of ATTR-CM in a real-world setting, ultimately aiming to optimize care for patients with this progressive disease.

开始日期2025-10-29

申办/合作机构

Bayer AG

100 项与 Acoramidis 相关的临床结果

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100 项与 Acoramidis 相关的专利（医药）

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100

项与 Acoramidis 相关的文献（医药）

2026-06-01·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications

Review

作者: Owens, Katie ; Argon, Sophie ; Yu, Jingjing ; Ragueneau-Majlessi, Isabelle ; Wang, Yan

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

2026-05-08·Circulation reports

Efficacy of Acoramidis in Adults With Transthyretin Amyloid Cardiomyopathy　― Propensity Score Comparison Between a Single-Arm Open-Label Phase 3 Trial in Japan and a Global Double-Blind Phase 3 Trial ―

Article

作者: Tahara, Nobuhiro ; Chen, Yi ; Minamisawa, Masatoshi ; Mercuri, Michele F. ; Raizner, Aileen

Background:

Acoramidis is an oral, highly selective transthyretin (TTR) stabilizer approved for transthyretin amyloid cardiomyopathy (ATTR-CM). This analysis compared efficacy outcomes over a 30-month period between acoramidis-treated patients from the Phase 3 Japan Study (NCT04622046) with propensity score (PS)-matched placebo patients from the global ATTRibute-CM trial (NCT03860935).

Methods and Results:

In the modified intention-to-treat population, PS matching using an optimal 1 : 2 matching ratio (absolute standardized mean difference ≤0.25 for all covariates) was used to match 25 acoramidis-treated Japanese patients with 50 placebo-treated patients from ATTRibute-CM. Baseline covariates for PS matching were age, sex, body mass index, New York Heart Association class, ATTR-CM genetic status, N-terminal pro-B-type natriuretic peptide (NT-proBNP) level, and 6-min walk distance (6MWD). There were no deaths reported with acoramidis over 30 months, compared with 24% all-cause mortality events among PS-matched placebo patients. There was an 81% reduction in the annualized frequency of cardiovascular-related hospitalizations with acoramidis compared with the PS-matched placebo group. The degree of worsening in 6MWD, Kansas City Cardiomyopathy Questionnaire overall score, and NT-proBNP was less with acoramidis than with placebo. Acoramidis produced a sustained increase in TTR levels and median TTR stabilization assessed using a fluorescent probe exclusion assay.

Conclusions:

Clinically meaningful benefits were observed favoring acoramidis over placebo (ATTRibute-CM trial) in survival, cardiovascular-related hospitalizations, functional endpoints, and quality of life assessments.

2026-05-08·Circulation reports

Efficacy and Safety of Acoramidis in Japanese Adults With Symptomatic Transthyretin Amyloid Cardiomyopathy

Article

作者: Ishii, Shunsuke ; Okumura, Takahiro ; Kitaoka, Hiroaki ; Sekijima, Yoshiki ; Ando, Yukio ; Otsuka, Yasushi ; Fukata, Mitsuhiro ; Yano, Toshiyuki ; Raizner, Aileen ; Minamisawa, Masatoshi ; Hashimoto, Toru ; Tsujita, Kenichi ; Izumi, Chisato ; Mercuri, Michele F. ; Tahara, Nobuhiro ; Chen, Yi ; Minamino, Tohru ; Endo, Jin

Background:

This multicenter open-label single-arm 2-part prospective Phase 3 trial (NCT04622046) evaluated the efficacy, safety, pharmacokinetics, and pharmacodynamics of acoramidis, an oral, highly selective transthyretin (TTR) stabilizer, in Japanese patients with wild-type or hereditary symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM).

Methods and Results:

Twenty-five patients (mean [±SD] age 76.5±6.3 years; 88% men) received acoramidis hydrochloride (800 mg twice daily) for up to 30 months. Primary endpoints were change from baseline to Month 12 in the 6-min walk distance (6MWD) and all-cause mortality and cardiovascular-related hospitalizations over 30 months. No deaths were reported over 30 months. The annualized frequency of cardiovascular-related hospitalizations was 0.133. Clinically meaningful treatment benefits were observed in least-squares mean change from baseline to Month 12 for 6MWD (-3.9 m; 95% confidence interval [CI] -22.9, 15.1 m) and to Month 30 for 6MWD (-36.2 m; 95% CI -58.5 to -13.9 m), Kansas City Cardiomyopathy Questionnaire overall score (-7.0; 95% CI -14.7, 0.8), TTR levels (9.4 mg/dL; 95% CI 6.7, 12.0 mg/dL), and median N-terminal pro B-type natriuretic peptide levels (169.5 pg/mL; interquartile range -557.0, 700.0 pg/mL). The median TTR stabilization (fluorescent probe exclusion assay) was 95.6%. Acoramidis was well tolerated.

Conclusions:

Acoramidis prolongs survival, provides functional and quality-of-life benefits, and is well tolerated in Japanese patients with ATTR-CM, consistent with results from the global ATTRibute-CM Phase 3 trial.

365

项与 Acoramidis 相关的新闻（医药）

2026-06-11

·PRNewswire

DKSH Enters Strategic Distribution Partnership with BridgeBio to Support Regulatory Evaluation and Potential Patient Access to Transthyretin Stabilizer for ATTR-CM in Selected Asia Pacific Markets

SINGAPORE, June 11, 2026 /PRNewswire/ -- DKSH entered a strategic distribution partnership with BridgeBio to support regulatory evaluation and potential patient access to a Transthyretin Stabilizer for ATTR-CM in Australia, Singapore, South Korea, and Taiwan. The partnership combines BridgeBio's biotech innovation and DKSH Healthcare's integrated end-to-end commercialization platform to bring a new treatment option to patients. DKSH Business Unit Healthcare, a strategic healthcare business partner and leading provider of Market Expansion Services for pharmaceutical, over-the-counter (OTC), consumer health and medical device companies, has entered a distribution partnership with BridgeBio, a biotechnology company focused on genetic diseases. Under the terms of the agreement, DKSH will be responsible for the distribution and commercialization of the transthyretin stabilizer for ATTR-CM across Australia, Singapore, South Korea, and Taiwan. Where approved, the product is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization1. ATTR-CM is a progressive, debilitating disease that diminishes quality of life, causes recurrent hospitalizations, and leads to premature death. This new treatment is not currently approved in the countries covered by this agreement and any commercialization will be subject to applicable regulatory approvals and local regulations. DKSH will provide end-to-end solutions prioritizing healthcare access to patients, leveraging its expertise in regulatory affairs, medical affairs, market access, and launch excellence, as well as its robust distribution infrastructure and integrated commercial capabilities. DKSH is well positioned to support the introduction of this biotech innovation across the Territory following regulatory approvals.2 3 4 "Our strategic partnership with BridgeBio represents an important milestone as DKSH Healthcare enters a new chapter with a renewed vision for growth. United by a shared commitment to improve outcomes for underserved patients, we are proud to combine BridgeBio's scientific innovation with DKSH Healthcare's industry‑leading, end‑to‑end commercialization platform. Leveraging our deep market expertise, strong capabilities, and trusted execution across complex healthcare systems, we are well positioned to launch this biotech innovation and expand access to important new treatment options reliably, responsibly, and at scale, advancing on our mission to deliver Healthcare for All," said Patrik Grande, Global Head of Business Unit Healthcare, DKSH. 1 FDA, Full Prescribing Information. Available at: Last accessed March 2026. 2 Lane T, et al. Circulation. 2019;140(1):16-26. 3 Rintell D, et al. Orphanet J Rare Dis. 2021;16(1):70. 4 Nativi-Nicolau J, et al. ESC Heart Fail. 2021;8(5):3875-3884 About DKSH For more than 160 years, DKSH has been delivering growth for companies in Asia Pacific, Europe, and North America across its Business Units Healthcare, Consumer Goods, Performance Materials, and Technology. As a leading Market Expansion Services provider, DKSH offers sourcing, market insights, marketing and sales, e-commerce, distribution and logistics as well as after-sales services, following its purpose of enriching people's lives. DKSH is a participant of the United Nations Global Compact and adheres to its principles-based approach to responsible business. Listed on the SIX Swiss Exchange, DKSH operates in 35 markets with 26,840 specialists, generating net sales of CHF 11.1 billion in 2025. DKSH Business Unit Healthcare distributes pharmaceuticals, consumer health, over-the-counter products and medical devices. With around 7,580 specialists, the Business Unit generated net sales of CHF 5.8 billion in 2025. For more information please contact: DKSH Holding Ltd. Till Leisner Head, Investor & Media Relations, Sustainability Phone +41 44 386 7315 [email protected] Melanie Grüter Manager, Group Media Relations Phone +41 44 386 7211 [email protected] SOURCE DKSH Management Ltd. 21% more press release views with Request a Demo

引进/卖出

2026-06-11

·allsci.com

BridgeBio partners with DKSH to distribute acoramidis across four Asia Pacific markets

Switzerland-based DKSH Management Ltd. has entered a distribution partnership with BridgeBio Pharma (Nasdaq: BBIO) to support regulatory filings and, subject to approval, commercialization of acoramidis (Attruby/Beyonttra) for the treatment of cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in Australia, Singapore, South Korea, and Taiwan. The arrangement covers an oral small molecule that has already secured approvals in the US, EU, UK, Japan, Switzerland, and Brazil, but remains unapproved across the four covered markets. Financial terms were not disclosed. Acoramidis is a selective oral TTR stabilizer described by BridgeBio as achieving near-complete (≥90%) stabilization of the transthyretin tetramer, a mechanism that prevents the protein from misfolding and depositing as amyloid in the heart. The drug is approved for adults with wild-type or variant ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization. The Phase III ATTRibute-CM study underpins the approvals secured to date. Long-term open-label extension data published in March 2026 showed a statistically significant 44.7% reduction in all-cause mortality and 49.3% reduction in cardiovascular mortality at Month 54 versus patients who had received placebo before crossing over. Under the deal, DKSH will handle regulatory affairs, medical affairs, market access, and distribution across the four territories. BridgeBio reported Q1 2026 US net product revenue of USD 180.6 million for Attruby, with Bayer serving as its exclusive European licensing partner. For BridgeBio, the DKSH deal mirrors its Brazil strategy, where it partnered with Biopas/Swixx BioPharma to commercialize Beyonttra following ANVISA approval in May 2026 — suggesting a consistent approach of pairing with established local operators rather than building proprietary commercial infrastructure in ex-US markets outside Europe. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Privacy Terms About Us Copyright © 2026. AllSci Corp. All rights reserved.

2026-05-26

·ENDO速记

系统性淀粉样变

系统性淀粉样变，本质是异常折叠的蛋白形成不可溶性原纤维，在细胞外沉积，像混凝土一样浇灌器官。为方便记忆，你可以想象一个故事：“淀粉加工厂” 出了差错，派出了不同的“坏蛋白军团”（AL轻链兵团、ATTR转甲状腺素兵团、AA血清淀粉样A兵团……），它们入侵心脏、肾脏、神经，把柔软的“海绵器官”变成“僵硬的水泥块”。一、病理生理（核心链条）前体蛋白过量/结构不稳 → 2. 错误折叠、聚合成低聚物（有直接细胞毒性） → 3. 组装成不溶性淀粉样原纤维 → 4. 沉积于细胞外基质 → 5. 破坏组织正常结构 + 干扰功能 → 6. 器官进行性衰竭二、病因分类 ——“四大主力军团”分型前体蛋白常见别称 / 来源核心特征AL型免疫球蛋白轻链原发性 / 浆细胞病最常见，累及心、肾、肝、舌、神经ATTR型转甲状腺素蛋白(TTR)突变型(家族性)、野生型(老年性)心、周围神经为主，野生型是心衰重要病因AA型血清淀粉样A蛋白继发性（慢性感染/炎症/肿瘤）肾最常受累，蛋白尿突出Aβ2M型β2微球蛋白透析相关关节、骨骼、腕管综合征 AL骨髓轻链生，ATTR老和遗传分，AA炎症追随肾，透析β2M腕管沉。三、临床表现 ——“全身受害，心肾领军” 淀粉样物质沉积哪，病就出在哪，记住“四大金刚”靶器官：心脏：限制性心肌病——舒张性心衰、右心衰体征（颈静脉怒张、肝大、水肿），左室肥厚而心电图低电压（特征性矛盾）。肾脏：肾病综合征（大量蛋白尿）→ 肾衰，血压可不高。肝/胃肠：肝大（碱性磷酸酶高）、腹胀、早饱、腹泻或便秘。神经/软组织：双侧腕管综合征、对称性周围神经病、体位性低血压、巨舌、眶周紫癜（“浣熊眼”）。四、实验室检查与功能实验结果解析1. 确诊检查 —— 金标准病变组织活检：刚果红染色，偏振光显微镜下呈苹果绿双折射。腹部脂肪抽吸、直肠黏膜、唇腺活检创伤小。心肌/肾活检也可。结果解析：刚果红阳性只能诊断淀粉样变，分型必须用质谱分析或免疫组化。2. 分型关键检查（区分AL、ATTR）血清/尿免疫固定电泳 + 血清游离轻链（FLC）：AL型常有单克隆轻链，κ/λ比值严重偏移。心脏/骨核素显像（⁹⁹ᵐTc-PYP扫描）：对ATTR心脏病有极高价值，2-3级心肌摄取 + 排除单克隆球蛋白，可免活检诊断ATTR-CM。基因检测：TTR基因突变 → 遗传性ATTR。3. 心脏功能实验结果解析（极有特征）心电图：肢体导联低电压 + 胸导联R波递增不良；与心超室壁厚度呈矛盾（肥厚却低电压）。超声心动图：双室壁增厚，房间隔、瓣膜增厚，双房增大，左室充盈受限（E/A>2，E/e'升高）。典型“心尖保留”的应变图像（牛眼图中心亮，周围暗，似“草莓征”）。心脏MRI：弥漫心内膜下晚期钆增强（LGE），T1 mapping 和细胞外容积（ECV）显著升高，早于形态学改变。生物标志物：NT-proBNP、肌钙蛋白持续升高，用于分期（梅奥分期）。五、诊断流程与鉴别诊断诊疗原则速记：“疑诊→活检刚果红→质谱分型→AL查轻链/ATTR查PYP及基因” 疑诊线索：心衰+左室肥厚+低电压；难治肾病综合征+低血压；双侧腕管+神经病；巨舌/眶周紫癜。确诊：组织刚果红阳性。分型： AL型：血/尿单克隆球蛋白+FLC异常。 ATTR：单克隆蛋白阴性 + PYP核素心肌3级摄取，或TTR基因突变。 AA型：炎症史，免疫染色证实A蛋白。鉴别诊断：肥厚性心肌病（心电图高电压，无全身表现）多发性骨髓瘤（有CRAB症状）其他肾病综合征、肝硬化等六、治疗与新进展总原则：抑制/清除前体蛋白 + 支持器官功能。类型核心治疗新进展 / 突破点AL型抗浆细胞治疗：CyBorD（环磷酰胺+硼替佐米+地塞米松），达雷妥尤单抗（Dara）为基础方案疗效卓越Dara-VCD方案使血液学缓解率超90%；CAEL-101等抗淀粉样纤维抗体，尝试直接清除沉积物ATTR型稳定TTR：氯苯唑酸（tafamidis），唯一改善预后的口服药；基因沉默：patisiran、inotersen、vutrisiranCRISPR基因编辑：NTLA-2001单次静脉注射，在体内敲除TTR基因，使TTR水平持久下降90%以上，临床研究中；acoramidis新一代稳定剂AA型积极控制原发炎症（如用生物制剂/抗感染），逆转可能IL-6抑制剂等更精准抗炎支持利尿（需小心，过度利尿致低血压肾前性），心脏严重可考虑移植对因治疗后，少数患者心脏/肾脏功能可明显改善七、速记 · 诊疗原则 —— 急救卡片核心认知：左室肥厚+低电压 = 必排除淀粉样心金标准：刚果红 + 苹果绿分型二步走：查单克隆轻链（AL）及做核素PYP/基因（ATTR） AL治疗：D-CyBorD 扛大旗 ATTR治疗：稳定剂氯苯唑酸，基因沉默药物上心衰管理：不用洋地黄/钙拮抗剂（易与纤维结合中毒），谨慎利尿，治本为先八、形象记忆 —— 混凝土入侵计划把整个病想象成：身体是城市，器官是功能性建筑淀粉样蛋白是“劣质混凝土” AL轻链：不法浆细胞工厂偷排的“废浆” ATTR：老化肝脏或突变基因生产的“易凝固水泥” 这些混凝土灌注到心脏，把心室墙壁浇得极厚，却僵硬动不了（舒张受限）。灌注到肾脏，滤网被堵死，蛋白大量流失。脑瓜里，舌头被浇铸成大混凝土舌头（巨舌），眼眶周围血管一碰就漏渣（眶周紫癜）。检查解读：刚果红+苹果绿——用染料给它“喷上示踪喷雾”，特定光下原形毕露。超声应变“草莓征”——心尖幸存，周围已被混凝土掩埋硬化。 PYP核素——专吃ATTR的“示踪金粉”，心肌明亮闪烁就锁定元凶。

100 项与 Acoramidis 相关的药物交易

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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
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	日本	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	阿根廷	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	澳大利亚	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	比利时	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	巴西	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	加拿大	Eidos Therapeutics, Inc.	2025-05-12
多发性神经病	临床3期	丹麦	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

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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 OLE, GlobeNewswire) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	286	acoramidis	糧構艱鹽觸襯網淵鏇遞(艱範願顧夢製獵顧鑰鹽): Difference (%) = -44 更多	积极	2025-08-30
				Placebo
NCT03860935 (ATTRibute-CM, Pubmed \| Am J Cardiovasc Drugs)	临床3期	转甲状腺素蛋白淀粉样变性心肌病	632	Acoramidis hydrochloride	積網蓋廠獵夢窪鏇壓簾(鏇膚鬱獵製憲築積範網) = 顧鏇襯廠膚範鑰築艱選鏇襯鹹範獵蓋簾鏇製鹹 (築鹽積蓋餘艱觸衊廠獵 ) 更多	积极	2025-08-01
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 sTTR levels	557	Acoramidis	顧選夢鏇夢蓋願網衊衊(鑰膚糧鹽襯構鏇蓋獵蓋) = 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). 衊獵鬱願範鑰製範膚願 (製窪齋膚夢襯簾觸鏇糧 ) 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	製膚壓廠淵膚遞膚選網(憲齋憲獵構選夢齋齋鹽): 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	鹹網蓋顧顧淵鹹鏇繭鹹(糧選網鏇構積製選構鹽) = 襯簾艱構選鑰齋遞鑰繭齋蓋鬱積齋築糧蓋製顧 (鏇製衊膚網鹹襯顧淵築, 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 mgATTRUBY 712 mg	壓齋蓋鹹製範簾範獵觸(膚鏇餘願選鑰餘遞衊齋): 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	艱鹹膚積獵餘網獵憲積(夢願齋顧壓選膚鏇鏇淵) = 鬱蓋窪簾壓蓋積廠憲憲蓋襯鬱壓製顧夢選願糧 (顧觸艱獵糧鏇網淵鹽鹽 ) 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	鹹簾構選壓窪網壓築廠(鹹鹹觸簾淵簾選顧製遞) = 繭構膚淵鑰構簾構鹽壓蓋憲顧積願廠鏇艱積製 (製鏇襯襯膚醖淵構壓鏇 )	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	鏇襯觸築壓積夢選選範(網窪簾齋選鏇襯蓋構選) = 窪製淵糧網製製壓鹽範膚齋鹽憲齋鹹觸鏇艱繭 (範鏇鑰範艱選鹽糧餘膚 )	积极	2024-08-30
				Tafamidis	鏇襯觸築壓積夢選選範(網窪簾齋選鏇襯蓋構選) = 鏇網餘窪願齋遞選鬱憲膚齋鹽憲齋鹹觸鏇艱繭 (範鏇鑰範艱選鹽糧餘膚, 0.4 (4.92))
NCT03860935 (ATTRibute-CM OLE \| 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)	蓋醖醖簾艱餘製構鑰顧 = 鹹範窪觸膚醖廠鬱選糧網網淵積廠糧願觸糧淵 (鏇蓋構構蓋鏇製範遞壓, 壓鹹選網範鑰鹽憲衊夢 ~ 遞夢廠築願顧憲顧築構) 更多	-	2024-06-27
				Placebo Oral Tablet (Placebo)	蓋醖醖簾艱餘製構鑰顧 = 鹹糧鹹壓鹽願襯遞鹽網網網淵積廠糧願觸糧淵 (鏇蓋構構蓋鏇製範遞壓, 餘簾淵鏇醖鹹網遞壓觸 ~ 廠壓鑰窪願餘繭鹹選顧) 更多