A PHASE 1, OPEN-LABEL, RANDOMIZED, CROSSOVER, SINGLE DOSE, PIVOTAL BIOEQUIVALENCE STUDY TO COMPARE TAFAMIDIS FREE ACID TABLET AND COMMERCIAL TAFAMIDIS FREE ACID CAPSULE ADMINISTERED UNDER FASTED CONDITIONS IN HEALTHY ADULT PARTICIPANTS

The purpose of this clinical trial is to compare the amount of tafamidis in the blood of healthy adult participants after taking two different forms of tafamidis by mouth.

开始日期2024-12-20

申办/合作机构

Pfizer Inc.

NCT06393465

/ Active, not recruitingN/A

Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Transthyretin Amyloidosis using data that already exist in patients' medical records

开始日期2024-06-15

申办/合作机构

Pfizer Inc.

NCT06086353

/ RecruitingN/A

A Prospective, Single-arm, Multicenter, Observational Safety Surveillance Study of VyndaMx® (Tafamidis Capsule 61 mg) in Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in India

The purpose of this study is to learn about the safety of Tafamidis for the treatment of Transthyretin amyloid cardiomyopathy (ATTR-CM) in India.
ATTR-CM is a condition that affects people's hearts. Transthyretin is a protein that is made in the liver. In some people this protein stops working and forms clumps called as amyloid. Transthyretin amyloid builds up in heart and stops the heart from pumping properly.
This study is seeking for participants who are:
* confirmed with ATTR-CM.
* given Tafamidis capsules to be taken by mouth.
The safety of Tafamidis capsules will be checked based on side effects. These side effects can happen within 6 months after taking Tafamidis. A side effect is something (expected or unexpected) that you feel was caused by a medicine or treatment you take. The study doctor will collect side effect information and put the information on patient's case form.
Follow-up of the patient's will be performed via clinic re-visit or over a call. It is not a rule for the participants to visit the clinic in this study.
This study will help to see if Tafamidis is safe.

开始日期2024-03-25

申办/合作机构

Pfizer Inc.

100 项与氯苯唑酸相关的临床结果

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100 项与氯苯唑酸相关的转化医学

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100 项与氯苯唑酸相关的专利（医药）

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项与氯苯唑酸相关的文献（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.

2025-10-01·INTERNATIONAL JOURNAL OF CARDIOLOGY

Impact of Tafamidis on survival in elderly patients: Insights from the Healthcare European Amyloidosis Registry

Article

作者: Inamo, Jocelyn ; Broussier, Amaury ; Gellen, Barnabas ; Taieb, Charles ; Mouhat, Basile ; Zaroui, Amira ; Damy, Thibaud ; Bauer, Fabrice ; Roubille, François ; Piriou, Nicolas ; Jeanneteau, Julien ; Fraix, Antoine ; Dagrenat, Charlotte ; Eicher, Jean-Christophe ; Habib, Gilbert ; Donal, Erwan ; Jobbé-Duval, Antoine ; Bisson, Arnaud ; Margerit, Léa ; Trésorier, Romain ; Legallois, Damien ; Bouchot, Océane ; Delelis, François ; Lairez, Olivier ; Kharoubi, Mounira ; Réant, Patricia ; Gueffet, Jean-Pierre ; Charron, Phillipe ; Von Hunolestein, Jean-Jacques ; Courand, Pierre-Yves ; Costa, Jérôme ; Puscas, Tania

BACKGROUND:

Cardiac transthyretin amyloidosis (ATTR-CM) is a life-threatening cardiomyopathy. Tafamidis has been demonstrated to be an effective treatment. Our aim was to analyze clinical characteristics and survival of patients with ATTR-CM aged ≥80 years diagnosed after November 2018, treated with tafamidis 80/61 mg, and compare them with a non-treated group diagnosed before that date.

METHODS:

Data from the two groups were extracted from the Healthcare European Amyloidosis Registry (HEAR). Propensity score matching was used to adjust for baseline differences between the groups. Kaplan-Meier survival curves and Cox regression analyses were applied to assess survival outcomes.

RESULTS:

Out of 1380 patients, 1194 were treated with tafamidis 80/61 mg. Treated patients were significantly less severe at baseline, with a lower occurrence of NYHA class III-IV compared to the untreated group (24 vs. 46 %, p < 0.001). The median NT-proBNP at baseline was lower in the treated group (2330 vs. 4854 pg/ml, p < 0.001), as was the average level of high-sensitivity troponin T (55 vs. 74 ng/ml, p < 0.001), and the interventricular septal thickness (16 vs. 18 mm, p < 0.001). The 3-year survival rate for treated patients was 57 %, and 40 % for untreated patients. In the treated group, the 3-year survival rate was 68 % for patients aged 80-85 years and 58 % for those over 85 years. Survival rates were confirmed after propensity score analyses.

CONCLUSIONS:

This study demonstrates that tafamidis provides significant survival benefits for elderly patients with ATTR-CM, even in those over 85 years old. The findings emphasize the importance of early diagnosis and treatment.

271

项与氯苯唑酸相关的新闻（医药）

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

·FiercePharma

Alnylam exceeds $50B market cap as Amvuttra heart disease launch wows Wall Street

Buoyed by Amvuttra's stellar performance, Alnylam significantly dialed up its full-year TTR sales guidance by about 34% at the midpoint. A stellar launch of rare heart disease med Amvuttra has boosted Alnylam’s market cap above $50 billion.With Alnylam’s second-quarter revenue of $774 million beating Wall Street’s expectations by 18.8%, the RNA interference biotech’s stock price surged more than 15% Thursday. The company’s market cap stood at $51.1 billion at the Nasdaq closing bell on July 31.The significant beat was driven by Amvuttra. In its first full quarter into a closely watched transthyretin amyloidosis cardiomyopathy (ATTR-CM) launch, Amvuttra more than doubled sales year over year to reach $492 million, coming in well above consensus expectations of $351 million.“The launch is off to a very strong start, and, whilst it’s still early days, we’re encouraged by the pace, and we’re deeply focused on laying the groundwork for long-term leadership in TTR,” Alnylam CEO Yvonne Greenstreet said on the company’s second-quarter earnings call Thursday.In what Alnylam Chief Commercial Officer Tolga Tanguler called a “clear signal of early momentum,” about 1,400 ATTR-CM patients have started treatment with Amvuttra since its FDA nod late March, translating to roughly $150 million in revenue, he told investors on the call. The RNA interference drug is also approved in ATTR-polyneuropathy (ATTR-PN).Utilization of Amvuttra among ATTR-CM patients has been “broad and balanced” between new patients and those who’ve progressed on a TTR stabilizer such as Pfizer’s Vyndamax (tafamidis) as well as across both academic and community settings, Tanguler said. Greenstreet noted that the initial uptake has been faster in the stabilizer-progressing patients because doctors are looking for new treatments for them, but the drug is also achieving a “really healthy share” of first-line patients.Nearly all of the approximately 170 priority health systems targeted by Alnylam have put Amvuttra on formulary, Tanguler said. Those networks cover about 80% of the ATTR-CM patient volume.Within just three months, the provider account setup has reached a level that Alnylam had originally planned to achieve in one year, the commercial chief said. The “large majority” of patients across all payer types—Medicare or commercial—have access to Amvuttra as a first-line treatment, according to Tanguler. That means Alnylam is only seeing a low, single-digit percentage of reimbursement policies that require patients try a stabilizer first. And the company finds these policies “relatively easy to manage” because there’s no limit on how long a patient must stay on another therapy, plus it’s up to the physician to determine whether the patient is progressing, Tanguler added.A ”very small portion” of patients are also getting Amvuttra in combination with a stabilizer, Tanguler said, a situation that Alnylam expects to be more popular once Pfizer’s tafamidis franchise goes generic.Buoyed by the stellar performance, Alnylam significantly dialed up its full-year TTR sales guidance by about 34% at the midpoint to a new range of $2.175 billion to $2.275 billion.Still, some analysts suggested that Alnylam may be conservative in its estimate, especially considering an uptick is expected from ex-U.S. markets such as Japan and Europe in the second half of the year.“[W]e think it’s likely Amvuttra will still beat its newly guided numbers this year as mgt continues executing on this high-profile launch,” Citi analysts wrote in a Thursday note. Based on the impressive performance seen in the quarter, William Blair analysts have raised their near-term and peak sales estimates for Amvuttra, now projecting the drug to reach $9 billion sales in 2035 across ATTR-CM and -PN. For reference, Pfizer’s tafamidis franchise, first approved by the FDA in 2019, is currently tracking at about $6 billion in annual sales.“Alnylam is firing on all cylinders,” Greenstreet said, “and we’re swiftly establishing ourselves as a top-tier biotech company.”

上市批准生物类似药

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 项与氯苯唑酸相关的药物交易

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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
转甲状腺素蛋白淀粉样变性心肌病	美国	FoldRx Pharmaceuticals LLC	2019-05-03
遗传性转甲状腺素蛋白淀粉样变性	日本	Pfizer Japan, Inc.	2019-03-26

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
家族性淀粉样神经病	临床3期	日本	Pfizer Inc.	2011-11-01
伴有中性粒细胞减少的皮肤异色病	临床3期	美国	Pfizer Inc.	2009-08-05
伴有中性粒细胞减少的皮肤异色病	临床3期	阿根廷	Pfizer Inc.	2009-08-05
伴有中性粒细胞减少的皮肤异色病	临床3期	巴西	Pfizer Inc.	2009-08-05
伴有中性粒细胞减少的皮肤异色病	临床3期	法国	Pfizer Inc.	2009-08-05
伴有中性粒细胞减少的皮肤异色病	临床3期	德国	Pfizer Inc.	2009-08-05
伴有中性粒细胞减少的皮肤异色病	临床3期	意大利	Pfizer Inc.	2009-08-05
伴有中性粒细胞减少的皮肤异色病	临床3期	葡萄牙	Pfizer Inc.	2009-08-05
伴有中性粒细胞减少的皮肤异色病	临床3期	瑞典	Pfizer Inc.	2009-08-05
老年性心脏淀粉样变性	临床3期	美国	Pfizer Inc.	2009-08-05

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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


ATTR-ACT (Pubmed \| Eur J Heart Fail)	N/A	转甲状腺素蛋白淀粉样变性心肌病 National Amyloidosis Centre (NAC) stage	353	Tafamidis 80 mg	積觸範選築衊觸簾顧網(繭顧製艱網構願製衊糧) = 繭鬱夢衊築壓衊壓壓簾鏇鑰築襯夢醖壓顧膚餘 (鏇醖鬱夢憲網繭構襯構 ) 更多	积极	2025-06-09
				Placebo	積觸範選築衊觸簾顧網(繭顧製艱網構願製衊糧) = 鑰繭簾鏇鬱鑰餘遞獵窪鏇鑰築襯夢醖壓顧膚餘 (鏇醖鬱夢憲網繭構襯構 ) 更多
NCT02791230 (ATTR-ACT, CTgov)	临床3期	转甲状腺素蛋白淀粉样变性心肌病	1,733	Tafamidis (Cohort A: Tafamidis (Parent Study) and Tafamidis (Extension Study))	簾醖醖廠艱窪醖鏇獵糧(鹽鑰醖壓壓鏇構衊簾製) = 鑰衊憲鏇窪觸鹹鏇窪積鑰蓋窪築獵網簾糧鏇憲 (襯壓餘網願網網糧艱觸, 餘顧鏇鬱鏇夢鬱壓蓋鹽 ~ 鏇網憲艱積夢齋鏇膚壓) 更多	-	2025-02-03
				Tafamidis+Placebo (Cohort A: Placebo (Parent Study) and Tafamidis (Extension Study))	簾醖醖廠艱窪醖鏇獵糧(鹽鑰醖壓壓鏇構衊簾製) = 積鑰餘糧鑰窪壓積醖鹽鑰蓋窪築獵網簾糧鏇憲 (襯壓餘網願網網糧艱觸, 構艱窪願鹽顧遞觸壓鹽 ~ 鑰廠齋積製壓鏇糧糧淵) 更多
NCT04814186 (CTgov)	临床4期	转甲状腺素蛋白淀粉样变性心肌病	53	tafamidis	製鹽憲鑰淵簾鹹壓製艱 = 繭鑰夢遞選膚糧憲範壓顧遞鏇淵遞鬱製壓觸醖 (構製窪範築範繭顧壓積, 艱窪蓋積衊鹹鏇膚憲製 ~ 簾醖餘齋鑰蓋襯顧艱鹽) 更多	-	2024-12-18
NCT01994889 \| NCT02791230 (ATTR-ACT, ESC2024)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 NT-proBNP \| eGFR	350	Tafamidis 80 mg	築選願憲簾繭壓網鹽淵(遞繭鬱築鑰鹽衊築淵鏇) = 憲窪蓋壓鏇鏇願願壓膚醖簾膚積鑰襯網鑰壓蓋 (淵衊膚築廠齋窪窪遞繭 ) 更多	积极	2024-09-01
				Placebo	築選願憲簾繭壓網鹽淵(遞繭鬱築鑰鹽衊築淵鏇) = 鬱醖鏇獵醖齋願鹹餘築醖簾膚積鑰襯網鑰壓蓋 (淵衊膚築廠齋窪窪遞繭 ) 更多
ESC2024	N/A	老年性心脏淀粉样变性	-	Tafamidis	醖範鹹淵蓋遞餘製鹽廠(襯餘夢製繭淵壓膚淵襯) = 窪壓襯窪製簾簾構壓鹹艱淵製鹹鑰窪願窪鏇壓 (願選窪蓋簾簾衊鹹壓艱 ) 更多	-	2024-08-31
				Placebo	醖範鹹淵蓋遞餘製鹽廠(襯餘夢製繭淵壓膚淵襯) = 獵憲餘廠廠選蓋顧襯膚艱淵製鹹鑰窪願窪鏇壓 (願選窪蓋簾簾衊鹹壓艱 ) 更多
ESC2024	N/A	老年性心脏淀粉样变性	710	Tafamidis treatment	積簾膚艱醖積簾繭簾夢(簾襯淵淵醖選壓壓壓積) = 壓獵餘獵網膚膚築蓋積齋衊膚鹽鏇憲鏇簾淵顧 (範獵艱積憲襯鏇製範壓 )	积极	2024-08-30
				No Tafamidis treatment	積簾膚艱醖積簾繭簾夢(簾襯淵淵醖選壓壓壓積) = 膚憲製願蓋願鹽壓鑰範齋衊膚鹽鏇憲鏇簾淵顧 (範獵艱積憲襯鏇製範壓 )
NCT02791230 (ATTR-ACT, ESC2024)	临床3期	转甲状腺素蛋白淀粉样变性心肌病	1,481	Tafamidis free acid 61 mg	夢廠鑰憲壓鏇膚壓網獵(膚範衊廠醖遞積艱範觸) = 鏇鏇醖獵鑰糧觸觸鑰廠繭襯醖網憲製鏇鬱積繭 (醖選願餘遞窪繭鹽窪獵, 0.6) 更多	积极	2024-08-30
NCT01994889 \| NCT02791230 (ATTR-ACT, Pubmed \| Eur J Heart Fail)	临床3期	转甲状腺素蛋白淀粉样变性心肌病	-	Tafamidis 80 mg meglumine or 61 mg free acid	獵膚鑰鑰憲蓋選鬱夢淵(構鏇廠鹹壓網蓋窪鑰鏇): P-Value = < 0.001	积极	2024-06-26
				Placebo
ESC2024	N/A	老年性心脏淀粉样变性	-	tafamidis (Females)	遞範鹹蓋鏇廠積鑰憲顧(觸積齋艱築衊鹹遞築鹽) = 齋遞廠糧鑰範醖膚繭遞淵構鏇築窪餘築膚壓鏇 (鑰積構襯艱艱範鏇積醖 ) 更多	-	2024-05-12
				tafamidis (Males)	遞範鹹蓋鏇廠積鑰憲顧(觸積齋艱築衊鹹遞築鹽) = 繭襯獵顧願鑰襯獵艱積淵構鏇築窪餘築膚壓鏇 (鑰積構襯艱艱範鏇積醖 ) 更多
NCT01994889 (ATTR-ACT, Pubmed \| JACC CardioOncol)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 eGFR	-	Tafamidis 20 mg and 80 mg	膚壓顧簾鹽淵鏇壓遞鬱(糧壓簾齋夢鬱範膚選廠) = 夢糧鑰獵襯齋範鑰窪襯壓蓋襯鹹鬱膚簾廠膚網 (艱選構醖蓋築鬱餘選鑰 ) 更多	积极	2024-04-01
				Placebo	膚壓顧簾鹽淵鏇壓遞鬱(糧壓簾齋夢鬱範膚選廠) = 遞窪醖鬱顧夢範築淵鬱壓蓋襯鹹鬱膚簾廠膚網 (艱選構醖蓋築鬱餘選鑰 ) 更多