A Single Arm, Multicenter, Open-label,Phase IV Clinical Study to Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients withTransthyretin Amyloid Polyneuropathy

The purpose of this study is to evaluate the efficacy and safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients with Transthyretin Amyloid Polyneuropathy

开始日期2025-09-01

申办/合作机构

齐鲁制药有限公司

CTR20234057

/ CompletedN/A

氯苯唑酸葡胺软胶囊的人体生物等效性试验

主要目的：在健康受试者体内，分别在空腹和餐后条件下，以Pfizer Europe MA EEIG 持有的氯苯唑酸葡胺软胶囊（商品名： Vyndaqel®；规格：20 mg（按C14H7Cl2NO3.C7H17NO5 计））为参比制剂，石家庄科仁医药科技有限公司生产的氯苯唑酸葡胺软胶囊（规格：20 mg（按C14H7Cl2NO3.C7H17NO5 计））为受试制剂，研究两制剂的药代动力学，评价两制剂是否具有生物等效性。次要目的：观察受试制剂和参比制剂在健康受试者中的安全性。

开始日期2024-01-12

申办/合作机构

石家庄科仁医药科技有限公司

NCT05489523

/ Recruiting临床4期IIT

An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart Transplantation

Transthyretin cardiac amyloidosis (ATTR-CA) is a relentlessly progressive disease that can progress to end stage heart failure, at which point recently approved transthyretin production silencing or structure stabilizing therapies provide no clinical benefit. For well-selected individuals, heart transplantation is an excellent therapeutic option to improve survival. Historically, concomitant liver transplantation has been used to halt the progression of non-cardiac transthyretin amyloidosis (ATTR) manifestations, especially for individuals with TTR genotypes associated with significant neuropathy. However, despite this, patients continue to experience progressive non-cardiac manifestations, particularly gastrointestinal and neuropathic, which can have a substantial influence on post-heart transplantation morbidity. Concomitant liver transplantation is also associated with substantial morbidity and its future therapeutic role is questionable with recently established therapies for ATTR. Therefore, there is a clear unmet need to determine the utility and safety of ATTR targeted therapies for patients with recent heart transplantation for end-stage ATTR-CA. The central hypothesis of this proposal is that in patients who have received a heart transplantation for end-stage ATTR-CA, tafamidis therapy will be efficacious and well-tolerated. We aim to determine the safety and efficacy of tafamidis in stable patients who have undergone heart or combined heart/liver transplantation for ATTR (wild-type or variant) cardiac amyloidosis. The proposed study will be a single-arm intervention clinical trial with tafamidis. Because of the efficacy of tafamidis for both variant ATTR-CA and wild-type ATTR-CA, there is no clinical equipoise for an inactive-comparator placebo arm. The primary endpoint of this study will be serial change in plasma transthyretin (TTR) levels from baseline to 12 months at 3-month intervals. The secondary endpoints of this study will include serial changes in neuropathy assessments, modified body mass indices, incident transplant-specific adverse events, and pharmacokinetics of tafamidis. Observations from this study will establish the role of tafamidis use for the management of ATTR in patients after transplantation for end-stage ATTR-CA.

开始日期2023-05-01

申办/合作机构

The University of Texas Southwestern Medical Center

100 项与氯苯唑酸葡甲胺相关的临床结果

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100 项与氯苯唑酸葡甲胺相关的转化医学

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100 项与氯苯唑酸葡甲胺相关的专利（医药）

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282

项与氯苯唑酸葡甲胺相关的文献（医药）

2025-06-01·JOURNAL OF MOLECULAR STRUCTURE

Host-guest inclusion complexes of Tafamidis with β-Cyclodextrin: Preparation, characterization, and in vitro antibacterial and antioxidant approach

作者: Chintha, Jagadeesh ; Sripada, Lakshminath ; Kamalakaran, Anand Solomon ; Swarnkar, Nikita ; Balakrishnan, Suganya Bharathi ; Kambhampati, Naga Sai Visweswar

This article investigates the interactions between β-Cyclodextrin (β-CD) and the anti-inflammatory drug Tafamidis (TF) to prepare an inclusion complex (IC) that enhances its pharmacol. effects.Here, three different methods have been employed to prepare inclusion complexes (ICs), which help the efficient combination of the drug with Cyclodextrin (CD) to produce a stable complex.Various characterization techniques were used to examine the formed ICs.Fourier transform IR spectroscopy (FTIR) was used to identify the complex formation.Comprehensive information on the chem. structure was obtained using 1H NMR (1H NMR) and 13C NMR (13C NMR).Thermogravimetric anal. (TGA) and differential scanning calorimetry (DSC) anal. were used to evaluate the complexes' thermal characteristics and their stability.The ICs were then examined with X-ray diffraction (XRD).Drug release studies showed that ICs increased drug solubilityThe antibacterial activity of the prepared ICs was tested against Staphylococcus aureus (S. aureus) and Escherichia coli (E. coli).Furthermore, the complexes demonstrated higher in vitro antioxidant activity than the pristine drug mol.This strategy could help improve the efficacy and delivery of poorly soluble drugs.

2025-04-01·Neural Regeneration Research

Design and redesign journey of a drug for transthyretin amyloidosis

Article

作者: Ventura, Salvador ; Pinheiro, Francisca

The misfolding and subsequent aggregation of proteins into amyloid fibrils underlie the onset of a variety of human disorders collectively known as amyloidosis.Transthyretin (TTR) is one of the > 30 amyloidogenic proteins identified to date and is associated with a group of highly debilitating and life-threatening disorders called TTR amyloidosis (ATTR).ATTR comprises senile systemic amyloidosis, which is linked to wild type (WT) TTR aggregation, and hereditary ATTR, a dominantly inherited disorder caused by the deposition of one of over 130 TTR genetic variants.The perspective summarized TTR aggregation mechanism and the crystal structure of WT-TTR bound to tolcapone, M-23, and pharmacokinetically improved transthyretin binder (PITB).

2025-04-01·European Journal of Nuclear Medicine and Molecular Imaging

Impact of Tafamidis on [99mTc]Tc-pyrophosphate Scintigraphy in Ala97Ser Hereditary Transthyretin amyloid cardiomyopathy: significant initial reduction with stable Long-Term effects

Article

作者: Lee, Ming-Jen ; Cheng, Mei-Fang ; Su, Mao-Yuan ; Chen, Yi-Chieh ; Lin, Yen-Hung ; Hsieh, Sung-Tsang ; Juang, Jyh-Ming Jimmy ; Hsueh, Hsueh-Wen ; Tsai, Cheng-Hsuan ; Shun, Chia-Tung ; Yu, An-Li ; Tseng, Ping-Huei ; Chao, Chi-Chao

OBJECTIVE:

Tafamidis has shown potential in slowing disease progression in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). This study aimed to evaluate serial changes on [^99mTc]Tc-pyrophosphate (PYP) scintigraphy during tafamidis treatment for hereditary ATTR-CM.

METHODS:

We retrospectively analyzed a prospectively collected cohort of Ala97Ser (A97S) hereditary ATTR-CM patients treated with tafamidis (61 mg/day) and a control group comprising A97S hereditary ATTR-CM patients who had not received disease-modifying medications. The tafamidis group was further divided into two cohorts: cohort A received [^99mTc]Tc-PYP SPECT/CT scans at baseline, 1 year, and 2 years; cohort B at baseline, 2 years, and 3 years. Visual score, planar heart to contralateral lung (H/CL) ratio, and volumetric heart to lung (H/L) ratio were measured.

RESULTS:

Nineteen patients were enrolled in the tafamidis group and nine in the control group. After 2 years of follow-up, a significant decrease in volumetric H/L ratio (3.86 ± 0.91 to 3.01 ± 0.19, p < 0.001) was noted in the tafamidis group, while there was no significant change in the control group. When evaluated over time, a significant decrease in volumetric H/L ratio was observed during the first year of tafamidis treatment (3.75 ± 0.37 to 2.82 ± 0.15, p = 0.004), followed by stable [^99mTc]Tc-PYP uptake in the subsequent two years (2.82 ± 0.15 to 2.83 ± 0.18, p = 0.934 and 3.20 ± 0.14 to 3.09 ± 0.16, p = 0.404, respectively).

CONCLUSION:

A significant reduction in [^99mTc]Tc-PYP uptake was observed in hereditary ATTR-CM patients after tafamidis treatment, particularly within the first year. While the effect appeared to be sustained, stable [^99mTc]Tc-PYP uptake without further significant reductions was observed in the subsequent years.

289

项与氯苯唑酸葡甲胺相关的新闻（医药）

2025-06-02

·同写意

辉瑞“寻宝”：谁来填补250亿美元专利悬崖？

同写意年度巨献！！7月25-26日，邀您相聚金鸡湖畔，与5000人一起为中国医药创新“同写意”！近日辉瑞（Pfizer）与三生制药就PD-1/VEGF双抗达成60亿美元的合作，在医药界引发广泛关注，也揭开了辉瑞新一轮战略布局的序幕。然而，对于这家年营收超600亿美元的巨头而言，真正的挑战在于未来3年内高达250亿美元销售额的产品将面临专利到期，形成巨大的“专利悬崖”。这直接导致近80亿美元的营收缺口亟待填补。手握100-150亿美元并购预算的辉瑞，将如何精准出击，寻找能带来快速增长的“猎物”？本文将深入剖析辉瑞的家底、困境与管线，并对其潜在的并购或BD目标进行更细致的展望。TONACEA01辉瑞当前营收版图增长引擎与潜在危机并存2024年，辉瑞总营收达到636亿美元。其业务结构主要分为三大块：基础护理（Primary Care）、专科护理（Specialty Care）和肿瘤（Oncology）。基础护理部分占比47%，但近三年来，这一板块营收占比逐年下滑，与去年相比，同比下降2%。与此同时，专科护理和肿瘤业务却呈现出快速增长的态势，尤其是肿瘤业务，同比增长率高达25%，成为增长的强劲引擎。从具体销售额来看，基础护理业务中，心血管药物、COVID-19相关产品、偏头痛类药物占据主导。其中，心血管药物Eliquis以73亿美元的销售额位居榜首，紧随其后的是Prevnar疫苗系列、新冠药物Paxlovid和Comirnaty，分别实现销售额64亿、57亿和53亿美元。在专科护理业务中，罕见病药物Vyndaqel系列表现抢眼，销售额达54亿美元；自免老将Xeljanz贡献11亿美元。肿瘤业务方面，乳腺癌药物Ibrance以43亿美元的销售额位居第一，前列腺癌药物Xtandi销售额为20亿美元。从增长趋势来看，基础护理业务中，除Paxlovid外，大部分产品增速放缓，专科护理业务中Vyndaqel、Octagam和Cibinqo等少数产品处于高增长阶段，其余多数产品呈现负增长。而肿瘤业务增长趋势明显，收购Seagen带来的Padcev和Adcetris两款产品分别实现了2896%和1845%的同比增长，强力支撑了肿瘤业务的高速扩张。TONACEA02“专利悬崖”压顶80亿美元的巨大挑战辉瑞未来3年将面临8个重磅产品在美国、欧洲、日本的专利集中到期。这些产品的销售额在2024年总营收中占比高达40%，累计达250亿美元。与此同时，所有正增长的产品（排除未来三年面临专利悬崖的产品）在2024年的累计营收占比仅为27%，即173亿美元，这意味着辉瑞将面临近80亿美元的营收缺口，填补这一缺口成为当务之急。目前，辉瑞现有高增长的重点产品包括Padcev、Nurtec ODT/Vydura和Adcetris。Padcev于2021年获得FDA批准，预测峰值销售额为27亿至34亿美元，如今三年过去，销售额已达16亿美元，距离峰值仍有一定距离。Nurtec ODT/Vydura分别于2020年和2022年获得FDA和EMA批准，预测峰值销售额为20亿美元。Adcetris自上市以来，凭借其在淋巴瘤治疗领域的独特优势，也取得了不俗的市场表现，其销售额持续攀升，有望成为辉瑞在肿瘤治疗领域的又一重要支柱产品。尽管这些重点产品呈现出强劲的增长势头，但与辉瑞面临的80亿美元专利悬崖空缺相比，仍显得力不从心，难以在短期内完全填补这一巨大的市场缺口。因此，仅依靠现有产品和内生增长难以跨越专利悬崖，辉瑞必须通过M&A和BD来寻求突破。TONACEA03研发管线剖析肿瘤重兵集结，内科亟待加强辉瑞的研发投入主要集中在自免、内科、肿瘤和疫苗四大领域，其中肿瘤是核心中的核心，研发投入占比高达50.9%。肿瘤管线方面，辉瑞重点聚焦乳腺癌领域，核心策略是寻找Palbociclib的替代者，重点布局PROTAC技术、CDK2和CDK4抑制剂以及HER2抑制剂，力图覆盖HER2+、ER+/HER2-及HR+/HER2-等多种亚型乳腺癌。辉瑞在大分子药物方面重仓ADC，拥有近10个不同靶点，包括Integrin beta-6、Nectin-4、HER2、Tissue Factor、CD228、B7H4、PD-L1、CEACAM5、CD30以及Mesothelin，旨在通过ADC技术拓展至膀胱癌、尿路上皮癌、宫颈癌和非小细胞肺癌等多种癌种。自免管线方面，主要火力集中在特应性皮炎和溃疡性结肠炎这两个大适应症上，多数研发项目已进入中期阶段。老产品拓展适应症的情况较少（以Ritlecitinib为主），大部分为新分子实体。靶点选择较为多样，白介素（IL）家族靶点相对较多，另有6条管线的靶点尚未披露。内科领域方面，整体管线相对薄弱。主要聚焦于MASH、心衰和肥胖等疾病。在MASH领域，辉瑞选择的靶点PNPLA3、DGAT2和ACC较为冷门；在肥胖领域，选择了GIPR和GLP-1R作为靶点，但研发进度显著落后，目前仍处于1期临床阶段。疫苗领域，辉瑞的产品主要分为细菌感染和病毒感染两大类。在病毒感染疫苗方面，与BioNTech合作开发的COVID-19 mRNA疫苗占据主导，此外还包括流感病毒、RSV等疫苗。细菌感染疫苗则相对多样，涵盖链球菌和艰难梭菌等。整体来看，肿瘤管线虽然广泛，但显得有些零散，缺乏具有基石地位的产品，近期引进三生制药的PD-1/VEGF双抗有望成为后续的支柱产品。内科管线尤其薄弱，急需通过外部引进高质量资产来强化。TONACEA04辉瑞的“猎物清单”百亿美金将砸向何方？在2025年第一季度，辉瑞透露其拥有100亿至150亿美元的预算，旨在快速填补未来三年因专利悬崖带来的营收缺口。辉瑞强调，其未来的商务拓展（BD）和并购（M&A）策略将聚焦于“增长型资产”（revenue growth accretive assets），首要考虑临床差异化，其次关注商业价值。01肿瘤领域辉瑞新获得的PD-1/VEGF双抗，其未来价值很大程度上取决于与何种疗法联用。ADC是目前的热门联用方向。竞争对手中，BioNTech的思路是将PD-1/VEGF双抗与TROP2、HER2、B7H3、HER3等一众ADC（抗体药物偶联物）联用，以实现更优的治疗效果。默克暂未公布联用计划，但已在其管线中储备了B7H3 ADC、HER3 ADC、TROP2 ADC等多种ADC，为联合用药提供了多种选择。康方生物目前主要精力放在其双抗与PD-(L)1单抗的优效性试验上，ADC联用计划披露不多。从现有临床数据看，TROP2 ADC和Nectin-4 ADC与IO疗法联用展现出较好潜力。但是考虑到TROP2 ADC赛道已相对拥挤（已有2款获批上市），辉瑞短期内再次BD一款全新TROP2 ADC的可能性或许不高，除非该分子具有极强的差异化优势。因此辉瑞的策略可能是优先评估其PD-1/VEGF双抗与自身ADC管线（如HER2 ADC, Nectin-4 ADC, B7H4 ADC等）的联用潜力。02MASH领域MASH市场巨大且远未被满足，辉瑞在该领域布局薄弱，并购优质标的可迅速形成战斗力。Madrigal Pharmaceuticals：核心资产为Rezdiffra（Resmetirom），一种口服、肝脏靶向的THR-β选择性激动剂。已于2024年初获得FDA批准，用于治疗伴有中度至晚期肝纤维化（F2/F3期）的NASH成人患者，是美国FDA批准的首个专门针对NASH的药物。自2024年Q2上市以来，销售额已迅速达到3亿美元，超过17000名患者正在用药，并已覆盖70%的顶尖处方医生群体，显示出强劲的市场渗透能力。预计峰值销售额可达35亿美元。目前Rezdiffra正积极向MASH F4C扩展，其MAESTRO-NASH OUTCOMES研究已于去年10月完成患者入组，预计试验将进行2-3年，最快可能于2028年获批F4C适应症。今年EALS大会上公布的MAESTRO-NAFLD-1研究扩展队列数据显示，药物对肝硬度等指标有积极影响。Madrigal目前市值约60亿美元，完全在辉瑞的预算射程内。产品已上市且快速放量，并购后能迅速为辉瑞贡献营收，解燃眉之急。Akero Therapeutics：核心资产为Efruxifermin（EFX），一种Fc-FGF21融合蛋白，目前正在进行三项3期临床试验，评估其治疗MASH F2/F3期和MASH F4C患者的疗效。EFX在抗肝纤维化方面展现出目前MASH在研药物中的领先效果。在针对MASHF4C的SYMMETRY研究中，第96周数据显示，50mg EFX组有39%的患者实现纤维化改善≥1个阶段且MASH没有恶化，显著优于安慰剂组的15% （p<0.01）。在针对F2/F3期纤维化患者的HARMONY研究中，F3亚组数据显示，50mg EFX组有68%的患者实现纤维化改善≥1个阶段，而安慰剂组仅为14%。从试验进度来看，efruxifermin预计最早可在2028年获批上市。Akero Therapeutics目前市值约37亿美元，近期也有寻求被收购的传闻。可作为参照的是，GSK此前以首付款12亿美元、总价20亿美元的价格收购了波士顿制药的FGF21类似物Efimosfermin（预计最早2029年上市），显示了大型药企对FGF21靶点的认可。对辉瑞而言，Akero是具有高潜力的前瞻性布局。03肥胖领域尽管辉瑞自身肥胖管线进度滞后，但该市场潜力巨大，通过并购快速入局不失为一种策略。Viking Therapeutics：其核心资产为VK2735，一种GLP-1/GIP双重激动剂，同时开发皮下注射和口服两种剂型。皮下注射版：2期临床数据显示，治疗13周时可实现14.7%的平均体重降幅，且患者在停药后4周内大多能维持减重效果，提示其具有开发为每月一次维持用药的潜力。口服版：1期临床数据显示，100mg剂量治疗4周可减重8.3%（vs 安慰剂），同样在给药后4周内可保持减重效果。口服版VK2735展现出较好的胃肠道耐受性，因副作用导致的停药率较低。目前，Viking Therapeutics的市值约为30亿美元，处于相对合理的估值区间。对于辉瑞而言，收购Viking Therapeutics不仅可以快速填补其在肥胖治疗领域的管线空白，还能借助VK2735的市场潜力，进一步拓展其在代谢疾病领域的布局。— 结语 —面对迫在眉睫的专利悬崖和激烈的市场竞争，辉瑞的百亿美金将如何投出，不仅关乎短期业绩的修复，更将深刻影响其未来十年的发展轨迹。MASH领域的成熟或后期资产（如Madrigal、Akero）因其能较快贡献营收或填补管线空白，优先级可能更高。肿瘤领域，在已有PD-1/VEGF双抗的基础上，寻找能与之高效协同的ADC或其他创新疗法，也是重要方向。而潜力巨大的肥胖市场，则是辉瑞是否愿意进行高风险高回报战略性布局的考验。未来一两年内，辉瑞在资本市场的每一次出手都将备受瞩目。这场“寻宝”之旅，最终将为辉瑞带来怎样的未来？我们拭目以待。参考资料：各公司演示材料

并购抗体药物偶联物疫苗专利到期

2025-06-02

·Science Daily

Combination therapy can prolong life in severe heart disease

Aortic valve narrowing (aortic stenosis) with concomitant cardiac amyloidosis is a severe heart disease of old age that is associated with a high risk of death. Until now, treatment has consisted of valve replacement, while the deposits in the heart muscle, known as amyloidosis, often remain untreated. Researchers have now demonstrated that combined treatment consisting of heart valve replacement and specific drug therapy offers a significant survival advantage for patients. Aortic valve narrowing (aortic stenosis) with concomitant cardiac amyloidosis is a severe heart disease of old age that is associated with a high risk of death. Until now, treatment has consisted of valve replacement, while the deposits in the heart muscle, known as amyloidosis, often remain untreated. An international research consortium led by MedUni Vienna and University College London has now been able to demonstrate for the first time that combined treatment consisting of heart valve replacement and specific drug therapy offers a significant survival advantage for patients. The study results have been published in the European Heart Journal. As part of the study conducted by the research team led by Christian Nitsche (Department of Medicine II, Clinical Division of Cardiology, MedUni Vienna) and Thomas Treibel (Department of Cardiovascular Imaging, University College London), data from 226 patients with aortic stenosis and concomitant cardiac amyloidosis from ten countries were examined. Aortic stenosis is a narrowing of the heart valve that directs blood from the left ventricle into the bloodstream. In cardiac amyloidosis, misfolded proteins are deposited in the heart muscle. Both diseases occur in older people and often together. Until now, it was unclear whether treating amyloidosis in addition to valve surgery would benefit patients. The analysis now published showed that both aortic valve replacement and treatment with the drug tafamidis for amyloidosis were associated with a significantly lower risk of death. The survival benefit was highest in patients who received both forms of treatment. "Our results even show that patients with both conditions who received valve replacement and specific amyloidosis therapy had similar long-term survival rates to people with aortic stenosis without amyloidosis," emphasises study leader Christian Nitsche. Targeted tests necessary Both aortic stenosis and cardiac amyloidosis impair the heart's pumping function and can lead to death if left untreated. Targeted therapy can slow the progression of amyloidosis, while valve replacement treats the mechanical stress caused by the narrowed heart valve. Around ten percent of patients with aortic stenosis also have amyloidosis, but this is often not diagnosed in everyday clinical practice. "Our findings also suggest that patients with severe aortic valve stenosis should be screened for amyloidosis so that we can offer them targeted life-prolonging treatment options," emphasises Christian Nitsche.

AHA会议

2025-05-29

·FierceBiotech

Intellia\'s stock sinks on gene therapy patient\'s raised enzyme levels, but analysts keep faith

Raised liver enzymes were a factor during Intellia Therapeutics\' phase 1 trial of nex-z in ATTR.\n Intellia Therapeutics’ stock may have taken a hit in the wake of details of a grade 4 adverse event in a phase 3 trial, but analysts are keeping faith in the gene therapy’s wider safety profile.The biotech used a Securities and Exchange Commission filing May 28 to provide an update on a trio of late-stage trials of its candidate nex-z. This included a study in transthyretin amyloid cardiomyopathy (ATTR-CM), which has seen more than 200 patients dosed to date.So far, the reported adverse events have been “similar in nature” to a phase 1 trial of the drug in ATTR-CM, Intellia said. These events have included infusion-related reactions and asymptomatic liver transaminase elevations, a term for raised levels of the ALT and AST enzymes that can be a sign of liver stress.One of the cases of raised liver transaminase was classed as grade 4—the most serious level of nonfatal adverse event. This recent event was asymptomatic and was identified by laboratory tests, according to Intellia. It then appeared to resolve without the need for hospitalization or medical intervention, and the ALT and AST enzyme levels of this individual are currently classified as grade 3 and 2, respectively.Still, the details appeared to trigger a sell-off of Intellia’s stock, which was 23% in the red in premarket trading Thursday morning. William Blair analysts branded the grade 4 event “obviously disappointing” and suggested the stock drop meant “there will be outstanding question about safety moving forward.”“However, we believe the asymptomatic nature of these elevations and low incidence still plays out for a favorable risk/benefit for nex-z in TTR amyloidosis,” the analysts added in a May 29 note. “Yes, there are other effective treatment options here with TTR stabilizers and silencers, so safety will be a consideration moving forward in this indication, but … we continue to see nex-z’s TTR knockdown profile as impressive and de-risking a positive clinical outcome.” As Intellia alluded to in yesterday’s filing, raised enzyme levels were a factor during its phase 1 trial of nex-z in ATTR, leading the company to add a lower fixed dose level to the polyneuropathy arm of that study back in 2022.There are a host of drugs on the market for several versions of ATTR including Alnylam’s Onpattro and Amvuttra, Ionis Pharmaceuticals\' Tegsedi and its AstraZeneca-partnered Wainua, which are used against polyneuropathy (damage to nerves) associated with ATTR. Pfizer’s Vyndaqel is also approved to help stabilize the transthyretin protein for cardiomyopathy associated with TTR.Intellia’s plan remains to submit an approval application to the FDA for nex-z in transthyretin amyloid polyneuropathy (ATTRv-PN) in 2028 with an anticipated U.S. commercial launch in 2029, the biotech said in its filing. Before that, the company hopes to submit a separate FDA approval application next year for the gene therapy in hereditary angioedema. The company is currently running phase 3 trials in both those indications, in addition to the ATTR-CM study.

$Intellia\'s stock sinks on gene therapy patient\'s raised enzyme levels, but analysts keep faith$

临床3期基因疗法临床1期上市批准临床结果

100 项与氯苯唑酸葡甲胺相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D09674	氯苯唑酸葡甲胺	N07XX08	DB11644

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
淀粉样神经病	中国	Pfizer Europe MA EEIG	2020-02-05
心肌疾病	美国	FoldRx Pharmaceuticals LLC	2019-05-03
转甲状腺素蛋白淀粉样变性心肌病	韩国	Pfizer Pharmaceuticals Korea Ltd.	2015-04-28
家族性淀粉样神经病	日本	Pfizer Japan, Inc.	2013-09-20
遗传性转甲状腺素蛋白淀粉样变性	欧盟	Pfizer Europe MA EEIG	2011-11-16
遗传性转甲状腺素蛋白淀粉样变性	冰岛	Pfizer Europe MA EEIG	2011-11-16
遗传性转甲状腺素蛋白淀粉样变性	列支敦士登	Pfizer Europe MA EEIG	2011-11-16
遗传性转甲状腺素蛋白淀粉样变性	挪威	Pfizer Europe MA EEIG	2011-11-16

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
伴有中性粒细胞减少的皮肤异色病	临床3期	阿根廷	Pfizer Inc.	2008-07-01
伴有中性粒细胞减少的皮肤异色病	临床3期	巴西	Pfizer Inc.	2008-07-01
伴有中性粒细胞减少的皮肤异色病	临床3期	法国	Pfizer Inc.	2008-07-01
伴有中性粒细胞减少的皮肤异色病	临床3期	德国	Pfizer Inc.	2008-07-01
伴有中性粒细胞减少的皮肤异色病	临床3期	葡萄牙	Pfizer Inc.	2008-07-01
伴有中性粒细胞减少的皮肤异色病	临床3期	瑞典	Pfizer Inc.	2008-07-01

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临床结果

适应症

分期

评价

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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


NCT05482308 (CTgov)	临床1期	-	12	Tafamidis Free Acid (Variant 12.2 mg Tafamidis Free Acid Tablet (Test))	鹽淵鏇網廠艱齋艱願醖(構鹹網鑰衊膚餘鑰膚顧) = 糧憲構觸廠膚廠鑰憲廠膚齋顧糧遞願膚醖餘簾 (獵遞鹽構網觸艱糧壓壓, 21)	-	2024-05-23
NCT05482308 (CTgov)	临床1期	-	12	Tafamidis Free Acid (Proposed Commercial 12.2 mg Tafamidis Free Acid Tablet (Reference))	鹽淵鏇網廠艱齋艱願醖(構鹹網鑰衊膚餘鑰膚顧) = 網簾獵窪願顧醖蓋顧廠膚齋顧糧遞願膚醖餘簾 (獵遞鹽構網觸艱糧壓壓, 25) 更多	-	2024-05-23
NCT04828993 (CTgov)	临床4期	家族性淀粉样神经病	15	Tafamidis Meglumine	鏇襯選鏇簾鹽選繭齋壓(鏇餘積簾願鑰鏇築壓築) = 構網鬱膚蓋淵鏇獵齋鹽顧鹽獵廠壓餘鬱壓艱襯 (構顧衊襯範觸艱夢願鹽, 鹹膚簾壓繭築襯衊膚積 ~ 願廠遞遞憲淵醖夢繭糧) 更多	-	2024-03-12
NCT02146378 (Pubmed \| Clin Ther)	N/A	遗传性转甲状腺素蛋白淀粉样变性	219	Tafamidis Meglumine 20 mg/d	艱簾鑰糧簾餘廠鹽積淵(淵積艱衊顧淵構顧積糧) = 窪製鏇築膚窪願齋餘夢遞齋夢網壓鏇顧網積鏇 (遞築積淵鏇繭壓鏇獵糧 ) 更多	-	2020-09-01
ATTR-ACT (ESC2019)	N/A	转甲状腺素蛋白淀粉样变性心肌病 variant (ATTRm) \| wild-type (ATTRwt) TTR	-	Tafamidis meglumine	壓夢醖範衊範鑰選艱構(構鹹憲夢構蓋衊遞鹽網): hazard ratio = 0.64 (95% CI, 0.47 ~ 0.85), P-Value = 0.001	积极	2019-09-01
ATTR-ACT (ESC2019)	N/A	转甲状腺素蛋白淀粉样变性心肌病 variant (ATTRm) \| wild-type (ATTRwt) TTR	-	Placebo		积极	2019-09-01
NCT00694161 (Pubmed \| Circ Heart Fail) 人工标引	临床2期	转甲状腺素蛋白淀粉样变性心肌病	31	tafamidis	夢構膚網簾範簾糧醖餘(鏇願淵鏇鏇艱製觸憲齋) = 遞顧餘鑰窪齋壓選繭夢衊製顧鹽範選鬱壓蓋鹽 (淵膚獵艱鑰蓋積網構顧 ) 更多	积极	2015-05-01
NCT00694161 (CTgov)	临床2期	心肌疾病 \| 转甲状腺素蛋白淀粉样变性心肌病	35	tafamidis (Fx-1006A)	餘艱糧願遞觸蓋淵鑰選 = 獵壓範築鑰衊膚願製獵製艱廠鏇艱壓衊餘築簾 (衊艱齋繭顧艱觸糧鏇艱, 獵獵鹽憲餘窪淵憲遞製 ~ 醖衊鹽鑰構選構製齋鑰) 更多	-	2012-12-18
NCT00791492 (Fx-005, CTgov)	临床2/3期	家族性淀粉样神经病 \| 伴有中性粒细胞减少的皮肤异色病	86	Tafamidis (Tafamidis-Tafamidis)	鏇繭鏇廠鑰繭積餘壓壓 = 構鏇窪網選獵壓築遞鏇醖壓蓋製願齋簾餘膚蓋 (衊顧蓋艱簾獵觸簾繭願, 範範醖鏇獵餘蓋齋糧獵 ~ 鑰積衊製鹽衊醖夢蓋醖) 更多	-	2012-12-17
NCT00791492 (Fx-005, CTgov)	临床2/3期	家族性淀粉样神经病 \| 伴有中性粒细胞减少的皮肤异色病	86	Tafamidis+Placebo (Placebo-Tafamidis)	鏇繭鏇廠鑰繭積餘壓壓 = 鹽鹽糧願壓製醖製鬱憲醖壓蓋製願齋簾餘膚蓋 (衊顧蓋艱簾獵觸簾繭願, 網鑰鹹顧醖遞鑰構膚艱 ~ 築餘簾窪齋築網願齋窪) 更多	-	2012-12-17
NCT00409175 (CTgov)	临床2/3期	家族性淀粉样神经病	128	Tafamidis (Fx-1006A) (Tafamidis)	鑰糧鬱鹹鏇鬱網衊製窪 = 構網簾願築鏇鏇壓蓋獵顧顧壓衊壓艱餘淵觸衊 (願顧願網築顧餘壓構鏇, 艱廠糧積醖願憲鹽鏇遞 ~ 觸構獵衊窪夢觸構憲積) 更多	-	2012-12-17
NCT00409175 (CTgov)	临床2/3期	家族性淀粉样神经病	128	Placebo+tafamidis (Fx-1006A) (Placebo)	鑰糧鬱鹹鏇鬱網衊製窪 = 選鏇積範鏇遞簾顧構鬱顧顧壓衊壓艱餘淵觸衊 (願顧願網築顧餘壓構鏇, 膚壓衊遞蓋鏇醖鑰鑰齋 ~ 鏇範蓋窪鹽憲選網糧憲) 更多	-	2012-12-17
NCT00630864 (CTgov)	临床2期	转甲状腺素蛋白淀粉样变性心肌病 \| 家族性淀粉样神经病	21	tafamidis (Fx-1006A)	壓衊廠觸遞遞構願簾簾 = 衊糧壓網網繭窪糧醖廠選蓋艱醖鑰糧鏇襯淵糧 (選壓蓋繭壓築艱繭淵膚, 觸壓膚簾餘餘夢遞鏇鹽 ~ 網遞鏇窪鹹蓋鬱鹽衊願) 更多	-	2012-12-17