A Phase 3, Randomized, Open-label, Parallel-arm, Active-controlled, Multicenter Study to Evaluate Safety and Efficacy of ALXN1850 Versus Asfotase Alfa Administered Subcutaneously in Pediatric Participants (2 to < 12 Years of Age) With Hypophosphatasia (HPP) Previously Treated With Asfotase Alfa

The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.

开始日期2024-04-02

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT05234567

/ RecruitingN/A

A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia

In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and potentially 1 or 2 other countries.

开始日期2022-08-25

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT04189315

/ Withdrawn临床4期

A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia

The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).

开始日期2020-04-01

申办/合作机构

Alexion Pharmaceuticals, Inc.

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项与阿司福酶α 相关的文献（医药）

2025-12-01·Current Osteoporosis Reports

Medical Management of Hypophosphatasia: Review of Data on Asfotase Alfa

Review

作者: Dunbar, Nancy S ; Dahir, Kathryn McCrystal

Abstract :

Purpose:

Hypophosphatasia (HPP) is a rare, dento-osseous disorder caused by impaired activity of tissue non-specific alkaline phosphatase (TNSALP), a key enzyme in tissue mineralization. This review provides a clinical perspective on the current medical treatment of both children and adults with HPP.

Recent Findings:

Dental problems, rickets in children, and osteomalacia in adults are common in HPP. However, disease manifestations in individual patients are exceptionally variable. Recent studies broadened our understanding of HPP symptoms. For example, data showed behavioral health challenges in HPP children, and a large, real-world data set from the Global HPP Registry demonstrated that HPP adults regardless of the time of disease onset exhibit significant disease burden and are broadly affected by non-skeletal impairments, such as pain and chronic fatigue. Treatment for HPP relies on the enzyme replacement asfotase alfa. Small, mostly pediatric trials initially established dosing, safety and efficacy of asfotase alfa, and latest data corroborated the long-term safety and efficacy in both children and pediatric-onset adults. Data from several recent observational studies, including the Global HPP Registry, underscored that asfotase alfa improves physical functions, non-skeletal symptoms such as pain, and quality-of-life (QoL) in adults irrespective of age-of-onset. Clinical use of asfotase alfa is based on prescribing information and evidence-based consensus guidelines. However, recommendations for initiation of therapy are just emerging. Alternatives to asfotase alfa remain limited, but a derivative, efzimfotase alfa, currently undergoes clinical testing.

Summary:

Studies in larger HPP patient populations suggest efficacy of enzyme replacement therapy independent of patient age and time of disease onset.

2025-05-01·Journal of Comparative Effectiveness Research

Effectiveness and safety of asfotase alfa for people with hypophosphatasia: a plain language summary of three studies

Review

作者: Belaya, Zhanna ; Jasmi, Fatma Al

What is this summary about?:

Hypophosphatasia (HPP for short) is a rare inherited condition that can present at any stage of life, with symptoms typically being more severe in cases that manifest earlier, such as during infancy or childhood. This article summarizes three published studies that looked at the safety and effectiveness of asfotase alfa for people with HPP. The aim of this article is to present the findings of three studies clearly and engagingly to make them accessible to readers without a scientific background.The first study (called study 1) discussed in this article looked at how safe and effective asfotase alfa was for children with life-threatening HPP. The second study (called study 2) looked at how safe and effective asfotase alfa was for children or adolescents who had showed the first signs of HPP at birth or in childhood. They received treatment in clinical practice in Japan, rather than in a clinical research study. The third study (called study 3) looked at how safe and effective asfotase alfa was for adults with HPP who had showed the first signs of the condition in childhood. They received treatment in clinical practice, rather than in a clinical research study.

What were the results?:

In study 1, asfotase alfa improved bone health, the ability to breathe, and growth in children with life-threatening HPP. The researchers saw these benefits over 6 years of follow-up by HPP care teams. The treatment was generally well-tolerated.In study 2, asfotase alfa relieved symptoms such as pain, improved growth, and improved the ability to breathe in children and adolescents with HPP in Japan. The treatment was generally well-tolerated, with no serious side effects. Some participants had mild reactions at injection sites.In study 3, asfotase alfa improved physical function and quality of life for adults with HPP. People were able to walk further and faster and had improved grip strength. The researchers saw these benefits as early as 3 months, and they continued over 2 years. The treatment was welltolerated. Some people had mild reactions at injection sites.

What do the results of the studies mean?:

The combined results from these studies indicate that asfotase alfa is effective and safe to treat people with HPP of different ages and levels of symptom severity. Asfotase alfa improves bone health, mobility, growth, and breathing ability, and relieves pain. Treatment can improve quality of life for people with HPP. The findings from these studies support the continued use and further development of asfotase alfa as a treatment for people with HPP.

2025-05-01·ADVANCES IN THERAPY

Mobility and Quality of Life in Adults with Paediatric-Onset Hypophosphatasia Treated with Asfotase Alfa: Results from UK Managed Access Agreement

Article

作者: Moss, Katie E ; Fang, Shona ; Zygouras, Alexandros ; Geberhiwot, Tarekegn ; Selby, Peter ; Bubbear, Judith S ; Walsh, Jennifer S ; Keen, Richard ; Javaid, Muhammad K ; Poole, Kenneth E S

INTRODUCTION:

Hypophosphatasia (HPP) is a rare disease caused by deficient tissue-non-specific alkaline phosphatase (ALP) activity. Asfotase alfa is a tissue-non-specific ALP enzyme-replacement therapy which was reimbursed in the UK under a Managed Access Agreement (MAA). This analysis assessed safety and effectiveness of asfotase alfa in adults with HPP.

METHODS:

This prospective, observational data collection included adults with paediatric-onset HPP enroled in the MAA and treated with asfotase alfa for ≥ 6 months to 5 years. Assessments included mobility, pain, and health-related quality of life (HRQoL), each reported at regular intervals through year 3. Analgesic use, fractures, and events of interest (EOIs) were each reported continuously throughout follow-up.

RESULTS:

Of 28 enroled treated adults, 24 were assessed for effectiveness. Distance walked in the 6-Minute Walk Test was median (min, max) 172.5 m (0.0, 380.0; n = 24) at baseline and improved by 157.3 m (- 171.0, 479.5; n = 16) at month 6; results were sustained throughout follow-up. Median (min, max) Bleck score was 6.0 (2.0, 9.0; n = 24) at baseline and increased to 6.5 (5.0, 9.0; n = 10) at month 36. Median (min, max) aggregate Brief Pain Inventory Short Form severity score was 8.0 (4.3, 10.0; n = 24) at baseline and improved to 4.4 (1.0, 7.8; n = 10) at month 36. During follow-up, 8 participants (33.3%) decreased or discontinued opioid use throughout follow-up and 4 (16.7%) reported fractures. Median (min, max) EQ-5D-3L utility scores improved from 0.21 (- 0.26, 0.60; n = 24) at baseline by 0.15 (- 0.36, 0.91; n = 24) at month 6 and were similar throughout follow-up. Injection site reactions were the most common treatment-related EOI, reported in 17 participants (60.7%). Three participants reported treatment-related serious adverse events.

CONCLUSION:

Asfotase alfa treatment improved mobility, physical function, pain, and HRQoL and was well tolerated. These data show the benefit of asfotase alfa in adults with paediatric-onset HPP.

项与阿司福酶α 相关的新闻（医药）

2025-05-09

·CPHI制药在线

图解深度分析制药巨头2025年Q1表现

关注并星标CPHI制药在线2025年第一季度，全球大型制药公司业绩呈现出明显的分化趋势。从营收体量上看，强生、罗氏和默沙东依然稳居“销售王座”；而从同比增长来看，礼来以45%的强劲增幅远远领先于其他竞争者，成为本季度最耀眼的明星（图1）。2025年Q1，强生以218.9亿美元继续领跑，略高于去年同期的213.8亿美元，增幅为2.4%。罗氏以186.8亿美元位居第二，同比增长7.2%。默沙东则以155.3亿美元排名第三，尽管营收略有下滑（同比减少1.6%），但其核心产品组合依然稳固。三家公司继续在肿瘤、自身免疫和疫苗等大病种领域占据主导地位。图1. 2025年大型制药公司Q1销售额与同比变化012025年Q1销售额“黜陟榜” 增长阵营：礼来、诺和诺德、诺华、赛诺菲、艾伯维等表现稳健多家公司录得两位数增长，显示出良好的产品线和市场适应力： ✔礼来：销售额127.3亿美元，同比增长高达惊人的45%，在所有大型制药公司中同比增长最为强劲。 ✔ 诺和诺德：同比增长率19.5%，在大型制药公司中排名第二。同时获得了118.9亿美元的季度销售业绩。 ✔ 诺华：营收132.3亿美元，同比增长11.8%。受益于心血管、眼科和创新口服药物的持续增长。 ✔ 赛诺菲：录得111.83亿美元，同比增长9.7%，主要由Dupixent和疫苗业务推动。 ✔ 艾伯维：同比增长8.4%，尽管Humira收入继续下滑，但Skyrizi与Rinvoq的组合表现强劲，部分抵消影响。 ✔ 阿斯利康与安进也分别实现7.2%和9.5%的稳健增长，前者依托肿瘤与罕见病产品线，后者则得益于骨质疏松与心血管产品市场的扩展。下滑阵营：辉瑞、BMS、百时美施贵宝、吉利德部分公司则面临增长乏力甚至下滑的困境： ✔ 辉瑞：2025年Q1营收为137.2亿美元，同比下降7.8%。随着新冠疫苗与治疗药物收入继续回落，其核心产品亟需填补“后疫情”空缺。 ✔ 百时美施贵宝（BMS）：收入下滑5.6%，主要受Revlimid专利悬崖与部分产品竞争加剧影响。 ✔ 吉利德：几乎持平，同比下降0.3%，HIV产品表现稳定，但抗病毒新管线推进缓慢。 ✔ 默沙东：虽仍排名靠前，但也同比下降1.6%，值得警惕。02大型制药公司2025Q1表现礼来2025年Q1，礼来实现营收127.3亿美元，同比增长45.0%，在所有大型制药公司中遥遥领先。这一强势表现背后，是其在代谢和体重管理领域的绝对统治力，尤其是GLP-1受体激动剂类药物的全线爆发，保证了礼来在2025年第一季度，甚至未来几年内充盈的销售额。Zepbound和Mounjaro的联合销售额已超过60亿美元，占礼来总营收的近一半，展示出GLP-1类产品对公司增长的“发动机”地位。 ● 礼来销售额最高的产品：Mounjaro（38.42亿美元，+113%） Mounjaro原用于2型糖尿病治疗，但强劲的减重效果使其被广泛“越权”使用，实际拉动了礼来在肥胖管理市场的渗透率。随着医保覆盖扩展及供货改善，该药销售额已超越传统肿瘤与免疫药物，成为礼来营收核心。 ● 礼来同比增长最高的产品：Zepbound（+347%，23.12亿美元） Zepbound是tirzepatide在减重适应症上的专属品牌，2023年底才上市，但在2025年Q1就创下23亿美元以上的销售额。其快速放量显示市场对减重治疗的巨大需求，也验证了礼来精准的商业化节奏。礼来正依靠Mounjaro与Zepbound构建起王牌组合，并逐步扩大其在代谢疾病、阿尔茨海默病和肿瘤领域的布局。如果产能与支付端不构成瓶颈，其增长有望在未来几个季度继续领跑行业。强生强生2025年Q1实现营收218.9亿美元，同比增长2.4%。作为全球收入规模最大的制药公司，强生的产品线覆盖肿瘤、自身免疫、精神疾病与细胞治疗等多个领域。当前，公司正处于“老药逐步失势、新药陆续接棒”的过渡阶段，整体业绩表现稳中有升但分化显著。● 销售额最高的产品：Darzalex（32.37亿美元，+20.2%）多发性骨髓瘤抗体药物Darzalex继续稳坐强生内部的头把交椅，增长来源于其皮下注射剂型的临床接受度提升，以及联合方案的不断拓展，在欧美市场的渗透率持续提升。● 同比增长最高的产品：Carvykti（+135%，3.69亿美元）这款BCMA靶向CAR-T疗法为强生在细胞治疗领域打开增长新篇章。尽管起步基数不高，但适应症拓展和产业化能力提升使其呈指数级增长，未来有望承担抗肿瘤业务“第二增长曲线”的角色。● 同比下降最多的产品：Stelara（-33.7%，16.25亿美元）这款自身免疫领域的明星产品受专利到期影响严重，生物类似药陆续上市，市场份额迅速被蚕食。尽管强生尝试以Tremfya接力，但目前替代幅度仍不够完全。强生正面临“旗舰产品失速+新兴药物爬坡”双重挑战。Darzalex与Carvykti为其提供稳定与希望，未来关键在于Tremfya、Spravato等产品能否接棒Stelara等逐渐式微的传统大单品。诺和诺德2025年第一季度，诺和诺德展现出持续稳健的增长势头，其业务结构进一步向GLP-1类创新药物倾斜。Ozempic维持公司最大单品地位，在糖尿病治疗市场中延续增长趋势；Wegovy则以超85%的同比增幅脱颖而出，成为体重管理领域最具动能的产品之一。与此同时，公司传统核心板块胰岛素产品整体增幅趋缓，仅录得4.4%的同比增长，反映出成熟市场竞争加剧和定价压力带来的挑战。整体来看，诺和诺德正处于产品结构调整的关键阶段，新一代GLP-1产品逐步取代胰岛素成为增长驱动力，公司未来的持续增长将更依赖于Wegovy在全球市场的渗透速度以及新适应症的开发进展。 ● 销售额最高的产品：Ozempic（51.60亿美元，+13.1%）GLP-1受体激动剂Ozempic继续作为诺和诺德营收的“顶梁柱”稳居榜首。其强劲增长得益于在全球2型糖尿病市场的广泛应用，尤其是在美国市场的处方持续增长。尽管竞争者试图进入GLP-1赛道，但Ozempic凭借先发优势与长期安全性数据，依然保持领先。 ● 同比增长最高的产品：Wegovy（+85.2%，26.44亿美元）这款以肥胖管理为主的GLP-1类药物实现了惊人的同比增长，几乎翻倍，成为公司最亮眼的新兴增长引擎。产能提升、医保覆盖扩展以及肥胖治疗理念的普及共同推动了Wegovy的爆发式增长。其强劲表现也印证了诺和诺德在体重管理市场上的战略前瞻性。诺和诺德正在经历从传统胰岛素巨头向GLP-1驱动型增长公司的转型期。Ozempic提供稳定现金流，Wegovy则展现出未来爆发潜力，两者共同撑起公司增长曲线。相较之下，胰岛素板块日益边缘化，公司未来能否维持领先地位，将取决于GLP-1类药物在肥胖、糖尿病及潜在新适应症上的持续突破与产能保障。艾伯维2025年Q1，艾伯维实现销售额133.4亿美元，同比增长8.4%，延续了温和增长趋势。虽然昔日“药王”Humira跌落神坛，但在炎症免疫、肿瘤和神经精神领域的多元布局，帮助公司成功构建了更为均衡的收入结构。Skyrizi与Rinvoq的强势放量成为公司最关键的增长动力。 ● 销售额最高的产品：Skyrizi（34.25亿美元，+70.6%）Skyrizi凭借银屑病和克罗恩病等适应症的快速扩展，继续巩固其作为艾伯维营收新支柱的地位。2025年Q1销售额已达34亿美元，首次超越Humira成为公司最赚钱的单品，其优异的疗效与较低的免疫抑制风险是市场快速接受的核心。 ● 同比增长最高的产品：Skyrizi（+70.6%）该产品不仅是收入之冠，也拥有最高的增长率。这体现了艾伯维战略上的清晰路径：以Skyrizi和Rinvoq联袂替代Humira构建新免疫双引擎，其商业推广策略已见成效。 ● 同比下降最多的产品：Humira（-50.6%，11.21亿美元）2025年Q1，Humira的营收腰斩，是艾伯维增长路上的最大拖累。这一方面反映出生物类似药在全球市场的迅速扩张（尤其是美国专利到期后的影响），另一方面也表明艾伯维未选择对Humira进行激进的价格防守，而是战略性让位于新药。艾伯维成功完成了“从单一依赖向多元稳定”的关键转型。尽管Humira退潮带来短期阵痛，但Skyrizi与Rinvoq的高增速为未来提供了持续成长基础，神经科（如Vraylar）和肿瘤产品（如Venclexta）也为其构建长期稳定组合提供了坚实支撑。罗氏2025年Q1，罗氏实现营收186.8亿美元，同比增长7.2%。作为一家以抗肿瘤药物起家的传统巨头，罗氏的营收结构正逐步发生变化：一些专利已过期的旧产品下滑明显，而部分新型抗体药和简化给药形式的组合药物成为主要增长来源。 ● 销售额最高的产品：Ocrevus（17.8亿瑞士法郎，+7.2%）Ocrevus用于治疗多发性硬化症，近年来持续增长，现已成为罗氏最畅销的产品之一。该药在欧美市场的处方数量保持稳定，治疗依从性较高，为罗氏提供了长期稳定的收入来源。 ● 同比增长最高的产品：Phesgo（+51.3%，5.9亿瑞士法郎）Phesgo是将Herceptin和Perjeta两种HER2抗体药物组合为一次皮下注射的固定联合制剂，显著缩短给药时间、简化流程。其快速增长反映出罗氏在用药便利性方面的策略取得积极成效，也部分减缓了Herceptin单品的下滑压力。 ● 同比下降最多的产品：Herceptin（-19.4%，2.9亿瑞士法郎）作为过去的明星产品，Herceptin正面临生物类似药的持续冲击，市场份额逐年下降。尽管罗氏已通过Phesgo进行产品形态上的优化接替，但Herceptin本身的衰退趋势难以逆转。此外，Avastin（-15.6%）和MabThera/Rituxan（-14.3%）等同样受生物类似药影响，收入继续萎缩。罗氏的整体业绩增长主要来自Ocrevus、Phesgo等新产品的拉动。老一代肿瘤药物正在加速退场，而新产品尚处于增长早期。要维持增长势头，罗氏仍需加快新药研发节奏，并提升新产品在市场中的占比。诺华2025年Q1，诺华实现营收132.3亿美元，同比增长11.8%，在传统跨国药企中增幅居前。这一增长主要由心血管和免疫相关创新药物驱动，多个产品线同步增长，形成较为健康的业务结构。 ● 销售额最高的产品：Entresto（22.61亿美元，+20.3%）Entresto继续作为诺华的“顶梁柱”存在，得益于其在心衰一线治疗中的临床价值和指南推荐。随着全球老龄化趋势加剧，心衰患者人群增长，Entresto渗透率进一步提升，巩固其核心地位。 ● 同比增长最高的产品：Leqvio（+70.2%，2.57亿美元）Leqvio是一种小干扰RNA（siRNA）药物，用于降脂治疗。虽然当前基数仍小，但其独特的半年给药一次机制在患者依从性方面有明显优势，显示出良好的市场接受度。未来若医保覆盖全面放开，有望成为诺华中长期增长的“后起之秀”。 ● 同比下降最多的产品：Sandostatin Group（-10.7%，3.19亿美元）该产品组合主要用于神经内分泌肿瘤等适应症，但近年来面临竞品压力、患者转向更新治疗方案等多重因素，导致销售持续下滑。尽管总体金额不大，但也反映出部分老药的增长乏力问题。诺华在2025年Q1展示了多产品稳健增长的良性态势。Entresto、Cosentyx和Kisqali等核心产品支撑稳定收入，Leqvio、Kesimpta等新药放量迅速。诺华正逐步从以专利保护为主的营收模式，向以产品力和适应症覆盖广度为基础的持续增长结构转型。阿斯利康2025年Q1，阿斯利康实现营收135.9亿美元，同比增长7.2%。公司继续稳扎稳打地推进其在肿瘤、心血管、呼吸和罕见病四大核心领域的全球业务。在一线靶向药稳定贡献的同时，Breztri、Ultomiris、Strensiq 等新兴产品成为新的增长亮点。 ● 销售额最高的产品：Tagrisso（16.79亿美元，+5.3%）Tagrisso是阿斯利康最重要的靶向抗癌药物之一，主要用于EGFR突变型非小细胞肺癌（NSCLC）治疗。虽然增长速度趋缓，但仍是公司最核心的收入来源，特别是在亚洲市场仍保持主导地位。 ● 同比增长最高的产品：Breztri（+37.0%，3.0亿美元）Breztri是一款三联吸入剂，用于慢性阻塞性肺病（COPD）治疗。凭借便利的用药方式和良好依从性，该产品在全球市场放量迅速，是呼吸产品线中新增长的重要支撑。 ● 同比下降最多的产品：Soliris（-39.9%，4.44亿美元）Soliris作为经典罕见病治疗药物，销售下滑明显，主要受到其“自家继任者”Ultomiris的替代冲击。后者以更长的给药周期和更好的依从性，正在加速蚕食Soliris的市场份额。阿斯利康整体表现稳健，核心抗癌产品提供稳定现金流，呼吸和罕见病新药增长迅速。未来增长能否持续，将取决于新产品（如Breztri、Ultomiris）能否快速覆盖更多市场，以及在ADC和RNA平台上的产品进展是否顺利落地。默沙东2025年Q1，默沙东实现销售额155.3亿美元，同比下降1.6%，是本轮榜单中少数出现负增长的跨国巨头之一。虽然抗PD-1免疫治疗药物Keytruda继续强劲增长，但疫苗板块和糖尿病产品下滑明显，对整体营收形成拖累。 ● 销售额最高的产品：Keytruda（72.05亿美元，+3.7%）Keytruda依然是默沙东的绝对领袖，占公司季度营收近一半。得益于在肺癌、黑色素瘤、胃癌等多个适应症中的广泛应用，其销售额持续走高，已成为全球范围内收入最高的抗癌药之一。尽管增长已放缓，但其临床优势和适应症广度仍能保持长期稳定表现。 ● 同比增长最高的产品：Januvia/Janumet（+18.8%，7.96亿美元）作为DPP-4类降糖药，这一组合的反弹增长较为意外，主要源于部分市场中的医保谈判成功及仿制药竞争阶段性缓解。不过，其长期增长仍受限于GLP-1类药物快速崛起的市场趋势。 ● 同比下降最多的产品：Gardasil/Gardasil 9（加卫苗）（-41.0%，13.27亿美元）HPV疫苗季度销售同比大幅下滑，受制于中国库存消化、渠道调整及2024年同期高基数影响。尽管产品长期需求仍然旺盛，但短期内波动较大，对整体疫苗业务造成明显影响。默沙东对Keytruda的依赖度过高，且HPV疫苗与其他产品线短期承压，使得整体营收面临增长瓶颈。要实现更均衡的发展，公司需加快ADC、RNA平台和新兴肿瘤靶点的布局，并降低对单一产品的结构性依赖。安进2025年Q1，安进实现营收81亿美元，同比增长9.5%。作为以生物制剂为核心优势的药企，安进本季度在骨代谢、免疫及肿瘤治疗等多个领域均有产品实现增长，销售表现稳定，结构日益多元。 ● 销售额最高的产品：Prolia（10.99亿美元，+10.0%）Prolia为抗RANKL骨质疏松治疗药物，长期处于安进产品线中的“顶流”。得益于全球范围内老龄化加剧、骨折风险人群扩大，其销售保持温和增长。临床依从性较好，是骨代谢板块最具商业价值的产品。 ● 同比增长最高的产品：Tezspire（+64.7%，2.85亿美元）Tezspire是一种用于重度哮喘治疗的单克隆抗体，由安进与阿斯利康联合开发。其上市时间尚短，目前处于快速放量期，凭借不依赖特定生物标志物的机制，正在打开更广泛患者人群。 ● 同比下降最多的产品：Kyprolis（-13.8%，3.24亿美元）Kyprolis为多发性骨髓瘤治疗药物，当前正面临激烈的市场竞争，包括同类蛋白酶体抑制剂与新兴CAR-T疗法的冲击，导致其销售下滑较为明显。安进目前销售结构较为多元，老牌产品如Prolia、Repatha保持稳定，新兴抗体药物如Tezspire、Blincyto呈现快速增长态势。尽管部分肿瘤产品面临竞争，但整体收入基础稳固，增长动力正逐步向新一代创新产品转移。辉瑞2025年Q1，辉瑞实现营收137.2亿美元，同比下降7.8%，为本季度跌幅最大的头部药企之一。随着新冠相关业务大幅回落，辉瑞正逐步依靠心血管、肿瘤和疫苗等常规产品线支撑业绩，多数产品增长幅度温和，但整体转型尚处于过渡阶段。 ● 销售额最高的产品：Eliquis（19.23亿美元，-5.7%）Eliquis为抗凝药，由辉瑞与BMS联合推广。虽然其临床地位稳固，但受仿制药预期和医保价格压缩影响，2025年Q1销售额略有下降，未来增长潜力有限，面临阶段性平台期。 ● 同比增长最高的产品：Comirnaty（+59.6%，5.65亿美元）作为辉瑞的mRNA疫苗品牌，Comirnaty在2024年大幅回落后，在部分新兴市场及南半球流感季出现反弹。虽然收入不及疫情高峰期，但该产品仍为辉瑞提供重要补充现金流。 ● 同比下降最多的产品：Paxlovid（-75.9%，4.91亿美元）Paxlovid为新冠口服抗病毒药物，随着新冠疫情防控常态化，全球用药需求大幅减少。2025年Q1销售额大幅下滑，几乎失去对辉瑞营收的边际贡献，标志其新冠板块“退潮”基本结束。辉瑞正面临新冠红利消退后的重构挑战。虽然Vyndaqel、Xtandi、Padcev 等创新药物保持增长，但难以完全对冲Paxlovid与Comirnaty退潮带来的空缺。未来业绩能否回升，取决于肿瘤、罕见病、RNA平台和ADC等新产品的推进速度。吉利德2025年Q1，吉利德实现营收66.7亿美元，同比微降0.3%，总体表现持平。公司仍主要依赖HIV抗病毒药物维持核心收入，但新冠治疗药物Veklury下降明显，影响了整体表现。 ● 销售额最高的产品：Biktarvy（31.5亿美元，+6.9%）Biktarvy继续作为吉利德的王牌HIV复方口服药物，稳居营收榜首。其单日一片、疗效强、耐药率低等特点，使其在全球HIV治疗方案中长期处于领先地位。 ● 同比增长最高的产品：Descovy（+37.6%，5.86亿美元）Descovy用于HIV暴露前预防（PrEP）以及治疗组合中的一部分。其增长反映出PrEP市场渗透率提升，同时也体现出公司在HIV防治全周期产品布局的优势。 ● 同比下降最多的产品：Veklury（-45.6%，5.55亿美元）该产品为新冠病毒治疗的早期主力药物，随着疫情相关住院治疗需求下降，其销售额大幅回落，几乎减半。Veklury的收入已不再是公司增长的重要来源。吉利德当前收入结构仍以HIV治疗为核心，Biktarvy和Descovy构成稳定增长基础。Veklury等疫情类产品退潮带来一定影响，而Yescarta等细胞治疗产品体量尚小，短期内对营收拉动作用有限。未来增长仍需依赖HIV管线的持续稳健与肿瘤产品的突破。赛诺菲2025年Q1，赛诺菲实现营收111.83亿美元，同比增长9.7%。得益于核心产品Dupixent的稳定扩张，以及ALTUVIIIO、Beyfortus等新产品的快速放量，公司整体呈现出积极的增长态势，业务结构向高附加值创新药转型。 ● 销售额最高的产品：Dupixent（34.8亿欧元，+20.3%）Dupixent继续稳居赛诺菲收入榜首，主要用于治疗特应性皮炎、哮喘和慢性鼻窦炎等免疫疾病。其持续的适应症扩展及全球市场渗透力，是推动公司整体增长的核心动力。 ● 同比增长最高的产品：ALTUVIIIO（+100%，2.51亿欧元）ALTUVIIIO为8因子重组蛋白，用于血友病A的预防性治疗。该药2023年获批上市后快速放量，显示出新一代长效凝血因子产品的强劲市场需求。 ● 同比下降最多的产品：Lovenox（-6.5%，2.38亿欧元）Lovenox为低分子肝素类抗凝药，属于赛诺菲经典产品之一。近年来在多国受到价格压制和替代性产品挑战，销售额持续下滑。赛诺菲正逐步从传统处方药业务中脱离，向创新免疫治疗和罕见病领域转型。Dupixent依然是赛诺菲需要倚重的支柱型产品。百时美施贵宝2025年Q1，百时美施贵宝实现营收112亿美元，同比下降5.6%。作为曾依靠Revlimid、Eliquis撑起业绩高峰的公司，BMS当前正处于“高峰期产品滑落+新产品爬坡”的过渡阶段。尽管免疫肿瘤产品组合表现稳定，但Revlimid等老产品的下滑冲击依然明显。 ● 销售额最高的产品：Eliquis（35.65亿美元，-4%）作为全球最畅销的口服抗凝药物之一，Eliquis销售额依然排名第一。但随着专利挑战临近和价格压力上升，其增长遇阻，收入略有下滑。 ● 同比增长最高的产品：Breyanzi（+146%，2.63亿美元）Breyanzi是靶向CD19的CAR-T细胞治疗产品，适应症涵盖多种类型的复发难治性B细胞恶性肿瘤。在全球多个市场获批后逐步放量，2025年Q1实现146%的增长，体现出BMS在细胞治疗领域的突破进展。 ● 同比下降最多的产品：Revlimid（-44%，9.36亿美元）作为曾经的“超级药王”，Revlimid受专利到期和仿制药竞争影响持续下滑，季度销售额同比下降44%，是拖累整体收入的主因之一。BMS当前产品组合呈现新旧分化明显的格局。Opdivo、Yervoy 等免疫检查点抑制剂维持稳定增长，Breyanzi 等新兴疗法开始提供新的拉动点。但Revlimid、Eliquis等产品的退坡对整体构成压力，未来增长将更多依赖细胞治疗与新一代肿瘤靶点药物的商业化推进。03亢龙有悔，无可奈何花落去的支柱资产在本季度各大药企的销售构成中，可以看到一类值得特别关注的现象：一些在公司整体营收中占据显著比重的支柱型产品出现了同比下滑（图3）。这些产品往往是企业近年来最成功的商业资产，代表着其在核心治疗领域的竞争优势，也常常承担着稳定现金流和利润来源的角色。图3. 负增长的支柱型资产气泡图（气泡大小代表该资产销售额规模）BMS 的 Eliquis 单季度销售额达 35.65 亿美元，占公司总营收的 31.8%，但同比下降了 4%。辉瑞的 Eliquis 也出现类似趋势，在其产品组合中收入排名第一，却录得 5.7% 的负增长。这类产品虽未出现大幅滑落，但因其基数庞大，哪怕个位数下滑也会对整体营收形成压力。另一类则是面临结构性调整的产品，如 BMS 的 Revlimid（-44%）与辉瑞的 Paxlovid（-75.9%），它们分别受到专利到期和疫情需求回落的影响，收入大幅下滑；但它们曾在过去多个财季中为公司业绩增长做出决定性贡献，因此其下滑标志着“增长来源的迁移”，而非简单的短期波动。还有一些产品则处于专利保护期结束后的典型“退坡阶段”，例如强生的 Stelara（16.25 亿美元，-33.7%），专利在2023年底失效，多个生物类似药已在美国和欧洲布局上市。这一阶段的收入下降不仅来自定价压力，也源于市场份额的结构性转移。类似的还有默沙东的 Gardasil 9（-41.0%），虽仍为公司疫苗板块的中坚，但本季度因中国市场库存调整及高基数效应影响，表现波动明显。老药王，艾伯维的 Humira（-50.6%）是本轮结构性下滑最典型的代表之一。尽管销售额仍达 11.21 亿美元，但由于专利保护陆续失效，其在北美市场已全面进入生物类似药竞争阶段，季度同比收入腰斩，标志其“后药王”周期也逐步进入暮年。这些变化提示我们，药企在应对“退坡型核心资产”时的接替策略至关重要。是通过差异化新药快速接棒，还是延长现有品种的生命周期（如新剂型、联合治疗、适应症扩展），将决定其营收结构是否能保持长期平衡。04飞龙在天，销售额大幅增长的支柱产品与部分核心产品进入调整期不同，另一些重磅产品在本季度不仅保持了两位数以上的同比增长，还具备10亿美元以上的销售体量，继续构成公司整体收入的重要支撑（图4）。它们代表了各大药企目前最稳健的增长基础。图4. 显著增长的支柱型资产气泡图（气泡大小代表该资产销售额规模）图4展示了2025年第一季度销售额同比显著增长的核心产品，从中可以看出，诺和诺德与礼来在新一代代谢类药物领域的领先优势正在快速转化为实际营收。诺和诺德的 Ozempic 和 Wegovy 分别实现了51.60亿美元和26.44亿美元的季度销售额，同时对公司整体营收的贡献比例也达到高位，凸显 GLP-1 类药物在糖尿病和减重治疗市场中的主导地位。礼来的 Mounjaro 和 Zepbound 分别录得38.42亿美元和23.12亿美元的销售额，与 Verzenio 一道，支撑起礼来在代谢和肿瘤领域的多点增长格局。艾伯维旗下的 Skyrizi 与 Rinvoq 延续强劲表现，合计带来超过50亿美元的季度收入，且其公司内部营收占比持续提升，说明在 Humira 专利悬崖之后，接替产品的增长动能正在稳步确立。赛诺菲的 Dupixent 在多项适应症拓展的带动下表现出色，季度销售额达到45.23亿美元（34.8亿欧元），稳居呼吸与免疫类慢病治疗的重要支柱。其他产品如安进的 Prolia、阿斯利康的 Ultomiris、罗氏的 Vabysmo、以及百健的 Qalsody 虽然体量尚不及头部产品，但在各自领域的增长趋势清晰，反映出中型产品逐步成为公司增长主力的过程正在展开。整体来看，这些同比增长显著且占比提升的产品构成了企业营收的关键来源，也反映出当前全球主要制药公司在产品布局与战略重心上的转变方向。END【企业推荐】领取CPHI & PMEC China 2025展会门票来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~

疫苗财报

2025-02-06

·Business Wire

AstraZeneca’s Full Year and Q4 2024 results

CAMBRIDGE, England--( BUSINESS WIRE )--AstraZeneca: Revenue and EPS summary FY 2024 % Change Q4 2024 % Change $m Actual CER i $m Actual CER - Product Sales 50,938 16 19 13,362 18 19 - Alliance Revenue 2,212 55 55 714 68 69 - Collaboration Revenue 923 56 54 815 >2x >2x Total Revenue 54,073 18 21 14,891 24 25 Reported EPS $4.54 18 29 $0.97 56 71 Core ii EPS $8.21 13 19 $2.09 44 49 Financial performance for FY 2024 (Growth numbers at constant exchange rates) Total Revenue up 21% to $54,073m, driven by a 19% increase in Product Sales, continued growth of partnered medicines (Alliance Revenue) and the achievement of sales-based milestones (Collaboration Revenue) Total Revenue growth from Oncology was 24%, CVRM 20%, R&I 25%, V&I 8% and Rare Disease 16% Core EPS increased 19% to $8.21 Second interim dividend declared of $2.10 per share, making a total annual dividend declared for FY 2024 of $3.10 per share, an increase of 7%. Dividend to be further increased in FY 2025 Guidance for FY 2025: Total Revenue is expected to increase by a high single-digit percentage and Core EPS is expected to increase by a low double-digit percentage, both at CER Pascal Soriot, Chief Executive Officer, AstraZeneca, said: “ Our company delivered a very strong performance in 2024 with Total Revenue and Core EPS up 21% and 19% respectively. We also delivered nine positive high value Phase III studies in the year, which coupled with increasing demand for our medicines in all key regions, will help sustain our growth momentum into 2025. This year marks the beginning of an unprecedented, catalyst-rich period for our company, an important step on our Ambition 2030 journey to deliver $80 billion Total Revenue by the end of the decade. In 2025 alone, we anticipate the first Phase III data for seven new medicines, along with several important new indication opportunities for our existing medicines. We are also investing in and making significant progress with transformative technologies that have the potential to drive our growth well beyond 2030, many of which have now entered pivotal trials.” Key milestones achieved since the prior results announcement Positive read-outs for Truqap in combination with abiraterone and androgen deprivation therapy in PTEN -deficient de novo metastatic hormone-sensitive prostate cancer (CAPItello-281) and Tagrisso with or without chemotherapy in resectable early-stage EGFR m NSCLC (NeoADAURA) US approvals for Imfinzi in limited-stage small cell lung cancer (ADRIATIC), Calquence in combination with bendamustine and rituximab in mantle cell lymphoma (ECHO), Datroway (datopotamab deruxtecan) in HR+ HER2- metastatic breast cancer (TROPION-Breast01) and Enhertu in chemotherapy-naïve HER2-low and -ultralow metastatic breast cancer (DESTINY-Breast06). EU approvals for Tagrisso in unresectable EGFR m NSCLC (LAURA) and Kavigale for prevention of COVID-19 (SUPERNOVA). Japan approvals for Imfinzi in endometrial cancer (DUO-E), Lynparza plus Imfinzi in pMMR endometrial cancer (DUO-E), Calquence tablet formulation in chronic / small lymphocytic leukaemia, Datroway in HR+ HER2- metastatic breast cancer, Fasenra in EGPA (MANDARA) and Kavigale for prevention of COVID-19. China approvals for Lynparza in gBRCAm HER2- early breast cancer (OlympiA), Orpathys in locally advanced or metastatic MET Exon 14 NSCLC (NCT04923945) Guidance The Company issues its Total Revenue and Core EPS guidance for FY 2025 at CER, based on the average foreign exchange rates through 2024. Total Revenue is expected to increase by a high single-digit percentage Core EPS is expected to increase by a low double-digit percentage The Core Tax rate is expected to be between 18-22% The Company is unable to provide guidance on a Reported basis because it cannot reliably forecast material elements of the Reported results, including any fair value adjustments arising on acquisition-related liabilities, intangible asset impairment charges and legal settlement provisions. Please refer to the cautionary statements section regarding forward-looking statements at the end of this announcement. Currency impact If foreign exchange rates for February 2025 to December 2025 were to remain at the average rates seen in January 2025, it is anticipated that Total Revenue in FY 2025 would incur a low single-digit percentage adverse impact compared to the performance at CER, and Core EPS would incur a mid-single-digit percentage adverse impact. The Company’s foreign exchange rate sensitivity analysis is provided in Table 17. Capital allocation In FY 2025, the Company intends to increase the annual dividend declared to $3.20 per share. The Company also expects to increase capital expenditure iii by approximately 50%, driven by manufacturing expansion projects and investment in IT systems, to support portfolio growth and build capacity for transformative technologies. China In relation to the illegal drug importation allegations, in January 2025, AstraZeneca received a Notice of Transfer to the Prosecutor and an Appraisal Opinion from the Shenzhen City Customs Office regarding suspected unpaid importation taxes amounting to $0.9 million. To the best of AstraZeneca’s knowledge, the importation taxes referred to in the Appraisal Opinion relate to Imfinzi and Imjudo . A fine of between one and five times the amount of unpaid importation taxes may also be levied if AstraZeneca is found liable. AstraZeneca continues to fully cooperate with the Chinese authorities. In December 2024 AstraZeneca announced the appointment of Iskra Reic as Executive Vice President, International, which encompasses China, Asian and Eurasian markets, Middle East & Africa, Latin America, Australia & New Zealand. Iskra succeeds Leon Wang who is on extended leave from the Company while under investigation in China. Table 1: Key elements of Total Revenue performance in Q4 2024 % Change Revenue type $m Actual % CER % Product Sales 13,362 18 19 Alliance Revenue 714 68 69 $392m Enhertu (Q4 2023: $281m) $133m Tezspire (Q4 2023: $80m) $161m Beyfortus (Q4 2023: $41m) Collaboration Revenue 815 >2x >2x $600m Lynparza (Q4 2023: $245m) $111m Beyfortus (Q4 2023: $27m) $100m Koselugo (Q4 2023: nil) Total Revenue 14,891 24 25 Therapy areas $m Actual % CER % Oncology 6,344 27 29 Tagrisso up 20% (21% at CER), Calquence up 20%, Enhertu up 48% (54% at CER) CVRM 3,138 16 17 Farxiga up 21% (22% at CER) , Lokelma up 35% R&I 2,127 27 28 Breztri up 29%. Saphnelo up 65%, Tezspire up 86% (85% at CER), Symbicort up 31% (33% CER) V&I 651 58 55 Beyfortus Total Revenue up >3x Rare Disease 2,377 21 22 Ultomiris up 32% (33% at CER), partially offset by decline in Soliris of 24% (22% at CER), Strensiq up 38% (37% at CER) and Koselugo up >3x Other Medicines 254 (7) (6) Total Revenue 14,891 24 25 Regions $m Actual % CER % US 6,532 28 28 Product Sales up 25% Emerging Markets 3,134 13 19 - China 1,364 (1) (3) Decline primarily due to low rates of seasonal respiratory viral infections, and impact from year-end hospital budget dynamics - Ex-China Emerging Markets 1,770 26 42 Europe 3,948 37 35 Product Sales up 20% (18% at CER) Established RoW 1,277 1 2 Total Revenue 14,891 24 25 Key alliance medicines Combined sales of Enhertu , recorded by Daiichi Sankyo Company Limited (Daiichi Sankyo) and AstraZeneca, amounted to $3,754m in FY 2024 (FY 2023: $2,566m) Combined sales of Tezspire , recorded by Amgen and AstraZeneca, amounted to $1,219m in FY 2024 (FY 2023: $653m) Table 2: Key elements of financial performance in Q4 2024 Metric Reported Reported change Core Core change Comments iv Total Revenue $14,891m 24% Actual 25% CER $14,891m 24% Actual 25% CER See Table 1 and the Total Revenue section of this document for further details Product Sales Gross Margin 80% Stable Actual +1pp CER 79% -1pp Actual Stable CER Variations in Product Sales Gross Margin can be expected between periods, due to product seasonality, foreign exchange fluctuations and other effects R&D expense $4,677m 52% Actual 52% CER $3,573m 23% Actual 22% CER Increased investment in the pipeline Core R&D-to-Total Revenue ratio of 24% (Q4 2023: 24%) Reported R&D includes $753m impairment recorded against the vemircopan (ALXN2050) intangible asset SG&A expense $5,410m 1% Actual 1% CER $4,275m 6% Actual 7% CER Market development for recent launches and pre-launch activities Core SG&A-to-Total Revenue ratio of 29% (Q4 2023: 34%) Other operating income and expense v $100m -7% Actual -6% CER $101m -7% Actual -6% CER Operating Margin 14% +3pp Actual +4pp CER 28% +5pp Actual +6pp CER See commentary above on Gross Margin, R&D, SG&A and Other operating income and expense Net finance expense $365m 9% Actual 8% CER $310m 20% Actual 20% CER Recent debt issued at higher interest rates Decrease in interest income Higher level of Net debt Tax rate 10% +17pp Actual +15pp CER 16% +7pp Actual +7pp CER Variations in the tax rate can be expected between periods EPS $0.97 56% Actual 71% CER $2.09 44% Actual 49% CER Further details of differences between Reported and Core are shown in Table 12 Table 3: Pipeline highlights since prior results announcement Event Medicine Indication / Trial Event Regulatory approvals and other regulatory actions Tagrisso EGFR m NSCLC (Stage III unresectable) (LAURA) Regulatory approval (EU, CN) Imfinzi Limited-stage SCLC (ADRIATIC) Regulatory approval (EU) Imfinzi Advanced endometrial cancer Regulatory approval (JP) Calquence Tablets for chronic lymphocytic leukaemia Regulatory approval (JP) Calquence Mantle cell lymphoma (1st-line) (ECHO) Regulatory approval (US) Lynparza + Imfinzi Advanced endometrial cancer with mismatch repair proficiency (DUO-E) Regulatory approval (JP) Lynparza gBRCAm HER2- eBC (OlympiA) Regulatory approval (CN) Enhertu HR+ HER2-low and -ultralow mBC (DESTINY-Breast06) Regulatory approval (US) Datroway HR+ HER2- mBC (TROPION-Breast01) Regulatory approval (JP, US) Orpathys MET exon 14 skipping altered NSCLC (NCT04923945) Regulatory approval (CN) Fasenra EGPA (MANDARA) Regulatory approval (JP) Kavigale Prevention of COVID-19 (SUPERNOVA) Regulatory approval (EU, JP) Regulatory submissions or acceptances* Imfinzi Muscle-invasive bladder cancer (NIAGARA) Regulatory submission (US, JP) Imfinzi + Imjudo NSCLC (1st-line) (POSEIDON) Regulatory submission (CN) Calquence Chronic lymphocytic leukaemia (1st-line) (AMPLIFY) Regulatory submission (EU) Datroway EGFR m NSCLC (later line) (TROPION-Lung05) Regulatory submission (US) Tezspire Severe uncontrolled asthma (NAVIGATOR/ DIRECTION) Regulatory submission (CN) Koselugo Neurofibromatosis type 1 adult (KOMET) Regulatory submission (EU, JP) Phase III / registrational data readouts and other developments Tagrisso Resectable early-stage EGFR m NSCLC (NeoADAURA) Primary endpoint met Truqap PTEN -deficient de novo metastatic hormone-sensitive prostate cancer (CAPItello-281) Primary endpoint met *US, EU and China regulatory submission denotes filing acceptance Other pipeline updates In January 2025, the vemircopan (ALXN2050) Phase II development programme was terminated. The decision was based on safety and efficacy data from Phase II trials. Upcoming pipeline catalysts For recent trial starts and anticipated timings of key trial readouts, please refer to the Clinical Trials Appendix, available on www.astrazeneca.com/investor-relations.html . Sustainability highlights The Company convened an event on health equity for investors and analysts in November that detailed AstraZeneca’s health equity strategy, which is embedded from the Company’s science through to healthcare delivery and community engagement. At the end of 2024, the Company’s cumulative reduction in Scope 1 and 2 greenhouse gas (GHG) emissions was 77.5% from the 2015 baseline. Conference call A conference call and webcast for investors and analysts will begin today, 6 February 2025, at 11:00 UK time. Details can be accessed via astrazeneca.com . Reporting calendar The Company intends to publish its Q1 2025 results on 29 April 2025. To read AstraZeneca's Full Year and Q4 2024 Financial Results press release in full, click here .

临床2期财报高管变更上市批准临床3期

2024-12-02

·蒲公英Ouryao

最新粤港澳大湾区内地九市临床急需进口港澳药品医疗器械目录

转自：广东省药监局编辑：水晶 12月2日，广东省药监局网省消息，广东省药监局和广东省卫健委发布粤港澳大湾区内地九市临床急需进口港澳药品医疗器械目录（2024年）的通告。《通告》指出，本批目录收录34个上市药品及37个医疗器械。对已列入目录的药械产品，将于2024年12月1日起按照《条例》第九条进行管理。 34个药品：卡那奴单抗、维莫德吉胶囊、盐酸维那卡兰注射液、阿仑珠单抗、罗氟司特片、氨己烯酸薄膜衣片、卡博替尼薄膜衣片、阿培利司薄膜衣片、厄达替尼片、注射用羟钴胺素、布西珠单抗、伊匹木单抗注射液、巴氯芬注射液、艾沙妥昔单抗注射用浓缩液、肾上腺素注射液（预充笔）、莱博雷生片、瑞玛奈珠单抗、硫酸瑞美吉泮口崩片、注射用坦昔妥单抗、二十碳五烯酸乙酯软胶囊、阿扎胞苷片、曲美木单抗、注射用芦比替定、阿司福酶α注射液、阿伏利尤单抗、特泽利尤单抗、磷酸芦可替尼乳膏、罗莫佐单抗、重组人促卵泡素α/促黄体素α注射液、注射用重组人促卵泡素α/促黄体素α、艾普奈珠单抗、伊曲莫德片、依洛硫酸酯酶α注射液、维替索妥尤单抗。根据《广东省粤港澳大湾区内地九市进口港澳药品医疗器械管理条例》（以下简称《条例》）中对急需港澳药械实施目录管理的要求，广东省药品监督管理局、广东省卫生健康委员会经研究决定发布广东省粤港澳大湾区内地九市临床急需港澳药品医疗器械目录（2024年）。本批目录将按《国家药品监督管理局国家卫生健康委员会关于临床急需境外新药审评审批相关事宜的公告》（2018年第79号）发布并符合《条例》要求的港澳已上市药品及《条例》实施前已批准的共71个药品及医疗器械收录。对已列入目录的药械产品，将于2024年12月1日起按照《条例》第九条进行管理。特此通告。附件：1.广东省粤港澳大湾区内地九市临床急需进口港澳药品目录（2024年） 2.广东省粤港澳大湾区内地九市临床急需进口港澳医疗器械目录（2024年）广东省药品监督管理局广东省卫生健康委员会 2024年11月29日

100 项与阿司福酶α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10595	阿司福酶α	A16AB13	DB09105

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
婴儿低磷酸酯脢症	美国	Alexion Pharmaceuticals, Inc.	2015-10-23
低碱性磷酸酶血症	日本	Alexion Pharmaceuticals, Inc.	2015-07-03

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
儿童低磷酸酯脢症	临床2期	美国	Alexion Pharmaceuticals, Inc.	2016-06-06
儿童低磷酸酯脢症	临床2期	德国	Alexion Pharmaceuticals, Inc.	2016-06-06
遗传性热变性异形红细胞增多症	临床2期	美国	Alexion Pharmaceuticals, Inc.	2008-09-01
遗传性热变性异形红细胞增多症	临床2期	加拿大	Alexion Pharmaceuticals, Inc.	2008-09-01
遗传性热变性异形红细胞增多症	临床2期	阿拉伯联合酋长国	Alexion Pharmaceuticals, Inc.	2008-09-01
遗传性热变性异形红细胞增多症	临床2期	英国	Alexion Pharmaceuticals, Inc.	2008-09-01

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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


ESPE2023	N/A	低碱性磷酸酶血症 alkaline phosphatase (ALP) \| PEA \| B6 vitamin	-	Asfotase alfa	構鏇窪遞廠範窪糧顧築(鏇積獵糧鬱淵鹽製構積) = 鬱餘廠壓鬱獵廠蓋膚膚範鑰齋鑰顧願窪壓襯範 (築製觸憲糧積積廠齋壓 ) 更多	-	2023-09-21
PROSPECTIVE STUDY (WCO_IOF_ESCEO2023)	N/A	-	50	Asfotase alfa (AA)	窪齋鬱憲獵構糧積窪鏇(願鏇齋餘顧壓鬱製繭獵) = 鬱糧窪齋壓淵鑰蓋膚糧壓膚憲積範憲憲製膚壓 (夢選蓋網鏇蓋襯蓋鹽夢 ) 更多	积极	2023-05-04
WCO_IOF_ESCEO2023	N/A	儿童低磷酸酯脢症 myelinotoxic activity (MTA)	110	ASFOTASE ALFA (Group 1)	網鹹選糧繭鏇鬱淵選廠(鑰築繭衊齋鑰鹹築鏇襯) = 觸遞簾糧餘淵積糧夢齋醖夢廠齋壓積糧窪餘糧 (憲構醖齋艱醖膚構積憲, 0.7)	积极	2023-05-04
WCO_IOF_ESCEO2023	N/A	儿童低磷酸酯脢症 myelinotoxic activity (MTA)	110	ASFOTASE ALFA (Group 2)	網鹹選糧繭鏇鬱淵選廠(鑰築繭衊齋鑰鹹築鏇襯) = 廠齋壓艱衊蓋鑰艱蓋夢醖夢廠齋壓積糧窪餘糧 (憲構醖齋艱醖膚構積憲, 0.81)	积极	2023-05-04
UK Managed Access Agreement (WCO_IOF_ESCEO2023)	N/A	低碱性磷酸酶血症	25	Asfotase alfa	觸襯鏇積淵壓淵糧憲餘(繭製構衊廠願窪憲範蓋) = 衊鏇構齋夢鹽蓋齋鬱夢範糧製遞艱廠淵選餘鹽 (鑰積窪簾齋獵願構積鏇 )	积极	2023-05-04
PROSPECTIVE STUDY (WCO_IOF_ESCEO2023)	N/A	儿童低磷酸酯脢症	50	Asfotase alfa	憲築夢簾淵選廠顧夢製(鹽範衊選夢糧蓋壓鏇糧) = 窪簾獵遞鹽網願鹹願齋襯網襯窪鹹簾淵夢獵範 (鹽製淵製獵範製繭夢遞 ) 更多	积极	2023-05-04
UK Managed Access Agreement (WCO_IOF_ESCEO2023)	N/A	低碱性磷酸酶血症	25	Asfotase alfa	壓積範醖製願膚餘鏇膚(餘襯範鏇壓顧顧窪齋構) = 壓積糧鑰餘顧餘遞膚餘憲窪築鬱構鹽鏇鬱鹽鏇 (選範餘積選餘獵齋繭選 )	积极	2023-05-04
NCT01176266 (Pubmed \| J Clin Endocrinol Metab) 人工标引	临床2期	低碱性磷酸酶血症	69	Asfotase Alfa	網願壓選壓淵築憲淵廠(願簾積鹽憲製蓋製範構) = 鬱觸製艱鹹繭廠築廠網觸膚鹹網築衊壓願餘築 (淵選醖願壓鏇鑰艱鹹餘 ) 更多	积极	2019-07-01
NCT01205152 \| EUCTR2009-009369-32-GB (Pubmed) 人工标引	临床2期	低碱性磷酸酶血症	11	Asfotase alfa	窪範夢構膚憲築獵糧繭(壓製壓願製鏇製鹽繭選) = 膚簾鹽壓衊鏇願蓋製壓築淵積糧範蓋獵範淵壓 (鬱鹹糧簾獵憲繭選選範 ) 更多	积极	2019-02-01
ESPE2018	N/A	低碱性磷酸酶血症 ALPL	-	Asfotase Alfa	壓遞餘網齋範範觸鹽衊(網繭衊糧製襯淵窪簾蓋) = The patient needed a wheelchair even for in-home movement, suffered from continuous skeletal pain, had undetectable ALP serum levels and extremely high plasma PLP (997 mcg/l) 遞鏇鑰遞齋遞窪膚選獵 (網鹽築鬱獵獵襯製鑰窪 )	-	2018-09-27
NCT02797821 (CTgov)	临床2期	儿童低磷酸酯脢症	27	Asfotase alfa (Asfotase Alfa 0.5 mg/kg Dose)	夢築網遞蓋夢範網衊鹹(壓糧觸窪網蓋餘鬱製膚) = 築蓋齋製積壓網醖壓壓鹽餘廠壓遞蓋衊鏇衊壓 (鬱窪鏇鑰網獵鹹鏇夢獵, 0.2399) 更多	-	2018-07-10
NCT02797821 (CTgov)	临床2期	儿童低磷酸酯脢症	27	Asfotase alfa (Asfotase Alfa 2.0 mg/kg Dose)	夢築網遞蓋夢範網衊鹹(壓糧觸窪網蓋餘鬱製膚) = 鬱蓋鑰鹽壓餘襯鏇蓋蓋鹽餘廠壓遞蓋衊鏇衊壓 (鬱窪鏇鑰網獵鹹鏇夢獵, 0.1949) 更多	-	2018-07-10