A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)

This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT.
Study details include:
* The study duration: total study duration is approximately 64 weeks.
* Screening period of up to 8 weeks
* Treatment period of 52 weeks
* Follow-up period of 4 weeks.
* The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.

开始日期2025-03-27

申办/合作机构

Genzyme Corp.

NCT06121011 / RecruitingN/A

A Global Prospective Observational Registry of Patients With Pompe Disease

This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate.
The objectives of the registry are:
* To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/serious adverse events (SAEs) occurring in Pompe disease patients
* To evaluate the long-term real-world effectiveness of Pompe disease treatments
* To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and patient-reported outcomes (PROs)
* To describe the natural history of untreated Pompe disease

开始日期2024-02-16

申办/合作机构

Amicus Therapeutics, Inc.

NCT05734521 / RecruitingN/A

A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviazyme/Nexviadyme (Avalglucosidase Alfa-ngpt/Avalglucosidase Alfa) During Pregnancy and/or Lactation in the Postmarketing Setting

This is a worldwide, descriptive safety study collecting data on women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation, to assess the risks of avalglucsodiase alfa on pregnancy and maternal complications and adverse effects in the developing fetus, neonate, and infant.
* Outcomes in exposed infants, including growth and development, will be assessed through at least the first year of life.
* Data will be collected for approximately 10 years.

开始日期2022-10-26

申办/合作机构

Sanofi

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项与艾夫糖苷酶α 相关的文献（医药）

2024-12-01·Molecular Genetics and Metabolism Reports

Optimizing clinical outcomes: The journey of twins with CRIM-negative infantile-onset Pompe disease on high-dose enzyme replacement therapy and immunomodulation

Article

作者: Desai, Ankit K. ; Case, Laura E. ; Koch, Rebecca L ; Lisi, Matthew T ; Klinepeter, Amy ; Sharon, Cassie ; Koch, Rebecca L. ; Desai, Ankit K ; Case, Laura E ; Lisi, Matthew T. ; Kishnani, Priya S ; Fares, Angie H ; Kishnani, Priya S. ; Kirby, Amelia ; Fares, Angie H.

Infantile-onset Pompe disease (IOPD) is caused by a deficiency in the enzyme acid alpha-glucosidase (GAA). It is characterized by severe and progressive hypertrophic cardiomyopathy and muscle weakness with death in the first 2 years of life if left untreated. Enzyme replacement therapy (ERT) with alglucosidase-alfa is lifesaving, but its effectiveness is influenced by the patient's cross-reactive immunologic material (CRIM) status, dose of ERT, and the development of high antibody titers, which can reduce the therapy's efficacy. The inability of CRIM-negative IOPD patients to produce native GAA exposes them to a high risk of development of anti-rhGAA IgG antibody titers, leading to treatment failure. We present the case of CRIM-negative dizygotic twins treated with high-dose alglucosidase-alfa (40 mg/kg/week), initiated at 28 days (Twin A) and 44 days (Twin B). Both twins received immune tolerance induction (ITI) with rituximab, methotrexate, and IVIG to mitigate antibody response. Initial evaluations revealed elevated left ventricular mass index (LVMI) and elevated biomarkers (urine glucose tetrasaccharide (Glc₄), creatine kinase (CK), and aspartate aminotransferase (AST)) in both twins. Following treatment, cardiac function and biomarkers normalized within several months, with a slight delay in Twin B compared to Twin A, likely attributed to the later initiation of ERT. Both twins safely tolerated ITI, achieving immune tolerance with low antibody titers. At 28 months, the twins transitioned to avalglucosidase-alfa (40 mg/kg every other week (EOW)), which was well tolerated without an increase in antibody titers. At 39 months, both twins exhibited normal cardiac function, LVMI, and biomarkers. Motor skills continued to improve, though some kinematic concerns persisted. These cases underscore the importance of early, high-dose ERT combined with ITI in managing CRIM-negative IOPD. While transitioning to avalglucosidase-alfa at 40 mg/kg/EOW was beneficial and well-tolerated in our patients, further studies are needed to confirm its long-term efficacy compared to the high-dose weekly 40 mg/kg alglucosidase-alfa.

2024-11-01·MOLECULAR GENETICS AND METABOLISM

Pompe disease: Unmet needs and emerging therapies

Review

作者: Tomassy, Giulio S. ; Berger, Kenneth I. ; Zhang, Tracy Y ; Anding, Allyson L. ; George, Kelly A ; Zhang, Tracy Y. ; Anding, Allyson L ; Tomassy, Giulio S ; Berger, Kenneth I ; George, Kelly A. ; van der Flier, Arjan ; Sardi, S Pablo ; Sardi, S. Pablo

Pompe disease is a debilitating and life-threatening disease caused by aberrant accumulation of glycogen resulting from reduced acid alpha-glucosidase activity. The first treatment for Pompe disease, the enzyme replacement therapy, Myozyme® (recombinant human acid alpha-glucosidase, alglucosidase alfa), is a lifesaving treatment for the most severe form of the disease and provided clinically meaningful benefits to patients with milder phenotypes. Nonetheless, many patients display suboptimal responses or clinical decline following years of alglucosidase alfa treatment. The approval of avalglucosidase alfa (Nexviazyme®) and cipaglucosidase alfa (Pombiliti®) with miglustat (Opfolda®) represents a new generation of enzyme replacement therapies seeking to further improve patient outcomes beyond alglucosidase alfa. However, the emergence of a complicated new phenotype with central nervous system involvement following long-term treatment, coupled with known and anticipated unmet needs of patients receiving enzyme replacement therapy, has prompted development of innovative new treatments. This review provides an overview of the challenges of existing treatments and a summary of emerging therapies currently in preclinical or clinical development for Pompe disease and related lysosomal storage disorders. Key treatments include tissue-targeted enzyme replacement therapy, which seeks to enhance enzyme concentration in target tissues such as the central nervous system; substrate reduction therapy, which reduces intracellular glycogen concentrations via novel mechanisms; and gene therapy, which may restore endogenous production of deficient acid alpha-glucosidase. Each of these proposed treatments shows promise as a future therapeutic option to improve quality of life in Pompe disease by more efficiently treating the underlying cause of disease progression: glycogen accumulation.

2024-11-01·BRAIN & DEVELOPMENT

Long-term observation of patients with advanced late-onset Pompe disease undergoing enzyme replacement therapy: A 15-year observation in a single center

Article

作者: Oya, Yasushi ; Takizawa, Hotake ; Yorimoto, Keisuke ; Mori-Yoshimura, Madoka ; Miyagi, Kenji ; Hara, Takatoshi ; Sato, Noriko ; Unuma, Atsushi ; Katsuta, Wakana ; Takahashi, Yuji

BACKGROUND:

There have been few descriptions in the literature on long-term enzyme replacement therapy (ERT) in patients with advanced late-onset Pompe disease (LOPD).

OBJECTIVES:

This study aimed to assess the efficacy and limitations of ERT in advanced LOPD patients.

METHODS:

We retrospectively reviewed the clinical courses of patients with advanced LOPD (two juvenile-onset and five adult-onset patients) who were treated with recombinant human alglucosidase alfa to examine improvements achieved with and limitations of ERT until their death or when switching to avalglucosidase alfa occurred.

RESULTS:

All patients were non-ambulant and ventilator dependent. The duration of follow-up ranged from 3.7 to 15.0 years (median 9.0 years). All patients reported improvements in their lives during the first two or three years of ERT. Vital capacity was clearly improved in patients with relatively spared respiratory function, although it deteriorated after respiratory complications such as pneumothorax. Pinch and grip power tended to be preserved during the treatment period. Muscle CT revealed progression of atrophy and fatty replacement predominantly in the proximal limb muscles without improvement after ERT. Four patients died due to aspergillosis, respiratory failure, ileus, and sudden death of unknown cause.

CONCLUSIONS:

Our findings demonstrate that patients undergoing ERT show certain improvements, even in the advanced stage of Pompe disease. Respiratory complications are lethal even during ERT, and early diagnosis and induction of therapy are critical. Muscle wasting progressed more severely in the proximal limbs, even after ERT.

项与艾夫糖苷酶α 相关的新闻（医药）

2024-10-28

·GBIHealth

赛诺菲Y24Q3财报速读：制药业务收入121.67亿欧元，中国市场增长3.6%

2024年10月25日，赛诺菲发布了2024年第三季度财务业绩。2024年第三季度（报告期），公司收入134.38亿欧元（约145.47亿美元），同比增长15.7%（按固定汇率计算，下同）。制药业务收入121.67亿欧元，中国市场增长3.6% 制药业务收入121.67亿欧元，同比增长16.6%；消费者保健业务Opella收入12.71亿欧元，同比增长7.9%。 2024年前三季度，公司收入达346.47亿欧元（约375.11亿美元），同比增长11.1%。制药业务增长强劲，主要由达必妥（度普利尤单抗）和疫苗驱动。报告期内（2024年第三季度），达必妥销售额34.76亿欧元，同比增长23.8%；达必妥前三季度销售额达96.14亿欧元，同比增长25.9%。公司预测，2024年全年，达必妥销售收入可达130亿欧元。报告期内，疫苗销售额38.02亿欧元，同比增长25.5%。其中，RSV疫苗Beyfortus（尼塞韦单抗）销售额增长381.8%达6.45亿欧元。报告期内，新上市药品收入7.27亿欧元，同比增长67.1%，主要由血友病疗法ALTUVIIIO（+278.3%）、庞贝病酶替代疗法Nexviazyme/Nexviadyme（+53.6%）等驱动。报告期内，其他药品销售收入41.62亿欧元，同比增长0.4%。按市场分美国市场收入68.86亿欧元，同比增长23.6%；欧洲市场收入28.86亿欧元，同比增长6.6%；其他市场收入36.66亿欧元，同比增长10.3%。中国市场收入增长3.6% 2024年第三季度，中国市场收入7.57亿欧元（约8.19亿美元），同比增长3.6%，主要由达必妥的强劲销售驱动，但又被依诺肝素（受VBP影响）和其他药品的销售低迷抵消。中国市场也驱动贝舒地尔、胰岛素来优时（甘精胰岛素）全球销售额分别增长57.8%和18.1%。波立维尽管在中国市场已进入带量采购，但由于应用范围持续扩大，推动全球销售增长8.3%。全球医疗情报领导者解锁隐藏在数据中的商业潜力关于 G B I ” 自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。联系我们投稿 | 发稿 | 媒体合作 ▶ zhangxinyue13@baidu.com 数据库 | 咨询服务 | 资讯追踪 ▶ 点击左下“阅读原文”完成表单填写点击阅读原文，解锁完整双语新闻

财报疫苗带量采购

2024-10-28

·echemi

Merck's RSV Antibody Faces Major Challenges vs. Sanofi's Beyfortus Exhibiting 83% Efficacy in Reducing Hospitalization

While Merck & Co. 's infant respiratory syncytial virus (RSV) antibody clesrovimab is nearing approval, sanofi also won approval last year for its RSV prevention drug beyforts in partnership with astrazeneca. This news undoubtedly brings new hope to the global healthcare industry, especially in the field of RSV vaccines. "We would very much welcome having more participants on board because it increases the scale of the competition," Thomas Triomphe, Sanofi's executive vice president for vaccines, said of the upcoming RSV competition during an analyst call on Friday. He stressed that more players mean more innovation and better healthcare solutions, which is good for the industry as a whole and for patients. Triomphe noted that Merck still has more data on clesrovimab after announcing phase 2b/3 clinical results last week. In the meantime, Sanofi remains "very confident" in the Beyfortus numbers it has released so far, he said. He mentioned that the protection data provided by Beyfortus in six months is very encouraging, as the effect has not diminished. "After six months, we were still seeing 83 percent hospitalization efficacy," he said, adding that he believed "the duration of protection from Beyfortus was significantly more favorable compared to clesrovimab." This data suggests that Beyfortus has a significant advantage in preventing RSV infection. In Merck's infant RSV study, clesrovimab successfully reduced RSV-related hospitalizations and RSV-related lower respiratory tract infection hospitalizations by 84 percent and 91 percent, respectively, over five months compared to placebo. This result is impressive and shows the great potential of clesrovimab in preventing RSV infection. At the trial's primary endpoint, clesrovimab reduced the incidence of RSV-related medical lower respiratory tract infections by 60 percent compared to placebo. This data provides further evidence of the effectiveness of clesrovimab in preventing RSV infection. Sanofi Chief Financial Officer franois-xavier Roger noted in a conference call on Friday that the inherent uncertainty of cross-trial comparisons suggested that existing Beyfortus data showed a reduction of about 75 percent in the incidence of lower respiratory tract infections with clesrovimab compared to 60 percent with Clesrovimab. This data further reinforces Beyfortus' advantage in preventing RSV infection. And, whether or not Merck brings clesrovimab into the 2025-2026 RSV season as planned, "2025 will be a year of growth for Beyfortus," the CFO stressed. He is confident that Beyfortus' sales will grow significantly as it gradually gains market share. Since it was approved last July, Beyfortus has gradually built up its sales profile. In the third quarter of 2024, sales of this preventive antibody reached 645 million euros ($699), an increase of 382% compared to the same period in 2023. Overall, sales of the drug have reached 845 million euros since the beginning of the year, which is close to the best-selling threshold. Sanofi said at the time that Beyfortus quickly fell into a supply crunch shortly after going public last year due to "higher than expected demand." To counter this, Sanofi and its partner Astrazeneca have moved quickly to ensure there is no repeat of the supply crunch in 2024. Recently they added a new filling line and shipped antibody doses ahead of the 2024 RSV season. "There are 20 markets and we don't see any capacity constraints," Triumph said on Sanofi's conference call. "We will increase supply next year and the year after to make sure there are no supply constraints." This initiative demonstrates Sanofi's confidence in the future of Beyfortus and its commitment to meeting the needs of the global market. The comments by Sanofi executives came as the French pharmaceutical company reported a 15.7 per cent rise in third-quarter sales. This growth was mainly driven by the excellent performance of its immunology product Dupixent, which brought in sales of nearly 3.5 billion euros (about $3.8 billion), an increase of about 24 percent over the third quarter of 2023. Sanofi confirmed DuPont's full-year sales target of about 13 billion euros. Elsewhere, newly launched drugs rose 67.1 percent to 727 million euros ($787), led by hemophilia drug Altuviiio, Pompeii disease treatment Nexviazyme and Rezurock for graft-versus-host disease. The strong performance of these new drugs further strengthens Sanofi's position in the field of innovative medicines. Meanwhile, Sanofi's consumer health division, Opella, saw revenue rise 7.9 percent. This growth shows that Sanofi is also making significant progress in consumer health. Earlier this week, Sanofi confirmed it had entered exclusive talks with U.S. private equity firm Clayton, Dubilier & Rice to sell a controlling 50 percent stake in its consumer healthcare business, which is valued at around 16 billion euros. This move will further strengthen Sanofi's financial strength and provide additional financial support for its future expansion.

上市批准临床结果疫苗

2024-09-20

·CPHI制药在线

25亿核药交易，赛诺菲强势加入“核药战”！

关注并星标CPHI制药在线近些年来，核药已然变成受行业瞩目的抗癌治疗方式。在过去的一年当中，众多大型药企借助收购专于开发核药的新兴公司，于这一范畴进行布局或者更进一步强化核药的开发实力，交易的总金额近乎100亿美元。就像诺华（Novartis）公司于今年5 月份投入超过 17亿美元，收购了致力于开发放射性配体疗法（RLT）的Mariana Oncology公司，以增强自身的 RLT研发产品线。阿斯利康（AstraZeneca）公司在今年3 月投入超过 20 亿美元收购了 Fusion Pharmaceuticals公司，纳入了基于同位素锕（actinium）、靶向前列腺特异性膜抗原（PSMA）的放射偶联药物，以及其他放射偶联药物产品线与Fusion 的研发与生产设施。百时美施贵宝（BristolMyers Squibb）在今年 2月完成了对 RayzeBio的收购，耗费近 41亿美元获取了临床期针对生长抑素受体（SSTR）的放射性靶向疗法和核药制造能力。 2024年9月12日，赛诺菲宣告与放射性制药公司 RadioMedix 和 OranoMed 达成了授权许可的协议。依据协议的条款要求，RadioMedix 和 Orano Med 会获取 1亿欧元的预付资金和最高可达2.2亿欧元的销售里程碑付款，（总计3.2亿欧元，约折合人民币 25.1亿）并且有权得到分级的特许权使用费，在合作达成以后，赛诺菲将会负责此项目的全球商业化运作，OranoMed 将会负责借助其全球工业平台生产 AlphaMedix。这次合作会着重于罕见癌症放射配体治疗领域RadioMedix所开发的领先项目 AlphaMedix（212Pb-DOTAMTATE）。AlphaMedix属于一种α核素靶向疗法（Targeted Alpha Therapy，TAT），由运用铅-212（Pb-212）放射性加以标记的生长抑素受体靶向肽复合物构成，当作α粒子的体内发生器。一、AlphaMedix：全球热点核药之一 AlphaMedix（²¹²Pb-DOTAMTATE）是RadioMedix 与Orano Med携手研发的一款放射治疗药物，同时也是当下全球热门的核素药物之一。AlphaMedix属于一种基于α粒子的靶向治疗手段（TAT），其是由经铅-212（212Pb）放射性标记的生长抑素受体靶向肽复合物所构成，能够于患者体内促使α粒子对肿瘤发起攻击。铅-212同位素由于其半衰期、高能量、衰变路径短小以及对双链DNA造成不可逆损伤等特性，尤适用于 SSTR治疗应用。此疗法在2018年获取了FDA 的孤儿药指定(ODD)，并于2020 年荣获了 TechConnect创新奖。近期，美国 FDA向 AlphaMedix授予了突破性疗法认定（BTD），用于医治未曾接受肽受体放射性疗法的胃肠胰神经内分泌肿瘤（GEP-NET）患者。神经内分泌肿瘤（NET）为一组源自神经内分泌细胞的异质性稀有癌症，多在胃肠道和胰腺出现，然而，也有可能在其他组织里产生，像胸腺、肺以及其他不常见区域（诸如卵巢、心脏和前列腺）。美国每年预计有12000 例NET 患者得以确诊，转移性 NET患者的平均5 年生存率达 60%。大多数NET 均高度表达 SSTR，故而 SSTR成为此类肿瘤的潜在治疗靶点。目前，AlphaMedix 已完成针对胃肠胰神经内分泌肿瘤（GEP-NET）成年患者的一项 II 期研究（ALPHAMEDIX02）。该研究涵盖了①未接受过多肽受体靶向放射治疗（PRRT）医治的不可切除或转移性且SSTR 呈阳性的 GEP-NET 患者（n=36），以及②接受 PRRT治疗后复发的不可切除或转移性且 SSTR呈阳性的GEP-NET患者（n=30）这两个队列。结果表明，队列①的患者接受治疗后，确认的客观缓解率（cORR）高达 47.2%（17/36）。基于此前的 I期研究成果和上述 II期研究结果，AlphaMedix 在今年 2月获得了 FDA赋予的突破性疗法资格。二、赛诺菲强势加入"核药战" 就在刚刚结束的Q2 财报会议中，赛诺菲研发负责人宣称暂时并无任何放射性疗法相关的项目，因而公司在核药方面打出的"回马枪"着实令人倍感意外。通过交易细节能够看出，赛诺菲为了涉足这样一个全新的领域，的确完成了充分的背景调研。放射性疗法对于赛诺菲而言是一个崭新的领域。在第二季度财报的电话会议上，赛诺菲研发负责人 Houman Ashrafian曾经表明，公司当下不存在任何放射性疗法的项目。"我们在这个领域颇为审慎，并思考实行真正独特化疗法的行动"。赛诺菲的一位发言人也予以证实，AlphaMedix属于赛诺菲管线当中的首个放射性配体疗法。但是，在赛诺菲尝试探索的这个新领域里面，当下在全球范畴内已经有一款针对 SSTR的治疗性放射性药物准许上市，那便是诺华的 Lutathera。AlphaMedix疗法一旦投入市场，会迅速和诺华所销售的放射性药物Lutathera产生竞争态势。 Lutathera属于基于镥-177的β粒子放射疗法，这是诺华于2017年耗费39 亿美元收购 Advanced AcceleratorApplications 而获得的。Lutathera 作为一种肽受体放射性核素治疗（PRRT）类药物，能够用以治疗生长抑素受体（SSTR）呈阳性的胃肠胰神经内分泌肿瘤（GEP-NETs）。不过，对比于β粒子放射型药物，在针对肿瘤的治疗领域，α核素放射性药物具备独有的优势。具体来讲，α核素在衰变期间能够释放出α粒子，其传能线密度（LET）相比于β核素大概高出百倍左右，在杀灭肿瘤方面的效果显著强于β核素。另一方面，β核素对肿瘤细胞 DNA单链予以破坏，肿瘤细胞存在修复的可能性，进而有造成肿瘤复发的风险。然而α核素能够直接破坏肿瘤细胞 DNA 双链，致使肿瘤细胞被永久性杀灭，被赞誉为"像手术刀一般的放疗"。此外，临床试验的数据还表明α核素在杀伤癌细胞的同时能够引发肿瘤免疫反应，和免疫疗法存在相互促进的作用，让肿瘤治疗达成"一加一大于二"的成效。在安全性的层面：α核素的射程较短，仅仅只有几个细胞的距离，对正常组织的损害极为微小且几乎不存在副作用，安全性更为出色。这也就意味着 AlphaMedix在肿瘤治疗当中具有潜藏的高效性与选择性，这或许会令其在某些情形下比β粒子疗法更具优势。但上述这些仅仅是从理论层面而言，具体的疗效还是需要看 AlphaMedix后续实际的临床数据。在市场前景的方面，当下在靶向 SSTR 的治疗性放射性药物赛道，诺华已经获得批准上市的 Lutathera在上市的头一年，销售额就达到了 1.67亿美元，在2023年，Lutathera 的销售额更是上升至6.05 亿美元。而 AlphaMedix的迅速跟进，在市场地位上给诺华增添了一层无形的压力。由此可以看出，赛诺菲在重组优化、布局罕见病、自免等领域，会越发注重具备差异化优势的重磅产品。三、AlphaMedix加速布局赛诺菲罕见病管线尽管赛诺菲在核药范畴尚属初次进入，然而此次合作管线的背后，所针对的实则是赛诺菲意欲"称雄"的罕见病市场。自从2011 年以201亿美元将孤儿药巨头 Genzyme收购之后，赛诺菲在罕见病药物市场中的地位持续攀升，牢牢占据着主导地位。依据医药魔方数据库的非完全统计，赛诺菲近五年于罕见疾病领域的受让交易项目达20项，涉及的交易总金额总计已经超过 125 亿美元。不过，当下赛诺菲的主导地位正遭受挑战，其中之一便是产品专利到期，从而面临着仿制药的冲击。举例来讲，用于治疗多发性硬化的 Aubagio曾是赛诺菲的王牌产品，其销量曾经仅仅位居 Dupixent、流感疫苗以及 Lantus 之后。可是自 2023年专利到期以后， Aubagio 在2023 年的销量同比降幅达52.6% 。为了巩固在罕见病市场的地位，赛诺菲屡次行动，引入全新的候选管线，期望能够寻得更具差异化优势的产品。连同本次的合作，这已然是赛诺菲本年度第三次在罕见病领域有所举措，现今，赛诺菲于罕见病领域总计布局了11 条在研管线，当中 6个候选产品处于3 期临床阶段。在2023 年，赛诺菲的营收约为470.97 亿美元，同比增加5.3%。就销售构成而言，罕见病领域产品变成了其主要的销售收入来源之一，其中 Nexviazyme/Nexviadzyme 、Enjaymo 、Xenpozyme这 3款罕见病产品表现出众，均保持着三位数以上的增长。但是，从销量的增长情况来看，即便 Enjaymo 和 Xenpozyme 表现不错，然而它们各自的销量仍然没有突破 1亿美金，很难在短期内填补Aubagio 因销量降低所造成的缺口。另外，在 470.97 亿美元的营收当中，达必妥的销量达到大约 115.8亿美元，约占赛诺菲2023 年收益的四分之一。过度依赖以及重仓单一产品或许会给赛诺菲带来发展风险。因此，赛诺菲正在展开重组优化工作，以筹措资金，加快布局罕见病和自免等领域，期望在其中找寻到下一个具备差异化优势的重磅产品。资料来源： 1.赛诺菲官网 2.医药魔方 END 【智药研习社直播预告】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~

并购放射疗法突破性疗法孤儿药

100 项与艾夫糖苷酶α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11744	艾夫糖苷酶α	A16AB07	DB16099

研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
II型糖原贮积病	美国	Genzyme Corp.	2021-08-06

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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


NCT02032524 (NEO1 and NEO-EXT \| NEO-EXT, CTgov)	临床2期	II型糖原贮积病	19	Avalglucosidase Alfa (Group 1: Avalglucosidase Alfa 5 mg/kg)	糧遞積醖憲膚築繭構範(艱獵壓積蓋蓋艱膚積襯) = 壓選鹹繭齋淵壓築繭範淵淵衊鬱構膚衊繭淵衊 (窪蓋壓艱衊蓋襯齋積憲, 廠餘齋壓鏇餘積選憲廠 ~ 繭廠廠鹹衊鹹壓範簾襯) 更多	-	2024-03-01
				Avalglucosidase Alfa (Group 1: Avalglucosidase Alfa 10 mg/kg)	糧遞積醖憲膚築繭構範(艱獵壓積蓋蓋艱膚積襯) = 壓餘鏇願廠鹹襯築衊窪淵淵衊鬱構膚衊繭淵衊 (窪蓋壓艱衊蓋襯齋積憲, 範齋壓窪鹹膚窪築齋簾 ~ 繭壓築鑰餘繭膚繭艱鏇) 更多
NCT03019406 (Mini-COMET, SSIEM2023)	临床2期	II型糖原贮积病 CK \| Hex4	22	Avalglucosidase alfa 20mg/kg/qow	襯製夢蓋衊齋鬱觸齋廠(鏇蓋顧窪鏇簾壓觸繭廠) = 獵鏇夢願淵繭觸鏇鑰醖鏇繭鏇壓艱築獵構積憲 (醖膚獵廠齋醖觸積衊鏇 )	-	2023-08-30
				Avalglucosidase alfa 40mg/kg/qow	襯製夢蓋衊齋鬱觸齋廠(鏇蓋顧窪鏇簾壓觸繭廠) = 淵遞壓廠簾窪繭齋網積鏇繭鏇壓艱築獵構積憲 (醖膚獵廠齋醖觸積衊鏇 )
NCT02782741 (COMET, pubmed) 人工标引	临床3期	II型糖原贮积病一线	100	Avalglucosidase alfa	鹹鑰憲鑰糧觸網簾餘醖(襯夢襯淵衊製壓膚艱衊) = 鏇夢顧餘膚襯網鬱簾膚觸遞醖獵壓艱鏇鏇糧簾 (鬱選夢憲憲憲壓築構範, 1.05) 更多	积极	2023-04-10
				Alglucosidase alfa (switched to avalglucosidase alfa after 49 weeks)	鹹鑰憲鑰糧觸網簾餘醖(襯夢襯淵衊製壓膚艱衊) = 窪積網糧簾壓觸範淵衊觸遞醖獵壓艱鏇鏇糧簾 (鬱選夢憲憲憲壓築構範, 1.12) 更多
NCT02782741 (COMET, MDA2023) 人工标引	临床3期	II型糖原贮积病	100	Avalglucosidase alfa 20 mg/kg (AVAL-arm)	淵願夢網蓋蓋餘憲膚醖(鬱夢鏇夢鬱淵膚製鬱艱) = 餘夢選鹹糧積選鹹窪鑰淵醖餘壓襯遞鏇鏇餘憲 (淵糧鬱淵窪觸選膚糧鹹, 9.60) 更多	积极	2023-03-19
				Alglucosidase alfa 20 mg/kg (Switch-arm)	淵願夢網蓋蓋餘憲膚醖(鬱夢鏇夢鬱淵膚製鬱艱) = 夢衊鑰襯憲醖艱繭壓壓淵醖餘壓襯遞鏇鏇餘憲 (淵糧鬱淵窪觸選膚糧鹹, 10.42) 更多
NCT02782741 (COMET, CTgov)	临床3期	II型糖原贮积病	101	PAP+Avalglucosidase Alfa (PAP: Avalglucosidase Alfa)	鹽鹽膚積鏇遞製築製鹽(壓製築鏇選餘憲鏇憲遞) = 襯選鑰網蓋窪鏇構選醖襯鑰構範糧蓋廠鑰齋遞 (觸繭衊襯觸築衊窪鬱衊, 窪衊鑰顧糧鬱積鬱選範 ~ 餘壓顧鏇醖築鏇獵壓窪) 更多	-	2021-04-08
				PAP+Alglucosidase Alfa (PAP: Alglucosidase Alfa)	鹽鹽膚積鏇遞製築製鹽(壓製築鏇選餘憲鏇憲遞) = 範鬱選鹹齋鬱簾選壓餘襯鑰構範糧蓋廠鑰齋遞 (觸繭衊襯觸築衊窪鬱衊, 壓觸築膚廠淵鑰網窪窪 ~ 鏇鬱獵網壓鹹製觸膚襯) 更多
NCT01898364 (Pubmed) 人工标引	临床1期	II型糖原贮积病	24	avalglucosidase alfa (Naïve patients)	艱願壓淵獵鹽選簾遞選(製獵夢齋鬱膚壓鹹觸製) = no deaths/life-threatening serious adverse events (SAEs). 築繭壓網艱遞醖鬱範網 (簾艱蓋構遞餘衊選積憲 ) 更多	积极	2019-03-01
				Alglucosidase Alfa (Switch patients)
NCT01898364 (AAN2016)	临床1期	II型糖原贮积病 acid α-glucosidase enzyme deficiency	-	neoGAA 5mg/kg	壓築簾顧糧獵顧淵糧糧(範衊積鑰醖簾願餘鬱鹽) = 鏇廠衊餘遞蓋簾艱願鏇鑰觸艱齋廠壓蓋獵蓋鏇 (醖網遞製鹹範鏇觸衊選 )	-	2016-04-05
				neoGAA 10mg/kg	壓築簾顧糧獵顧淵糧糧(範衊積鑰醖簾願餘鬱鹽) = 願選齋廠遞齋窪窪選鑰鑰觸艱齋廠壓蓋獵蓋鏇 (醖網遞製鹹範鏇觸衊選 )
NCT01898364 (I4.011, AAN2016)	临床1期	II型糖原贮积病	24	NeoGAA 5mg/kg	網齋艱襯鹹繭鏇獵膚窪(觸廠膚艱鬱淵膚觸網壓) = minimal changes 襯餘遞獵廠窪獵齋壓鹹 (夢憲艱簾獵製膚蓋範艱 ) 更多	-	2016-04-05
				NeoGAA 10mg/kg
NCT01898364 (S38.006, AAN2016)	临床1期	II型糖原贮积病	24	NeoGAA 5mg/kg	製築鏇憲廠獵壓鏇蓋構(遞繭醖網膚醖選製網願) = showed minimal changes across groups and all dose levels 鹹鏇鑰遞憲範鹽鏇窪構 (膚顧膚遞構窪夢蓋艱選 ) 更多	积极	2016-04-05
				NeoGAA 10mg/kg