Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.

开始日期2024-10-01

申办/合作机构

CHIESI Farmaceutici SpA

[+1]

NCT06663358 / Not yet recruitingN/A

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.

开始日期2024-10-01

申办/合作机构

CHIESI Farmaceutici SpA

NCT06095713 / RecruitingN/AIIT

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

Pegunigalsidase-alfa may represent an advance in ERT for FD, based on its unique pharmacokinetics and apparent low immunogenicity. The objective of the study is to document long term data on treatment with pegunigalsidase-alfa under "real world" conditions. 60 patients with FD (therapy-naïve or pretreated with agalsidase-alfa or agalsidase-beta) will be recruited in 8 German Fabry centers. The treatment duration/patient will be 2 years. All patients will be followed-up by the above listed Fabry expert centers.

开始日期2023-10-01

申办/合作机构

Westfälische Wilhelms-Universität Münster

[+1]

100 项与 Pegunigalsidase alfa 相关的临床结果

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100 项与 Pegunigalsidase alfa 相关的转化医学

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100 项与 Pegunigalsidase alfa 相关的专利（医药）

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项与 Pegunigalsidase alfa 相关的文献（医药）

2024-06-01·Journal of medical genetics

Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study

Article

作者: Rocco, Rossana ; Karaa, Amel ; Muriello, Michael ; Langeveld, Mirjam ; Warnock, David G ; Ortiz, Damara ; Giraldo, Pilar ; Jovanovic, Ana ; Bernat, John ; Geberhiwot, Tarekegn ; Linhart, Aleš ; Deegan, Patrick B ; Holida, Myrl ; Kantola, Ilkka ; Nedd, Khan ; Nowak, Albina ; Waldek, Stephen ; Barshop, Bruce A ; Germain, Dominique Paul ; Sakov, Anat ; Alon, Sari ; Hopkin, Robert J ; Brill-Almon, Einat ; Pisani, Antonio ; Hughes, Derralynn A ; Goker-Alpan, Ozlem ; Longo, Nicola ; Wallace, Eric L ; Kimonis, Virginia ; Wilcox, William R ; Knoll, Jasmine ; Chertkoff, Raul ; Mehta, Ankit ; Vujkovac, Bojan ; Tøndel, Camilla ; Molnar, Maria Judit

Background:

Pegunigalsidase alfa is a PEGylated α-galactosidase A enzyme replacement therapy. BALANCE (NCT02795676) assessed non-inferiority of pegunigalsidase alfa versus agalsidase beta in adults with Fabry disease with an annualised estimated glomerular filtration rate (eGFR) slope more negative than −2 mL/min/1.73 m2/year who had received agalsidase beta for ≥1 year.

Methods:

Patients were randomly assigned 2:1 to receive 1 mg/kg pegunigalsidase alfa or agalsidase beta every 2 weeks for 2 years. The primary efficacy analysis assessed non-inferiority based on median annualised eGFR slope differences between treatment arms.

Results:

Seventy-seven patients received either pegunigalsidase alfa (n=52) or agalsidase beta (n=25). At baseline, mean (range) age was 44 (18–60) years, 47 (61%) patients were male, median eGFR was 74.5 mL/min/1.73 m2and median (range) eGFR slope was −7.3 (−30.5, 6.3) mL/min/1.73 m2/year. At 2 years, the difference between median eGFR slopes was −0.36 mL/min/1.73 m2/year, meeting the prespecified non-inferiority margin. Minimal changes were observed in lyso-Gb3 concentrations in both treatment arms at 2 years. Proportions of patients experiencing treatment-related adverse events and mild or moderate infusion-related reactions were similar in both groups, yet exposure-adjusted rates were 3.6-fold and 7.8-fold higher, respectively, with agalsidase beta than pegunigalsidase alfa. At the end of the study, neutralising antibodies were detected in 7 out of 47 (15%) pegunigalsidase alfa-treated patients and 6 out of 23 (26%) agalsidase beta-treated patients. There were no deaths.

Conclusions:

Based on rate of eGFR decline over 2 years, pegunigalsidase alfa was non-inferior to agalsidase beta. Pegunigalsidase alfa had lower rates of treatment-emergent adverse events and mild or moderate infusion-related reactions.

Trial registration number:

NCT02795676.

2024-06-01·Journal of medical genetics

Comment to: Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study—determination of immunogenicity

Letter

作者: Lenders, Malte ; Brand, Eva

A review.The recent studies by Linhart and colleagues as well as Wallace and colleagues if of great interest, .These studies were decisive for approval of pegunigalsidase alfa as a new enzyme replacement therapy in patients with Fabry disease (FD) by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) in May 2023.In the BRIDGE study, the authors report about the safety and efficacy in patients with FD under pegunigalsidase alfa, previously treated with agalsidase alfa.The annualized slope of the estimated glomerular filtration rate (eGFR) changed from -5.90 mL/ min/1.73 m²/yr under agalsidase alfa to -1.19 mL/ min/1.73 m²/yr under pegunigalsidase alfa.As a biochem. response, treatment with pegunigalsidase alfa led to a mean plasma lyso-Gb3 reduction by 31.5%.

2023-12-01·Genetics in medicine : official journal of the American College of Medical Genetics

Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease

Article

作者: Holida, Myrl ; Alon, Sari ; Hughes, Derralynn ; Maegawa, Gustavo ; Goker-Alpan, Ozlem ; Rocco, Rossana ; Atta, Mohamed G ; Almon, Einat Brill ; Giraldo, Pilar ; Bernat, John A ; Chertkoff, Raul ; Gonzalez, Derlis

PURPOSE:

Fabry disease (FD) is a rare lysosomal storage disorder caused by pathogenic variants in the GLA gene encoding α-galactosidase (α-Gal)-A. We evaluated long-term safety/efficacy of pegunigalsidase alfa, a novel PEGylated α-Gal-A enzyme replacement therapy (ERT) now approved for FD.

METHODS:

In a phase-1/2 dose-ranging study, 15 ERT-naive adults with FD completed 12 months of pegunigalsidase alfa and enrolled in this 60-month open-label extension of 1 mg/kg pegunigalsidase alfa infusions every 2 weeks.

RESULTS:

Fifteen patients enrolled (8 males; 7 females); 10 completed ≥48 months (60 months total treatment), and 2 completed 60 months (72 months total treatment). During treatment, most treatment-emergent adverse events were mild/moderate in severity and all infusion-related reactions were mild/moderate in severity. Four patients were transiently positive for anti-pegunigalsidase alfa IgG. Patients showed continuous reduction in plasma lyso-Gb3 concentrations with mean (standard error) reduction of 76.1 [25.1] ng/mL from baseline to month 24. At 60 months, the estimated glomerular filtration rate slope was comparable to that observed in patients treated with other ERTs. Cardiac function assessments revealed stability; no cardiac fibrosis was observed.

CONCLUSION:

In this first long-term assessment of pegunigalsidase alfa administration in patients with FD, we found favorable safety/efficacy. Our data suggest long-term continuous benefits of pegunigalsidase alfa treatment in adults with FD.

项与 Pegunigalsidase alfa 相关的新闻（医药）

2024-11-07

·PRNewswire

Protalix BioTherapeutics to Announce Third Quarter 2024 Financial and Business Results on November 14, 2024

Company to host conference call and webcast at 8:30 a.m. EST CARMIEL, Israel, Nov. 7, 2024 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced that it will release its financial results for the quarter ended September 30, 2024 and provide a business and clinical update on November 14, 2024. Management will host a conference call to discuss the financial results and provide an update on recent corporate and regulatory developments. Conference Call Details: Date: Thursday, November 14, 2024 Time: 8:30 a.m. Eastern Standard Time (EST) Toll Free: 1-877-423-9813 International: 1-201-689-8573 Israeli Toll Free: 1-809-406-247 Conference ID: 13749493 Call me™: The Call me™ feature allows you to avoid the wait for an operator; you enter your phone number on the platform and the system calls you right away. Webcast Details: Company Link: Webcast Link: Conference ID: 13749493 Participants are requested to access the call at least 15 minutes ahead of the conference to register, download and install any necessary audio software. A replay of the call will be available for two weeks on the Events Calendar of the Investors section of the Company's website, at the above link. About Protalix BioTherapeutics, Inc. Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. Protalix's second product, Elfabrio®, was approved by both the FDA and the European Medicines Agency in May 2023. Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of Elfabrio. Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of uncontrolled gout; PRX–119, a plant cell-expressed long action DNase I for the treatment of NETs-related diseases; and others. Logo - Investor Contact Mike Moyer, Managing Director LifeSci Advisors +1-617-308-4306 [email protected] SOURCE Protalix BioTherapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上市批准财报

2024-10-25

·PRNewswire

Protalix BioTherapeutics to Present Phase I PRX-115 Data in Late-Breaking Poster at ACR Convergence 2024

CARMIEL, Israel, Oct. 25, 2024 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced that data from the phase I clinical trial of PRX-115, the Company's recombinant PEGylated uricase product candidate in development for the treatment of uncontrolled gout, will be presented in a late-breaking poster at the American College of Rheumatology (ACR) Convergence 2024, being held November 14-19, 2024 at the Walter E. Washington Convention Center in Washington, D.C. Details of the presentation are as follows: ACR Convergence 2024 Title: Prolonged Plasma Urate-Lowering After a Single Intravenous Administration of PRX-115, a Novel PEGylated Uricase, in Participants with Elevated Urate Levels Session: Late-Breaking Posters (L01-L14) Session Date/Time: Monday, November 18, 2024, 10:30 AM – 12:30 PM Eastern Standard Time Presenting Author: Orit Cohen Barak, Ph.D. (Protalix Ltd.) Abstract Number: L05 The accepted abstract can be accessed on the ACR Convergence 2024 website at . A copy of the poster will be made available on the Protalix website. About Protalix BioTherapeutics, Inc. Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. Protalix's second product, Elfabrio®, was approved by both the FDA and the European Medicines Agency in May 2023. Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of Elfabrio. Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of uncontrolled gout; PRX–119, a plant cell-expressed long action DNase I for the treatment of NETs–related diseases; and others. Forward-Looking Statements To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "expect," "anticipate," "believe," "estimate," "project," "may," "plan," "will," "would," "should" and "intend," and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: failure or delay in the commencement or completion of clinical trials which may be caused by several factors, including: slower than expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to satisfactorily demonstrate non-inferiority to approved therapies; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; inability to monitor patients adequately during or after treatment; and/or lack of sufficient funding to finance our clinical trials; the risk that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; our dependence on performance by third-party providers of services and supplies, including without limitation, clinical trial services; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies; potential product liability risks, and risks of securing adequate levels of related insurance coverage; the possible disruption of our operations due to terrorist activities and armed conflict, including as a result of the disruption of the operations of certain regulatory authorities and of certain of our suppliers, collaborative partners, licensees, clinical trial sites, distributors and customers; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law. Investor Contact Mike Moyer, Managing Director LifeSci Advisors +1-617-308-4306 [email protected] Logo - SOURCE Protalix BioTherapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床1期上市批准临床结果

2024-10-24

·行舟Drug

推荐文章 | 2023 年全球获批上市的原创新药: 回顾与展望

点击上方的行舟Drug ▲ 添加关注摘要 2023 年对于生物医药行业而言是具有里程碑意义的一年，在这一年中美国、欧洲、日本与中国均积极推动新药创制和新兴技术发展，在针对新型疾病的治疗方案、基因编辑技术等领域获得重大突破，利用人工智能/大数据等先进技术提升了研发效率和质量，全球生物医药行业在创新、市场需求、产业链整合和国际化等方面呈现出积极的发展趋势。本文梳理与总结 2023 年美国、欧洲、日本和中国获批上市的新药，并对 2024 年新药研发和生物医药发展态势进行展望，以期为新药研发相关研究提供参考。【关键词】创新药; 首创类药物; 细胞与基因治疗; 孤儿药; 基因编辑随着医药科技的飞速进步和临床需求的日益增长，创新药研发已成为引领全球医疗健康领域前行的核心动力。2023 年全球新型冠状病毒感染逐渐受控，经济复苏为创新药研发提供了更多资金与市场机遇，全球生物医药市场规模持续扩大，创新药研发的投入和产出均实现了显著增长，然而地缘政治紧张等因素为全球创新药研发带来了一定的挑战与不确定性。美国在创新药研发的全球竞技场中仍占据主力，欧洲与日本也保持强劲竞争力，中国、印度等新兴市场国家基于庞大的患者群体、独特的疾病谱、不断优化的政策环境和日益增强的研发实力，逐渐成为全球新药研发的重要力量。本文对 2023 年美国、欧洲、日本和中国获批上市的新药进行梳理和重点介绍，并对 2024 年新药研发和生物医药发展态势进行展望，旨在为新药研发相关研究提供参考。2023 年之前已获批上市药物的相关仿制药、新剂型、新适应证以及血液制品等不列入本文讨论范围内。一 2023 年美国 FDA 批准的全新药物 2023 年美国 FDA 药品评估和研究中心( Centerof Drug Evaluation and Ｒesearch，CDEＲ) 共批准了 55款新药( 见表1) ，其在药物设计、作用机制和治疗效果方面展现出高度创新性。20 款( 36% ) 为首创类药物( first-in-class) ，其适应证范围广泛，涵盖肿瘤、感染、神经系统疾病、罕见病等多个领域，其中，罕见病领域的成绩尤为突出，有 28 款药物( 51% ) 以孤儿药资格获批。按治疗领域划分，肿瘤学领域获批药物最多，CDEＲ在 2023 年批准了 13 款抗肿瘤药物( 24% ) ; 神经学领域排名第 2，9 款药物( 16% ) 获批; 传染病学和血液学领域并列第 3，各有 5 款药物( 9% ) 获批［1］。CDEＲ通过综合应用各种加速开发与审评审批的方法，使得具有显著疗效或针对严重未被满足临床需求的药物更早惠及患者。55 款获批新药中，25 款( 45% ) 被授予快速通道( fast track) 资格，9 款( 16% ) 获突破性疗法( breakthrough therapy)认定，31 款( 56% ) 获优先审评( priority review) 资格，9 款( 16% ) 获加速审批( accelerated approval) ;共有 36 款( 65% ) 使用了快速通道、突破性疗法、优先审评和加速审批中的至少 1 种加速程序［2］。 ▲表1-2023 年美国 FDA 药品评估和研究中心批准的全新药物 EGFＲ2: 表皮生长因子受体 2; Nrf2: 核转录因子红系 2 相关因子 2; PD-1: 程序性死亡受体 1; PI3K: 磷脂酰肌醇 3 激酶; SGLT2: 钠-葡萄糖共转运体 2; JAK: Janus激酶; FcＲn: 新生儿 Fc 受体; ＲSV: 呼吸道合胞病毒; FLT3: Fms 样酪氨酸激酶3; GABA-A: γ-氨基丁酸 A; GPＲC5D: G 蛋白偶联受体家族 C 组5 成员 D; CXCＲ4: 趋化因子受体 4; ALK-2 /ACVＲ1: 激活素受体ⅠA; 5-HT1A: 5-羟色胺 1A; AChＲ: 乙酰胆碱受体; IL: 白细胞介素; VEGFＲ: 血管内皮生长因子受体; NSCLC: 非小细胞肺癌;COVID-19: 新型冠状病毒感染 2023 年美国 FDA 生物制品评价与研究中心( Center for Biologics Evaluation and Ｒesearch，CBEＲ)批准了 25 款产品，涉及细胞和基因疗法、疫苗和血液制品[3] 。值得一提的是由Vertex 公司与 CＲISPＲ公司合作开发的 exagamglogene autotemcel( 商品名:Casgevy) 于 2023 年 12 月 8 日获批上市，该产品是一种基于 CＲISPＲ /Cas9 基因编辑技术的疗法，用于治疗 12 岁及以上反复出现血管阻塞性危机( vasoocclusive crisis，VOC) 的镰状细胞病( sickle cell disease，SCD) 患者，其作为全球首个获批的 CＲISPＲ /Cas9 基因编辑疗法，在生物医药行业发展过程中极具里程碑意义。二 2023 年欧盟批准的全新药物 2024 年初欧洲 EMA 发布了 Human MedicinesHighlights 2023 手册，该手册概述了欧洲 EMA 关于人用药品的重大决定和推荐，77 款产品获得积极意见。其中新活性物质 39 款、优先药物计划( prioritymedicines，PＲIME) 认定药物 3 款、孤儿药 17 款、生物类似药 8 款、先进技术治疗医学产品( advanced therapy medicinal product，ATMP) 1 款; 产品数量最多的适应证依次为肿瘤( 25 款) 、神经疾病( 11 款) 与心脏疾病( 6 款) ［4］。pegunigalsidase alfa( 商品名: Elfabrio) ，lebrikizumab ( 商品名: Ebglyss) 与 palopeg teriparatide( 商品名: Yorvipath) 先于其他国家首次在欧洲获批( 其中 pegunigalsidase alfa 在 2023 年 5 月相继获得了欧洲 EMA 与美国 FDA 的批准，欧洲EMA 的批准时间略早于美国 FDA，见表 2) 。前述基因编辑疗法产品 Casgevy 也被欧洲 EMA 予以重视和积极推荐，但其最先在英国获批( 2023 年 11 月16 日) ，直至 2024 年 2 月 13 日才获欧盟批准。 ▲表 2-2023 年欧洲 EMA 批准的全新药物( 全球首批) 三 2023 年日本批准的全新药物 2023 年日本医药品和医疗器械局( Pharmaceuticals and Medical Devices Agency，PMDA) 共批准 106款新药，其中新活性成分新药数量达 30 款，以疫苗和抗肿瘤药居多，约占新活性成分新药数量的 1 /3。mirikizumab( 商品名: Omvoh) ，zilucoplan( 商品名: Zilbrysq) 与 AＲCT-154( 商品名: コスタイベ筋注用) 先于其他国家首次在日本获批( 见表3) 。AＲCT-154 由CSL 公司与 Arcturus therapeutics 公司共同开发，作为全球首个自扩增 mＲNA ( self-amplifying mＲNA，sa-mＲNA) 抗 COVID-19 疫苗，成为2023 年度获批药物中备受瞩目的产品。sa-mＲNA 技术可诱导较强的细胞免疫反应并延长保护期，同时使 mＲNA 剂量明显减少，有可能成为持久的疫苗选择［5］。 ▲表3-2023 年 PMDA 批准的全新药物( 全球首批) 四 2023 年中国批准的 1 类创新药在药品审评审批制度改革不断深化的背景下，我国国家药品监督管理局( National Medical Products Administration，NMPA) 致力于加速临床急需新药、儿童用药以及罕见疾病用药的上市进程。2023 年NMPA 以临床价值为导向，采取多项举措使得获批上市的新药数量稳步增长、药品审评审批速度明显加快，从而为患者带来更多获益。2024 年 2 月国家药品监督管理局药品审评中心( Center for Drug Evaluation，CDE) 发布了《2023 年度药品审评报告》，报告指出 2023 年共有 40 款 1 类新药获批，其中化学药 19 款、预防用生物制品 2 款、治疗用生物制品14 款、中药 5 款，36 款新药先于其他国家首次在中国获批。获批产品涉及最多的 2 种适应证分别为肿瘤和感染性疾病，数量分别为 16 款( 40% ) 和 8款( 20% ) 。本文仅列出使用了至少 1 种加快程序的 1 类新药( 共 15 款，见表 4) ，其中 9 款( 22. 5% )通过优先审评审批程序获批，13 款( 32. 5% ) 为附条件批准上市，8 款( 20% ) 在临床研究阶段纳入了突破性治疗药物程序，4 款( 10% ) COVID-19 治疗药物通过特别审批程序获批。此外，2023 年 NMPA 批准罕见病用药 45 款、儿童用药 92 款、CAＲ-T 细胞治疗产品 3 款、境外已上市、境内未上市的原研药品 86款［6］，以下不再详述。 ▲表4-2023 年我国 NMPA 通过加快上市程序批准的 1 类新药五 2024 年生物医药领域展望 2023 年底至 2024 年初，已有多家机构如 Evaluate 公司、科睿唯安公司等对 2024 年可能最值得关注的药物进行了展望。Evaluate 公司报道了 2024年可能最有价值的 10 个管线，其中诺和诺德公司与礼来公司治疗糖尿病和肥胖症的新药、罗氏公司的TIGHT 靶向抗体与强生公司治疗自身免疫性疾病新药尤其引人关注。10 个重磅在研管线中小分子药物高达 7 款，单抗 2 款，多肽药物 1 款，可见小分子药物在生物医药领域仍占据重要地位［7］。科睿唯安公司发布《最值得关注的药物预测》报告，介绍了13 种可能在 2024 年进入市场的新疗法和新药物，其适应证覆盖乳腺癌、A 型血友病、镰状细胞病、克罗恩病、溃疡性结肠炎、ＲSV 感染和多发性骨髓瘤等临床需求未被满足的疾病领域，并预测这些药物在 2029 年前将成为“重磅炸弹药物”，其可能改变临床治疗模式，从而使患者受益［8］。此外，2024 年生物医药领域将展现出更为多元化、个性化和智能化的发展趋势。基于 2023 年首个CＲISPＲ /Cas9 基因编辑产品成功获批，基因编辑技术可能取得更为显著的突破，为罕见遗传病的治疗带来希望，并引领个性化医疗新时代来临; 细胞疗法在再生医学、肿瘤治疗等领域的应用将会更加广泛，而免疫疗法也会继续保持热度，在更多疾病领域发挥重要作用; 人工智能( artificial intelligence，AI) 将进一步释放其巨大潜力，帮助药物研发机构降本效，从而更快地为患者提供创新药物，这些热门领域和技术突破将共同推动创新药研发迈向新的高峰。六结语与展望从全球视角来看，2023 年无疑是创新药的丰收之年。与 2022 年相比，2023 年新药不仅在数量上实现了增长，更在质量和疗效上取得了显著突破。多款针对肿瘤、罕见病、神经系统疾病等领域的新药获批上市，为全球患者带来了全新的治疗选择。同时 CＲISPＲ /Cas9 基因编辑技术、mＲNA 疫苗技术、AI 等新兴技术的快速发展充分赋能新药与生物制品研发，推动其向更高效、更精准和更个性化的方向发展。 2023 年我们见证了中国新药稳健发展与走向世界舞台，3 款国产创新药———特瑞普利单抗( tori palimab，由上海君实生物医药科技股份有限公司研发，其合作伙伴 Coherus BioSciences 公司负责该产品在美国和加拿大的开发和商业化) 、呋喹替尼( fruquintinib，由和黄医药公司研发) 以及艾贝格司亭 α 注射液( efbemalenograstim alfa，由亿帆医药股份有限公司研发) 在美国获批上市，反映出本土制药企业创新质量的提升，其产品价值已开始获得国际认可［9］。需要强调的是，全球生物医药领域仍面临诸多挑战，如何在药物创新性与安全性之间寻求平衡点、如何确保新药可及性和可负担性以及如何加强国际合作与交流等问题，仍需我们共同努力与探索。总体而言，2023 年全球上市新药呈现出积极向好的态势，我们期待更多具有全新机制与卓越疗效的药物在未来问世，造福广大患者。参考文献［1］ MULLAＲD A． 2023 FDA approvals［J］． Nat Ｒev Drug Discov，2024，23( 2) : 88 － 95．［2］ FDA． Advancing health through innovation: new drug therapy approvals 2023［EB /OL］．［2024 － 03 － 10］． https: / /www． fda．gov /media /175253 /download? attachment．［3］ FDA． 2023 Biological License Application Approvals［EB/OL］．( 2024 － 01 － 24) ［2024 － 04 － 11］． https: / /www． fda． gov /vaccines-blood-biologics/development-approval-process-cber/2023-biological-license-application-approvals．［4］ European Medicines Agency． Human medicines: highlights of 2023［EB /OL］． ( 2024 － 01 － 16) ［2024 － 03 － 18］． https: / /www． ema． europa． eu /en /news/human-medicines-highlights-2023．［5］ Arcturus Therapeutics． Japan’s Ministry of Health，Labour and Welfare Approves CSL and Arcturus Therapeutics’AＲCT-154，thefirst self-amplifying mＲNA vaccine approved for COVID in adults［EB/OL］． ( 2023 － 11 － 28) ［2024 － 04 － 10］． https: / /ir． arcturusrx． com/news-releases/news-release-details/japans-ministry-health-labour-and-welfare-approves-csl-and．［6］国家药品监督管理局． 2023 年度药品审评报告［EB/OL］．( 2024 － 02 － 04) ［2024 － 04 － 10］． https: / /www． nmpa． gov． cn /xxgk /fgwj /gzwj /gzwjyp /20240204154334141． html．［7］ Evaluate． 2024 Preview Ｒeport eBook［EB /OL］． ( 2023 － 12 －21) ［2024 － 04 － 11］． https: / /www． evaluate． com/thought-leadership /2024-preview-report-ebook / ? utm_ source = website＆utm_medium = banner＆utm_campaign = 2024_preview＆utm_content =ebook．［8］ Clarivate．科睿唯安发布 2024 年度《最值得关注的药物预测》报告，聚焦 13 种潜在重磅炸弹药物［EB/OL］． ( 2024 － 01 －09) ［2024 － 04 － 10］． https: / /clarivate． com． cn /2024/01/09 /clarivate-identifies-thirteen-potential-blockbuster-drugs-and-gamechangersin-annual-drugs-to-watch-report/．［9］戈乾玮，孔令辉，邵黎明．展望 2024: 提振信心，推动中国生物医药产业高质量平衡发展［J］．药学进展，2024，48 ( 3) :237 － 242．文章信息源于公众号注册圈，登载该文章目的为更广泛的传递行业信息，不代表赞同其观点或对其真实性负责。文章版权归原作者及原出处所有，文章内容仅供参考。本网拥有对此声明的最终解释权，若无意侵犯版权，请联系小编删除。学如逆水行舟，不进则退；心似平原走马，易放难收。行舟Drug 每日更新欢迎订阅+ 医药大数据|行业动态|政策解读

孤儿药上市批准快速通道基因疗法

100 项与 Pegunigalsidase alfa 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11685	-	-	DB14992

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
法布里病	欧盟	CHIESI Farmaceutici SpA	2023-05-04
法布里病	冰岛	CHIESI Farmaceutici SpA	2023-05-04
法布里病	列支敦士登	CHIESI Farmaceutici SpA	2023-05-04
法布里病	挪威	CHIESI Farmaceutici SpA	2023-05-04

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临床结果

适应症

分期

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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


NCT02795676 (BALANCE, Pubmed \| J Med Genet)	临床3期	法布里病 lyso-Gb3 concentrations	-	Pegunigalsidase alfa 1 mg/kg	醖艱壓顧壓糧鑰製膚窪(襯鏇製衊鹹鹽鑰糧廠製) = 簾鹽鬱積糧襯壓齋蓋齋淵範憲鏇淵顧遞鹹選鹹 (鬱糧製艱願築鬱製範膚 )	积极	2024-06-01
				Agalsidase beta 1 mg/kg	醖艱壓顧壓糧鑰製膚窪(襯鏇製衊鹹鹽鑰糧廠製) = 繭願衊窪築憲遞憲蓋醖淵範憲鏇淵顧遞鹹選鹹 (鬱糧製艱願築鬱製範膚 )
NCT02795676 (BALANCE, PRNewswire) 人工标引	临床3期	法布里病	77	pegunigalsidase alfa-iwxj	糧憲鹽鹹觸鑰觸鹹鑰蓋(鑰壓蓋簾選襯鏇網衊構) = 艱鏇觸構憲繭範鑰蓋遞鏇積糧蓋範鑰齋構願窪 (襯鬱範餘艱膚壓顧鹹築 ) 更多	积极	2024-01-10
				agalsidase beta	糧憲鹽鹹觸鑰觸鹹鑰蓋(鑰壓蓋簾選襯鏇網衊構) = 壓網願範選築選鬱醖構鏇積糧蓋範鑰齋構願窪 (襯鬱範餘艱膚壓顧鹹築 ) 更多
NCT03018730 (BRIDGE, Pubmed \| Orphanet J Rare Dis)	临床3期	法布里病 plasma lyso-Gb3 level	-	Pegunigalsidase alfa 1 mg/kg	膚積鏇淵鬱艱窪艱淵獵(襯廠觸餘齋鹹齋膚窪獵) = 35% out of 20 patients were positive for pegunigalsidase alfa antidrug antibodies (ADAs) at ≥ 1 study timepoint, two of whom had pre-existing ADAs at baseline 窪憲繭獵鹹製夢鬱獵襯 (糧鏇糧顧齋淵選鹹繭襯 ) 更多	积极	2023-10-21
SSIEM2023	N/A	-	113	Pegunigalsidase alfa 1 mg/kg every other week	築繭簾艱遞鹽餘簾襯願(衊艱糧衊製鑰構鬱製獵) = 衊衊鹹餘窪顧願繭艱蓋選選觸繭壓餘襯憲顧齋 (網築積鏇網鏇觸窪壓繭 ) 更多	-	2023-08-30
				Pegunigalsidase alfa 2 mg/kg every 4 weeks	築繭簾艱遞鹽餘簾襯願(衊艱糧衊製鑰構鬱製獵) = 襯齋範鏇糧簾淵憲積蓋選選觸繭壓餘襯憲顧齋 (網築積鏇網鏇觸窪壓繭 ) 更多
SSIEM2023	N/A	-	142	Pegunigalsidase alfa	製蓋襯齋憲醖積築艱衊(觸簾壓憲艱鹽餘鏇憲鑰) = 25% of pts had an IRR 鑰簾廠製憲願廠衊鏇願 (積廠築繭顧鹽網製糧淵 ) 更多	-	2023-08-30
NCT02795676 (BALANCE study \| BALANCE, CTgov)	临床3期	法布里病	78	PRX-102 (pegunigalsidase alfa) (PRX-102 (Pegunigalsidase Alfa))	構鏇膚憲廠遞夢廠願壓(構齋積窪廠顧鏇糧築製) = 遞簾餘憲憲膚艱夢廠鏇壓壓衊鏇鹽築網齋積鑰 (構襯餘艱窪遞鏇構顧顧, 願廠艱鹽壓遞構憲範餘 ~ 鬱壓獵壓餘廠觸糧範製) 更多	-	2023-08-02
				agalsidase beta (Agalsidase Beta)	構鏇膚憲廠遞夢廠願壓(構齋積窪廠顧鏇糧築製) = 選壓繭壓鹽製憲蓋顧蓋壓壓衊鏇鹽築網齋積鑰 (構襯餘艱窪遞鏇構顧顧, 鑰製襯憲蓋壓憲積繭鏇 ~ 淵窪窪淵夢膚獵窪壓壓) 更多
NCT01678898 (FDA) 人工标引	临床1/2期	法布里病	14	ELFABRIO	淵鑰廠艱衊構構蓋願衊(齋鑰衊窪衊選網襯鏇築) = 廠網繭淵積襯壓齋鹹獵顧範夢獵蓋觸獵獵壓鏇 (築糧衊壓窪選糧願鹹願 ) 更多	积极	2023-05-09
				ELFABRIO (Males)	淵鑰廠艱衊構構蓋願衊(齋鑰衊窪衊選網襯鏇築) = 壓鑰願鹽選獵憲鹽網顧顧範夢獵蓋觸獵獵壓鏇 (築糧衊壓窪選糧願鹹願 ) 更多
NCT03566017 (FDA) 人工标引	临床3期	法布里病	77	ELFABRIO	繭選夢築壓遞繭廠簾蓋(範憲鹹夢淵齋選衊窪壓) = 繭製觸襯壓膚鹽網鬱糧願簾製願糧窪齋網艱願 (壓膚鬱鏇襯獵鑰獵憲獵 ) 更多	积极	2023-05-09
				agalsidase beta	繭選夢築壓遞繭廠簾蓋(範憲鹹夢淵齋選衊窪壓) = 襯積鑰襯襯齋範選繭網願簾製願糧窪齋網艱願 (壓膚鬱鏇襯獵鑰獵憲獵 )
EMA 人工标引	临床1/2期	法布里病	13	Elfabrio	鏇選鹹鏇網觸構繭遞鹽(壓簾鹽醖鏇鹹獵襯選淵) = 鹽鹽壓蓋糧範艱鏇觸顧鹹築糧壓鏇網鏇獵艱築 (築遞齋積鏇顧窪鏇襯選 ) 更多	积极	2023-05-08
ASN2022	N/A	法布里病	-	Pegunigalsidase alfa (PA)	膚壓鬱簾觸鬱網積餘積(窪顧顧鏇鬱觸淵鬱憲鑰) = 鹹繭廠淵鹽壓鏇艱艱築窪窪衊膚餘觸鹹繭襯築 (膚鹽簾夢顧選淵網鹹觸, 14.1 ~ 131.9)	-	2022-11-04