首页 > 最新文献

Acta pharmaceutica Hungarica最新文献

英文 中文
Same Product Different Regulatory Approach Around the World: Glatiramer Acetate 同一产品在世界各地不同的监管方法:醋酸格拉替雷
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.112-113
P. Rocco, P. Minghetti
Complex drugs may be either biological, if the active ingredients are derived from a biological source, or non-biological, if obtained by chemical synthesis. In both cases, their quality depends considerably on the manufacturing process. For Non Biological Complex Drugs (NBCDs), in particular, complexity may arise either from the active substance, as in the case of glatiramer acetate (GA), or from other sources, such as the formulation, as in the case of liposomes (Figure 1) (1). GA is approved, in the US and the EU, as a diseasemodifying treatment for patients with relapsing forms of Multiple Sclerosis. It is a heterogeneous mixture of not fully characterized synthetic polypeptides, containing L-alanine, L-lysine, L-glutamic acid, L-tyrosine in the constant molar ratio 0.43:0.34:0.14:0.09, with and average molecular weight from 5 to 9 kDa and distribution range from 2.5 to 20 kDa (2). The amino acid sequences are not completely random, being the result of both the physicochemical properties of the starting materials and the fundamental reaction scheme. However, they are not completely conserved from batch to batch, even when the process is tightly controlled. Indeed along with conserved characteristics such as amino acid molar ratio other characteristics such as the specific amino acid sequences will show batch-to-batch variability (1). To address this complexity, for the marketing of GA copies, US and EU regulatory agencies have chosen a generic approach integrated with additional data. However, the implementation is different in the two jurisdictions (Figure 1).
复合药物可以是生物的,如果其活性成分来源于生物来源,也可以是非生物的,如果通过化学合成获得。在这两种情况下,它们的质量在很大程度上取决于制造过程。特别是对于非生物复合药物(nbcd),复杂性可能来自活性物质,如醋酸格拉替默(GA),也可能来自其他来源,如制剂,如脂质体(图1)(1)。GA在美国和欧盟被批准用于多发性硬化症复发型患者的疾病改善治疗。它是一种不完全表征的合成多肽的异质混合物,含有l -丙氨酸、l -赖氨酸、l -谷氨酸、l -酪氨酸,摩尔比为0.43:0.34:0.14:0.09,平均分子量为5 ~ 9 kDa,分布范围为2.5 ~ 20 kDa(2)。氨基酸序列不是完全随机的,这是由原料的理化性质和基本反应方案共同决定的。然而,即使在严格控制过程的情况下,它们也不能在批次之间完全守恒。事实上,除了氨基酸摩尔比等保守特征外,其他特征(如特定的氨基酸序列)也会显示出批次间的可变性(1)。为了解决这种复杂性,对于GA拷贝的营销,美国和欧盟监管机构选择了一种整合了额外数据的通用方法。然而,这两个司法管辖区的实现不同(图1)。
{"title":"Same Product Different Regulatory Approach Around the World: Glatiramer Acetate","authors":"P. Rocco, P. Minghetti","doi":"10.33892/aph.2021.91.112-113","DOIUrl":"https://doi.org/10.33892/aph.2021.91.112-113","url":null,"abstract":"Complex drugs may be either biological, if the active ingredients are derived from a biological source, or non-biological, if obtained by chemical synthesis. In both cases, their quality depends considerably on the manufacturing process. For Non Biological Complex Drugs (NBCDs), in particular, complexity may arise either from the active substance, as in the case of glatiramer acetate (GA), or from other sources, such as the formulation, as in the case of liposomes (Figure 1) (1). GA is approved, in the US and the EU, as a diseasemodifying treatment for patients with relapsing forms of Multiple Sclerosis. It is a heterogeneous mixture of not fully characterized synthetic polypeptides, containing L-alanine, L-lysine, L-glutamic acid, L-tyrosine in the constant molar ratio 0.43:0.34:0.14:0.09, with and average molecular weight from 5 to 9 kDa and distribution range from 2.5 to 20 kDa (2). The amino acid sequences are not completely random, being the result of both the physicochemical properties of the starting materials and the fundamental reaction scheme. However, they are not completely conserved from batch to batch, even when the process is tightly controlled. Indeed along with conserved characteristics such as amino acid molar ratio other characteristics such as the specific amino acid sequences will show batch-to-batch variability (1). To address this complexity, for the marketing of GA copies, US and EU regulatory agencies have chosen a generic approach integrated with additional data. However, the implementation is different in the two jurisdictions (Figure 1).","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"69 6 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"83677605","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Revolutionary Application of 3D-Two-Photon Microscopes for Human Therapy and Drug Research 3d双光子显微镜在人类治疗和药物研究中的革命性应用
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.278-279
Z. Mucsi, Gergely Szalay, G. Katona, B. Rózsa
{"title":"Revolutionary Application of 3D-Two-Photon Microscopes for Human Therapy and Drug Research","authors":"Z. Mucsi, Gergely Szalay, G. Katona, B. Rózsa","doi":"10.33892/aph.2021.91.278-279","DOIUrl":"https://doi.org/10.33892/aph.2021.91.278-279","url":null,"abstract":"","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"48 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"78489905","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Biogenic Iron, Silver and Gold Nanoparticles Against Opportunistic Pathogenic Yeasts and Dermatophytes 生物源铁、银和金纳米颗粒对条件致病性酵母和皮肤真菌的抑制作用
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.310-311
Andrea Rónavári, N. Igaz, M. K. Gopisetty, Bettina Szerencsés, Dávid Kovács, C. Vágvölgyi, Z. Kónya, M. Kiricsi, I. Pfeiffer
Epidemiologic observations indicate that the number of systemic fungal infections has increased significantly during the past decades, however in human mycosis, mainly cutaneous infections predominate, generating major public health concerns and providing much of the impetus for current attempts to develop novel and efficient agents against cutaneous mycosis causing species1. Innovative, environmentally benign and economic nanotechnology-based approaches have recently emerged utilizing principally biological sources to produce nanosized structures with unique antimicrobial properties2. Due to the obvious advantages, the green synthesis of nanoparticles is a rapidly progressing area of the nanobiotechnology. In line with this, the aim of this present study was to investigate the suitability of various green materials such as Parthenocissus quinquefolia plant extract and Phaffia rhodozyma cell-free extract for the preparation of iron nanoparticles (FeNPs), silver nanoparticles (AgNPs) and gold nanoparticles (AuNPs) by biological synthesis and to determine the toxicity of nanoparticles to human keratinocyte cells as well as against various fungal species with a special emphasis on antifungal efficiency against dermatophytes.
流行病学观察表明,在过去的几十年里,全身真菌感染的数量显著增加,然而在人类真菌病中,主要是皮肤感染占主导地位,引起了重大的公共卫生问题,并为目前开发新的有效药物对抗皮肤真菌病引起的物种提供了很大的动力。最近出现了基于创新、环保和经济的纳米技术的方法,主要利用生物来源来生产具有独特抗菌性能的纳米结构2。纳米颗粒的绿色合成由于其明显的优点,是纳米生物技术发展的一个迅速的领域。基于此,本研究旨在探讨各种绿色材料如西番莲植物提取物和法非罗酵母无细胞提取物在制备铁纳米粒子(FeNPs)中的适用性。通过生物合成银纳米颗粒(AgNPs)和金纳米颗粒(AuNPs),并确定纳米颗粒对人角质细胞的毒性以及对各种真菌的毒性,特别强调对皮肤真菌的抗真菌效率。
{"title":"Biogenic Iron, Silver and Gold Nanoparticles Against Opportunistic Pathogenic Yeasts and Dermatophytes","authors":"Andrea Rónavári, N. Igaz, M. K. Gopisetty, Bettina Szerencsés, Dávid Kovács, C. Vágvölgyi, Z. Kónya, M. Kiricsi, I. Pfeiffer","doi":"10.33892/aph.2021.91.310-311","DOIUrl":"https://doi.org/10.33892/aph.2021.91.310-311","url":null,"abstract":"Epidemiologic observations indicate that the number of systemic fungal infections has increased significantly during the past decades, however in human mycosis, mainly cutaneous infections predominate, generating major public health concerns and providing much of the impetus for current attempts to develop novel and efficient agents against cutaneous mycosis causing species1. Innovative, environmentally benign and economic nanotechnology-based approaches have recently emerged utilizing principally biological sources to produce nanosized structures with unique antimicrobial properties2. Due to the obvious advantages, the green synthesis of nanoparticles is a rapidly progressing area of the nanobiotechnology. In line with this, the aim of this present study was to investigate the suitability of various green materials such as Parthenocissus quinquefolia plant extract and Phaffia rhodozyma cell-free extract for the preparation of iron nanoparticles (FeNPs), silver nanoparticles (AgNPs) and gold nanoparticles (AuNPs) by biological synthesis and to determine the toxicity of nanoparticles to human keratinocyte cells as well as against various fungal species with a special emphasis on antifungal efficiency against dermatophytes.","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"8 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"82683520","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Novel E3 Ligase Ligand Libraries for Degradation of Proteins Implicated in Malignant Diseases 新的E3连接酶配体文库用于降解与恶性疾病相关的蛋白质
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.187-188
B. Bertók, G. Dormán, Z. Várkonyi, C. Magyar
{"title":"Novel E3 Ligase Ligand Libraries for Degradation of Proteins Implicated in Malignant Diseases","authors":"B. Bertók, G. Dormán, Z. Várkonyi, C. Magyar","doi":"10.33892/aph.2021.91.187-188","DOIUrl":"https://doi.org/10.33892/aph.2021.91.187-188","url":null,"abstract":"","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"50 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"80910603","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evaluation of Different Pre-Processed Directly Compressible Paracetamol 不同预处理直接可压缩扑热息痛的评价
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.290-292
Musaab Saada, Y. Özalp, Nailla Jiwa, J. T. Chunu, Emre Kara, D. Taşkent, Zeynep Atabay Taşkent
density values. True density was measured using helium pycnometer
密度值。用氦浓度计测定真密度
{"title":"Evaluation of Different Pre-Processed Directly Compressible Paracetamol","authors":"Musaab Saada, Y. Özalp, Nailla Jiwa, J. T. Chunu, Emre Kara, D. Taşkent, Zeynep Atabay Taşkent","doi":"10.33892/aph.2021.91.290-292","DOIUrl":"https://doi.org/10.33892/aph.2021.91.290-292","url":null,"abstract":"density values. True density was measured using helium pycnometer","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"32 3 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"80357898","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Is the Multi-Attribute Method (MAM) the Next Big Thing? A High-Resolution Accurate Mass Multi-Attribute Method for Critical Quality Attribute Monitoring 多属性方法(MAM)是下一个大事件吗?一种高分辨率精确质量多属性关键质量属性监测方法
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.154-155
Orsolya Kóréh, L. Milivojevic
the focus has turned to automation and software development to allow for automated identification and quantitative analysis of each molecular attribute. With automation and software developments, a true benefit of MAM can be realized by building a comprehensive molecular attribute database linked to process conditions which can then be used to increase product and process knowledge throughout the development pipeline. With this increased product and process knowledge, MAM can reduce the amount of time taken to develop a product, reduce the time needed to manufacture and release a product by add-ing efficient process controls, and reduce the time needed to investigate a process issue. MAM can help to solve major manufacturing challenges plaguing the biopharmaceutical industry today as they strive to develop more biotherapeutics on a faster timeline. The multi-attribute for the of monoclonal critical quality describe the optimization and applica-tion of the Multi- Attribute Method as a complete workflow to monitor CQAs of the NISTmAb including glycosylation, deamidation, isomerization, succinimide formation, oxidation,
重点已经转向自动化和软件开发,以允许每个分子属性的自动识别和定量分析。随着自动化和软件的发展,MAM的真正好处可以通过建立一个与工艺条件相关联的综合分子属性数据库来实现,然后可以在整个开发管道中使用该数据库来增加产品和工艺知识。随着产品和工艺知识的增加,MAM可以减少开发产品所需的时间,通过增加有效的过程控制减少制造和发布产品所需的时间,并减少调查过程问题所需的时间。随着生物制药行业努力在更快的时间内开发出更多的生物疗法,MAM可以帮助解决困扰生物制药行业的主要制造挑战。单克隆关键质量的多属性描述了多属性方法的优化和应用,作为一个完整的工作流程来监测NISTmAb的cqa,包括糖基化、脱酰胺化、异构化、琥珀酰亚胺形成、氧化、
{"title":"Is the Multi-Attribute Method (MAM) the Next Big Thing? A High-Resolution Accurate Mass Multi-Attribute Method for Critical Quality Attribute Monitoring","authors":"Orsolya Kóréh, L. Milivojevic","doi":"10.33892/aph.2021.91.154-155","DOIUrl":"https://doi.org/10.33892/aph.2021.91.154-155","url":null,"abstract":"the focus has turned to automation and software development to allow for automated identification and quantitative analysis of each molecular attribute. With automation and software developments, a true benefit of MAM can be realized by building a comprehensive molecular attribute database linked to process conditions which can then be used to increase product and process knowledge throughout the development pipeline. With this increased product and process knowledge, MAM can reduce the amount of time taken to develop a product, reduce the time needed to manufacture and release a product by add-ing efficient process controls, and reduce the time needed to investigate a process issue. MAM can help to solve major manufacturing challenges plaguing the biopharmaceutical industry today as they strive to develop more biotherapeutics on a faster timeline. The multi-attribute for the of monoclonal critical quality describe the optimization and applica-tion of the Multi- Attribute Method as a complete workflow to monitor CQAs of the NISTmAb including glycosylation, deamidation, isomerization, succinimide formation, oxidation,","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"138 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"73017269","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 1
Regulatory Science Challenges : Encouraging Innovation Through an Adaptive Regulatory System 监管科学挑战:通过适应性监管体系鼓励创新
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.106-107
K. Fodor
ATMPs - a new era A boy from Hungary, Zente, was one and a half years old when the crowd-funding campaign to finance his life-saving medicine Zolgensma concluded with a happy end. He was the third European patient that received the new gene therapy, which replaces the function of the missing or nonworking survival motor neuron 1 (SMN1) gene with a new, working copy of a human SMN gene that helps motor neuron cells work properly and survive. From a European perspective, it has been almost 15 years by now since regulatory framework for advanced therapy medicinal products (ATMPs) had been established to ensure the free movement of these medicines within the European Union, to facilitate their access to the EU market, and to foster the competitiveness of European pharmaceutical companies in the field. Zolgensma has been approved in the EU in May 2020. The FDA expects it will be reviewing and approving up to 20 cell and gene therapies each year until 2025. Rapid development of technology and better understanding of the manufacturing challenges are not the only prerequisites of the growth. Assessment of products like Zolgensma requires very specific knowledge and often an adaptive approach from regulators. They have to gain enough experience and need to be able to summarize knowledge in guidelines that would help developers of products that are substantially different from traditional medicines. FDA issued seven new guidelines in January 2020, in which, for example, they highlight the importance of long-term follow-up for gene therapies that offer one-time fix for inherited diseases and where pre-market studies may have limited value. 2. Regulatory tools These examples may already show that rapid change in technology leads to new kinds of medicines that require a properly adapted regulatory system. Patients would expect state-of-the-art medicines within the shortest possible time frame, however, authorities are traditionally more cautious. Still, there are several various initiatives from the EMA and the FDA to foster early access to medicines. Some of these have been available for a longer time. EMA's accelerated assessment reduces the timeframe for review of innovative applications of medicines with major public health interest. Conditional marketing authorisation grants authorization before a complete dataset is available, and compassionate use allows the use of an unauthorized medicine for patients with an unmet medical need. A more recent regulatory tool of EMA is the priority medicines scheme (PRIME) that aims to enhance support for the development of medicines that are expected to make a real difference to patients. Early dialogue between EMA and the developers is a crucial part of the tool, together with accelerated assessment and continuous scientific advice and protocol assistance. Up to now, 282 applications for PRIME eligibility have been assessed by the CHMP of which 95 have received a green light. Most of the applicants are small and medi
当为他的救命药物Zolgensma筹集资金的众筹活动圆满结束时,匈牙利男孩Zente才一岁半。他是第三位接受这种新基因疗法的欧洲患者,这种疗法用一种新的、有效的人类SMN基因拷贝取代了缺失的或不起作用的存活运动神经元1 (SMN1)基因的功能,这种基因有助于运动神经元细胞正常工作和存活。从欧洲的角度来看,先进治疗药物(atmp)的监管框架已经建立了近15年,以确保这些药物在欧盟内部的自由流动,促进它们进入欧盟市场,并促进欧洲制药公司在该领域的竞争力。Zolgensma已于2020年5月在欧盟获得批准。FDA预计,到2025年,每年将审查和批准多达20种细胞和基因疗法。技术的快速发展和对制造挑战的更好理解并不是增长的唯一先决条件。对像Zolgensma这样的产品进行评估需要非常具体的知识,而且监管机构通常需要采用适应性方法。他们必须获得足够的经验,并且需要能够在指导方针中总结知识,以帮助开发与传统药物有很大不同的产品。FDA于2020年1月发布了七项新指南,例如,它们强调了对基因疗法进行长期随访的重要性,这些疗法为遗传性疾病提供了一次性修复,而上市前研究的价值可能有限。2. 这些例子可能已经表明,技术的快速变化导致了需要适当调整的监管系统的新型药物。患者希望在尽可能短的时间内获得最先进的药物,然而,当局传统上更为谨慎。尽管如此,EMA和FDA仍有一些不同的举措来促进药物的早期获取。其中一些已经存在很长时间了。EMA的加速评估缩短了对具有重大公共卫生利益的药物创新应用的审查时间框架。有条件上市许可在完整的数据集可用之前授予许可,而同情使用允许将未经授权的药物用于未满足医疗需求的患者。EMA最近的一个监管工具是优先药物计划(PRIME),旨在加强对有望对患者产生真正影响的药物开发的支持。EMA和开发人员之间的早期对话是该工具的关键部分,同时还有加速评估和持续的科学建议和协议协助。到目前为止,CHMP已经评估了282份PRIME资格申请,其中95份获得了绿灯。大多数申请者是中小型企业,主要治疗领域是肿瘤学。FDA也有类似的项目,如快速通道、突破性治疗和优先审查指定,并且也旨在促进和加速关键药物的开发和上市授权。到2018年,FDA加快了约70%的新药审批,而2010年这一比例约为50%。其结果是,越来越多的药物在上市前证据较少的情况下获得批准,这是一种权衡,大多数致命或使人衰弱的疾病患者可能会接受。然而,有条件的批准需要监管机构在上市后给予强烈关注,而缺乏足够的证据有时会导致难以做出决定。2019年4月,快速追踪的癌症药物Lartruvo被撤回,因为一项大型研究无法证明有利的收益-风险概况,这是之前在较小的患者群体中建立的。预计监管机构的做法不会改变,但此类案例的经验将逐渐融入决策过程。除了这个真实世界的证据(RWE)和病人记录的结果也可能有助于决策。3.快速发展的生物技术并不是唯一需要适应性监管方法的领域。随着智能手机和传感器开辟了生成数据的新途径,数字医学是一个新领域。例如,在随机试验不可行的情况下,收集和分析RWE似乎是单组研究的一个很好的解决方案。FDA已经批准了易于使用的设备,这些设备能够跟踪我们身体的几个生理系统,这反过来又可以促进这一领域的发展。除了这些简单的设备,人工智能(AI)和认知机器学习方面的数字革命是我们的监管系统应该应对的另一个挑战。
{"title":"Regulatory Science Challenges : Encouraging Innovation Through an Adaptive Regulatory System","authors":"K. Fodor","doi":"10.33892/aph.2021.91.106-107","DOIUrl":"https://doi.org/10.33892/aph.2021.91.106-107","url":null,"abstract":"ATMPs - a new era A boy from Hungary, Zente, was one and a half years old when the crowd-funding campaign to finance his life-saving medicine Zolgensma concluded with a happy end. He was the third European patient that received the new gene therapy, which replaces the function of the missing or nonworking survival motor neuron 1 (SMN1) gene with a new, working copy of a human SMN gene that helps motor neuron cells work properly and survive. From a European perspective, it has been almost 15 years by now since regulatory framework for advanced therapy medicinal products (ATMPs) had been established to ensure the free movement of these medicines within the European Union, to facilitate their access to the EU market, and to foster the competitiveness of European pharmaceutical companies in the field. Zolgensma has been approved in the EU in May 2020. The FDA expects it will be reviewing and approving up to 20 cell and gene therapies each year until 2025. Rapid development of technology and better understanding of the manufacturing challenges are not the only prerequisites of the growth. Assessment of products like Zolgensma requires very specific knowledge and often an adaptive approach from regulators. They have to gain enough experience and need to be able to summarize knowledge in guidelines that would help developers of products that are substantially different from traditional medicines. FDA issued seven new guidelines in January 2020, in which, for example, they highlight the importance of long-term follow-up for gene therapies that offer one-time fix for inherited diseases and where pre-market studies may have limited value. 2. Regulatory tools These examples may already show that rapid change in technology leads to new kinds of medicines that require a properly adapted regulatory system. Patients would expect state-of-the-art medicines within the shortest possible time frame, however, authorities are traditionally more cautious. Still, there are several various initiatives from the EMA and the FDA to foster early access to medicines. Some of these have been available for a longer time. EMA's accelerated assessment reduces the timeframe for review of innovative applications of medicines with major public health interest. Conditional marketing authorisation grants authorization before a complete dataset is available, and compassionate use allows the use of an unauthorized medicine for patients with an unmet medical need. A more recent regulatory tool of EMA is the priority medicines scheme (PRIME) that aims to enhance support for the development of medicines that are expected to make a real difference to patients. Early dialogue between EMA and the developers is a crucial part of the tool, together with accelerated assessment and continuous scientific advice and protocol assistance. Up to now, 282 applications for PRIME eligibility have been assessed by the CHMP of which 95 have received a green light. Most of the applicants are small and medi","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"28 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"73460152","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Advanced Drug Delivery Systems: From Nano- and Microparticles to Smart Pills 先进的药物输送系统:从纳米和微粒到智能药丸
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.128-129
I. Antal
microcapsules are compo-nents of multiparticulate drug carriers structural patient-centered Depending on the mode of be into solid (capsules, tablets, semi-solid (gels, creams, pastes) or liquid (solutions, suspen-sions, or parenteral) dosage forms
微胶囊是多颗粒药物载体的组成部分,其结构以患者为中心,取决于其进入固体(胶囊、片剂、半固体(凝胶、膏状、糊状)或液体(溶液、悬浮液或非肠道)剂型的方式
{"title":"Advanced Drug Delivery Systems: From Nano- and Microparticles to Smart Pills","authors":"I. Antal","doi":"10.33892/aph.2021.91.128-129","DOIUrl":"https://doi.org/10.33892/aph.2021.91.128-129","url":null,"abstract":"microcapsules are compo-nents of multiparticulate drug carriers structural patient-centered Depending on the mode of be into solid (capsules, tablets, semi-solid (gels, creams, pastes) or liquid (solutions, suspen-sions, or parenteral) dosage forms","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"46 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"88831325","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Future of the Hungarian Drug Market : Role of the Hungarian Regulatory Authority 匈牙利药品市场的未来:匈牙利监管机构的作用
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.131-132
M. Szentiványi
The National Institute of Pharmacy and Nutrition (NIPN) has a long tradition as the safeguard of patient safety and as an institution that is committed to ensure that Hungarian patients have access to good quality, safe and efficacious medicines. During the last few years responsibilities of the Institute has been broaden and now we are also a key player in the regulation of special food and medical devices. During the past two decades a transition can be seen in the role of medicines agencies. While in the past the role of regulators was mainly acting as a gatekeeper, nowadays we also need to act as enablers. And while ensuring regulatory compliance of new medicines is still a key activity, our role cannot be narrowed down to the review of documents and other authorization tasks. This is due to the enormous development in the field of biotechnology, precision medicine, the revolution in synthetic biology, just to name a few. On one hand regulators need to be prepared for these new scientific challenges, on the other hand we have to be ready to give the necessary regulatory support to those developers that are coming from various fields and have limited knowledge and experience in regulatory issues.
国家药学和营养研究所(NIPN)作为患者安全保障和致力于确保匈牙利患者获得高质量、安全和有效药物的机构有着悠久的传统。在过去的几年里,研究所的职责已经扩大,现在我们也是特殊食品和医疗设备监管的关键参与者。在过去二十年中,可以看到药品机构的作用发生了转变。过去,监管机构的角色主要是看门人,而如今,我们也需要充当推动者。虽然确保新药的法规遵从性仍然是一项关键活动,但我们的作用不能缩小到审查文件和其他授权任务。这是由于生物技术领域的巨大发展,精准医学,合成生物学的革命,仅举几例。一方面,监管机构需要为这些新的科学挑战做好准备,另一方面,我们必须准备好为那些来自不同领域、在监管问题上知识和经验有限的开发商提供必要的监管支持。
{"title":"Future of the Hungarian Drug Market : Role of the Hungarian Regulatory Authority","authors":"M. Szentiványi","doi":"10.33892/aph.2021.91.131-132","DOIUrl":"https://doi.org/10.33892/aph.2021.91.131-132","url":null,"abstract":"The National Institute of Pharmacy and Nutrition (NIPN) has a long tradition as the safeguard of patient safety and as an institution that is committed to ensure that Hungarian patients have access to good quality, safe and efficacious medicines. During the last few years responsibilities of the Institute has been broaden and now we are also a key player in the regulation of special food and medical devices. During the past two decades a transition can be seen in the role of medicines agencies. While in the past the role of regulators was mainly acting as a gatekeeper, nowadays we also need to act as enablers. And while ensuring regulatory compliance of new medicines is still a key activity, our role cannot be narrowed down to the review of documents and other authorization tasks. This is due to the enormous development in the field of biotechnology, precision medicine, the revolution in synthetic biology, just to name a few. On one hand regulators need to be prepared for these new scientific challenges, on the other hand we have to be ready to give the necessary regulatory support to those developers that are coming from various fields and have limited knowledge and experience in regulatory issues.","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"2 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"78573439","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Ionone: The Molecule that Shaped the History of Western Civilization Ionone:塑造西方文明史的分子
Pub Date : 2021-11-15 DOI: 10.33892/aph.2021.91.158-159
G. Petroianu
Cleopatra VII (69–30 BC) was the last Ptolemaic ruler of Hellenistic Egypt (the Dynasty was started upon the death of Alexander the Great by Ptolemy the Savior, one of his generals or body guards, and ended with the death of Cleopatra and the Roman conquest in 30 BC). The Queen is probably best known for her love affairs with Julius Caesar (10044 BC) and then Marcus Antonius (83-30 BC). Rightly or wrongly she became the epitome of shrewd seduction, leading brave Roman commanders on a path to debauchery and destruction. Among the customs of the time was the heavy use of perfumes. The sails of the ship on which Cleopatra received Marcus Antonius, we are told by Enobarbus in Shakespeare’s eponymous play, were soaked in fragrances: Purple the sails and so perfumed that the winds were lovesick. Cleopatra’s willingness to please went beyond the external use of perfumes; attributed to her is the ingestion of small amounts of turpentine [the resin of the terebinth tree (Pistacia terebinthus)] or of the derived oil (Oleum terebinthinae) with the purpose of conferring to her urine a more pleasing scent reminding of violets (Figure 1). In his textbook of pediatrics John Apley (19081980) states I have sniffed a smell like violets in the urine of a child who drank turpentine, as Cleopatra of Egypt did for that effect (1,2).
克利奥帕特拉七世(公元前69-30年)是希腊化埃及的最后一位托勒密统治者(王朝在亚历山大大帝去世后由他的一位将军或保镖托勒密救世主开始,并于公元前30年克利奥帕特拉死亡和罗马征服而结束)。女王最出名的可能是她与朱利叶斯·凯撒(公元前10044年)和马库斯·安东尼(公元前83-30年)的恋情。不管是对是错,她成了精明诱惑的缩影,带领勇敢的罗马指挥官走上了放荡和毁灭的道路。当时的习俗之一是大量使用香水。在莎士比亚的同名戏剧中,埃诺巴布斯告诉我们,克利奥帕特拉接待马库斯·安东尼乌斯的那艘船的船帆浸透了芬芳:船帆是紫色的,香气如此之浓,以至于风都患了相思病。克利奥帕特拉取悦人的意愿不仅仅是在外面使用香水;她的原因是摄入了少量的松节油(松脂树(Pistacia terebinthus)的树脂)或从松节油中提取的油(oil terebinthinae),目的是使她的尿液具有更令人愉快的紫罗兰气味(图1)。约翰·阿普雷(John Apley, 1908 - 1980)在他的儿科学教科书中说,我在一个喝了松节油的孩子的尿液中闻到了一种紫罗兰气味,埃及的克利奥帕特拉就是这样做的(1,2)。
{"title":"Ionone: The Molecule that Shaped the History of Western Civilization","authors":"G. Petroianu","doi":"10.33892/aph.2021.91.158-159","DOIUrl":"https://doi.org/10.33892/aph.2021.91.158-159","url":null,"abstract":"Cleopatra VII (69–30 BC) was the last Ptolemaic ruler of Hellenistic Egypt (the Dynasty was started upon the death of Alexander the Great by Ptolemy the Savior, one of his generals or body guards, and ended with the death of Cleopatra and the Roman conquest in 30 BC). The Queen is probably best known for her love affairs with Julius Caesar (10044 BC) and then Marcus Antonius (83-30 BC). Rightly or wrongly she became the epitome of shrewd seduction, leading brave Roman commanders on a path to debauchery and destruction. Among the customs of the time was the heavy use of perfumes. The sails of the ship on which Cleopatra received Marcus Antonius, we are told by Enobarbus in Shakespeare’s eponymous play, were soaked in fragrances: Purple the sails and so perfumed that the winds were lovesick. Cleopatra’s willingness to please went beyond the external use of perfumes; attributed to her is the ingestion of small amounts of turpentine [the resin of the terebinth tree (Pistacia terebinthus)] or of the derived oil (Oleum terebinthinae) with the purpose of conferring to her urine a more pleasing scent reminding of violets (Figure 1). In his textbook of pediatrics John Apley (19081980) states I have sniffed a smell like violets in the urine of a child who drank turpentine, as Cleopatra of Egypt did for that effect (1,2).","PeriodicalId":6941,"journal":{"name":"Acta pharmaceutica Hungarica","volume":"1 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"83760275","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
期刊
Acta pharmaceutica Hungarica
全部 Acc. Chem. Res. ACS Applied Bio Materials ACS Appl. Electron. Mater. ACS Appl. Energy Mater. ACS Appl. Mater. Interfaces ACS Appl. Nano Mater. ACS Appl. Polym. Mater. ACS BIOMATER-SCI ENG ACS Catal. ACS Cent. Sci. ACS Chem. Biol. ACS Chemical Health & Safety ACS Chem. Neurosci. ACS Comb. Sci. ACS Earth Space Chem. ACS Energy Lett. ACS Infect. Dis. ACS Macro Lett. ACS Mater. Lett. ACS Med. Chem. Lett. ACS Nano ACS Omega ACS Photonics ACS Sens. ACS Sustainable Chem. Eng. ACS Synth. Biol. Anal. Chem. BIOCHEMISTRY-US Bioconjugate Chem. BIOMACROMOLECULES Chem. Res. Toxicol. Chem. Rev. Chem. Mater. CRYST GROWTH DES ENERG FUEL Environ. Sci. Technol. Environ. Sci. Technol. Lett. Eur. J. Inorg. Chem. IND ENG CHEM RES Inorg. Chem. J. Agric. Food. Chem. J. Chem. Eng. Data J. Chem. Educ. J. Chem. Inf. Model. J. Chem. Theory Comput. J. Med. Chem. J. Nat. Prod. J PROTEOME RES J. Am. Chem. Soc. LANGMUIR MACROMOLECULES Mol. Pharmaceutics Nano Lett. Org. Lett. ORG PROCESS RES DEV ORGANOMETALLICS J. Org. Chem. J. Phys. Chem. J. Phys. Chem. A J. Phys. Chem. B J. Phys. Chem. C J. Phys. Chem. Lett. Analyst Anal. Methods Biomater. Sci. Catal. Sci. Technol. Chem. Commun. Chem. Soc. Rev. CHEM EDUC RES PRACT CRYSTENGCOMM Dalton Trans. Energy Environ. Sci. ENVIRON SCI-NANO ENVIRON SCI-PROC IMP ENVIRON SCI-WAT RES Faraday Discuss. Food Funct. Green Chem. Inorg. Chem. Front. Integr. Biol. J. Anal. At. Spectrom. J. Mater. Chem. A J. Mater. Chem. B J. Mater. Chem. C Lab Chip Mater. Chem. Front. Mater. Horiz. MEDCHEMCOMM Metallomics Mol. Biosyst. Mol. Syst. Des. Eng. Nanoscale Nanoscale Horiz. Nat. Prod. Rep. New J. Chem. Org. Biomol. Chem. Org. Chem. Front. PHOTOCH PHOTOBIO SCI PCCP Polym. Chem.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1