scholarly journals Advances in Antifungal Drug Development: An Up-To-Date Mini Review

2021 ◽  
Vol 14 (12) ◽  
pp. 1312
Author(s):  
Ghada Bouz ◽  
Martin Doležal
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Clinical Development ◽  
Antifungal Drug ◽  
Current Status ◽  
Special Focus ◽  
High Toxicity ◽  
Clinical Stages ◽  
Promising Treatment ◽  
Novel Targets

The utility of clinically available antifungals is limited by their narrow spectrum of activity, high toxicity, and emerging resistance. Antifungal drug discovery has always been a challenging area, since fungi and their human host are eukaryotes, making it difficult to identify unique targets for antifungals. Novel antifungals in clinical development include first-in-class agents, new structures for an established target, and formulation modifications to marketed antifungals, in addition to repurposed agents. Membrane interacting peptides and aromatherapy are gaining increased attention in the field. Immunotherapy is another promising treatment option, with antifungal antibodies advancing into clinical trials. Novel targets for antifungal therapy are also being discovered, allowing the design of new promising agents that may overcome the resistance issue. In this mini review, we will summarize the current status of antifungal drug pipelines in clinical stages, and the most recent advancements in preclinical antifungal drug development, with special focus on their chemistry.

scholarly journals Current Status and Trend of Clinical Development of Orphan Drugs in China

2021 ◽  
Author(s):  
Ziling Xiang ◽  
Wengao Jiang ◽  
Bo Yan ◽  
Junhao Jiang ◽  
Hang Zheng
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
Clinical Development ◽  
Current Status ◽  
Policy Makers ◽  
Critical Issues ◽  
Low Coverage ◽  
Orphan Drug Development

Abstract Background:Rare diseases have been increasingly recognized as medical and healthy burden worldwide, a growing demand for the development of orphan drugs emerges subsequently. Therefore, it is of great interest for both the regulatory agency and pharmaceutical companies to keep tract on the clinical orphan drug development in China.Objective and Method:This study aims to reveal the current situation and trend of the clinical development of orphan drugs in China, based on the data collected from the Platform for Drug Clinical Trials and Information Registration（http://www.chinadrugtrials.org.cn）of China Food and Drug Administration, dating from 2013 to March 8, 2021.Results:A total of 246 clinical trials for orphan drugs are extracted from the Platform, covering 22 rare diseases and 90 drugs. Among the 22 rare diseases, 3 (14 %) have more than 50 trials each , 17 (77%) had less than10 trials, and 10 (46%) only with one trial. Among 90 orphan drugs, 60 (67%) were chemical drugs, and 30 (33 %) were biological products. In addition, international multi-center trials accounts for nearly 10% of the total trials. The number of the trials with the Data Monitoring Security Committee (DMC) is 25 (10%) and the number of the trials with the trial injury insurance for subjects is 154 (63%). Furthermore, more than half of the total trials are carried in east (333, 30%) and north China (298, 27%), whereas a small portion are in the northwest (62, 6%) and northeast china (45, 4%).Conclusions:The clinical development of orphan drugs for rare diseases in China has made some progress in the passing decades. However, a couple of critical issues still need to be addressed, such as unmet needs for some rare diseases, low coverage of insurance and DMC, and uneven distribution of medical resources for clinical researches. Recommendations are put forward accordingly, which can provide improvement goals for policy makers and stakeholders involved in drug development for rare diseases.

scholarly journals Exon-Skipping in Duchenne Muscular Dystrophy

2021 ◽  
pp. 1-16
Author(s):  
Shin’ichi Takeda ◽  
Paula R. Clemens ◽  
Eric P. Hoffman
Keyword(s):  
Clinical Trials ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development ◽  
Rare Disease ◽  
19Th Century ◽  
Exon Skipping ◽  
Clinical Development ◽  
Therapeutic Development ◽  
The 19Th Century

Duchenne muscular dystrophy (DMD) is a devastating, rare disease. While clinically described in the 19th century, the genetic foundation of DMD was not discovered until more than 100 years later. This genetic understanding opened the door to the development of genetic treatments for DMD. Over the course of the last 30 years, the research that supports this development has moved into the realm of clinical trials and regulatory drug approvals. Exon skipping to therapeutically restore the frame of an out-of-frame dystrophin mutation has taken center stage in drug development for DMD. The research reviewed here focuses on the clinical development of exon skipping for the treatment of DMD. In addition to the generation of clinical treatments that are being used for patient care, this research sets the stage for future therapeutic development with a focus on increasing efficacy while providing safety and addressing the multi-systemic aspects of DMD.

scholarly journals Human coronaviruses and therapeutic drug discovery

2021 ◽  
Vol 10 (1) ◽  
Author(s):  
Lan-Gui Song ◽  
Qing-Xing Xie ◽  
Hui-Lin Lao ◽  
Zhi-Yue Lv
Keyword(s):  
Clinical Trials ◽  
Drug Discovery ◽  
Drug Development ◽  
Adverse Reactions ◽  
Current Status ◽  
Therapeutic Drug ◽  
Pharmacological Effects ◽  
Comprehensive Understanding ◽  
Human Coronaviruses ◽  
Made In

Abstract Background Coronaviruses (CoVs) are distributed worldwide and have various susceptible hosts; CoVs infecting humans are called human coronaviruses (HCoVs). Although HCoV-specific drugs are still lacking, many potent targets for drug discovery are being explored, and many vigorously designed clinical trials are being carried out in an orderly manner. The aim of this review was to gain a comprehensive understanding of the current status of drug development against HCoVs, particularly severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Main text A scoping review was conducted by electronically searching research studies, reviews, and clinical trials in PubMed and the CNKI. Studies on HCoVs and therapeutic drug discovery published between January 2000 and October 2020 and in English or Chinese were included, and the information was summarized. Of the 3248 studies identified, 159 publication were finally included. Advances in drug development against HCoV, especially SARS-CoV-2, are summarized under three categories: antiviral drugs aimed at inhibiting the HCoV proliferation process, drugs acting on the host's immune system, and drugs derived from plants with potent activity. Furthermore, clinical trials of drugs targeting SARS-CoV-2 are summarized. Conclusions During the spread of COVID-19 outbreak, great efforts have been made in therapeutic drug discovery against the virus, although the pharmacological effects and adverse reactions of some drugs under study are still unclear. However, well-designed high-quality studies are needed to further study the effectiveness and safety of these potential drugs so as to provide valid recommendations for better control of the COVID-19 pandemic.

scholarly journals Mapping the Global Research and Clinical Trials in COVID-19

2020 ◽  
Author(s):  
Ranjana Aggarwal ◽  
Sujit Bhattacharya ◽  
Shubham Singh
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Value Chain ◽  
Academic Research ◽  
Current Status ◽  
Innovation Activity ◽  
Research Papers ◽  
Private Funding ◽  
Development Activity ◽  
Research And Innovation

AbstractCOVID-19 has created an unprecedented level of research and innovation activity globally to bring out drug to control or cure the disease, and develop vaccine for long time prevention. A ‘new/better normal’ is emerging that is trying to push this time period for drug development and vaccine within a year and earlier than that from typical 8 to 10 years for drugs and 10 to 15 years for vaccine. This is happening due to multiple factors: strong policy push by different government that includes dedicated investment, speeding up regulation process, multiple agencies involvement, and multilateral bodies led by WHO trying to create a global platform, huge grants from private funding bodies, strategic linkages across the whole research and innovation value chain between firms, academic, research organisations and start-ups.The paper maps the research papers and ongoing clinical trials to provide an informed view of the current status of the research and drug development activity as seen through the lens of research papers and clinical trials. The intended goal of this study is to help the research community and policy makers to keep track of highly relevant research and drug development in COVID-19.

NEW ORIGINAL DRUG ROSEOFUNGIN-AS, OINTMENT 2%

2021 ◽  
pp. 55-64
Author(s):  
А.К. САДАНОВ ◽  
В.Э. БЕРЕЗИН ◽  
И.Р. КУЛМАГАМБЕТОВ ◽  
Л.П. ТРЕНОЖНИКОВА ◽  
А.С. БАЛГИМБАЕВА
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Physicochemical Properties ◽  
Phase I ◽  
Mechanism Of Action ◽  
Chemical Structure ◽  
Antifungal Drug ◽  
Polyene Antibiotic

В статье приводятся сведения о разработке нового отечественного противогрибкового препарата «Розеофунгин-АС, мазь 2%» для наружного применения на основе оригинального природного полиенового антибиотика розеофунгина. Приводятся данные о продуценте антибиотика, процессе его биосинтеза и получения, его физико-химических свойствах и химической структуре, рассматриваются его антифунгальные и антивирусные свойства, механизм его действия, а также основные этапы разработки противогрибкового препарата - доклинические и I, II и III фазы клинических исследований. This paper provides the information on the development of new domestic antifungal drug Roseofungin-AS, ointment 2% for external use based on the original natural polyene antibiotic roseofungin. Data on the antibiotic producer, the process of its biosynthesis and production, its physicochemical properties and chemical structure are presented, its antifungal and antiviral properties, the mechanism of action as well as the main stages of the antifungal drug development including preclinical and phase I, II, III clinical trials are discussed.

scholarly journals Clinical Trials in a Dish: A Perspective on the Coming Revolution in Drug Development

2018 ◽  
Vol 23 (8) ◽  
pp. 765-776 ◽  
Author(s):  
Bernard Fermini ◽  
Shawn T. Coyne ◽  
Kevin P. Coyne
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Clinical Development ◽  
New Drugs ◽  
Attrition Rate ◽  
Preclinical Drug Development ◽  
Current Article ◽  
Induced Pluripotent ◽  
The Cost ◽  
Further Development

The pharmaceutical industry is facing unprecedented challenges as the cost of developing new drugs has reached unsustainable levels, fueled in large parts by a high attrition rate in clinical development. Strategies to bridge studies between preclinical testing and clinical trials are needed to reduce the knowledge gap and allow earlier decisions to be made on the continuation or discontinuation of further development of drugs. The discovery and development of human induced pluripotent stem cells (hiPSCs) have opened up new avenues that support the concept of screening for cell-based safety and toxicity at the level of a population. This approach, termed “Clinical Trials in a Dish” (CTiD), allows testing medical therapies for safety or efficacy on cells collected from a representative sample of human patients, before moving into actual clinical trials. It can be applied to the development of drugs for specific populations, and it allows predicting not only the magnitude of effects but also the incidence of patients in a population who will benefit or be harmed by these drugs. This, in turn, can lead to the selection of safer drugs to move into clinical development, resulting in a reduction in attrition. The current article offers a perspective of this new model for “humanized” preclinical drug development.

Clinical Drug Development for the Treatment of Pulmonary Arterial Hypertension: Collaboration With the Pharmaceutical Industry and Regulatory Agencies

2010 ◽  
Vol 9 (4) ◽  
pp. 214-219
Author(s):  
Robyn J. Barst
Keyword(s):  
Clinical Trials ◽  
Pulmonary Arterial Hypertension ◽  
Drug Development ◽  
Phase 1 ◽  
Preclinical Studies ◽  
Phase 2 ◽  
Phase 3 ◽  
Preclinical Testing ◽  
New Drug ◽  
Pulmonary Arterial

Drug development is the entire process of introducing a new drug to the market. It involves drug discovery, screening, preclinical testing, an Investigational New Drug (IND) application in the US or a Clinical Trial Application (CTA) in the EU, phase 1–3 clinical trials, a New Drug Application (NDA), Food and Drug Administration (FDA) review and approval, and postapproval studies required for continuing safety evaluation. Preclinical testing assesses safety and biologic activity, phase 1 determines safety and dosage, phase 2 evaluates efficacy and side effects, and phase 3 confirms efficacy and monitors adverse effects in a larger number of patients. Postapproval studies provide additional postmarketing data. On average, it takes 15 years from preclinical studies to regulatory approval by the FDA: about 3.5–6.5 years for preclinical, 1–1.5 years for phase 1, 2 years for phase 2, 3–3.5 years for phase 3, and 1.5–2.5 years for filing the NDA and completing the FDA review process. Of approximately 5000 compounds evaluated in preclinical studies, about 5 compounds enter clinical trials, and 1 compound is approved (Tufts Center for the Study of Drug Development, 2011). Most drug development programs include approximately 35–40 phase 1 studies, 15 phase 2 studies, and 3–5 pivotal trials with more than 5000 patients enrolled. Thus, to produce safe and effective drugs in a regulated environment is a highly complex process. Against this backdrop, what is the best way to develop drugs for pulmonary arterial hypertension (PAH), an orphan disease often rapidly fatal within several years of diagnosis and in which spontaneous regression does not occur?

New Anti-VEGF Drugs in Ophthalmology

2020 ◽  
Vol 21 (12) ◽  
pp. 1194-1200
Author(s):  
Claudio Campa
Keyword(s):  
Clinical Trials ◽  
Delivery System ◽  
Clinical Development ◽  
Advanced Stage ◽  
Phase 3 ◽  
Anti Vegf

: This review focuses on 5 new anti-VEGF drugs in the advanced stage of clinical development (i.e., phase 3): conbercept, brolucizumab, port delivery system with ranibizumab, abicipar pegol and faricimab. : Results of clinical trials and the advantages of each drug compared to the available molecules are discussed in detail.

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