Drugs最新文献

{"title":"Chronic Obstructive Pulmonary Disease (COPD): Developments in Pharmacological Treatments.","authors":"Dave Singh, Andrew Higham, Alexander G Mathioudakis, Augusta Beech","doi":"10.1007/s40265-025-02188-8","DOIUrl":"https://doi.org/10.1007/s40265-025-02188-8","url":null,"abstract":"<p><p>The immediate goals of pharmacological management in chronic obstructive pulmonary disease (COPD) are to minimise symptoms and improve exercise performance. The longer-term goals are to reduce the future risk of exacerbations, lung function decline and mortality. It is now recognised that a subset of COPD patients have type 2 inflammation, which is identified by the presence of higher blood eosinophil counts (BEC). Individuals with higher BEC show a greater response to pharmacological interventions targeting type 2 inflammation, including inhaled corticosteroids and the monoclonal antibody, dupilumab. The use of BEC as a biomarker to guide pharmacological treatment has enabled a precision medicine approach in COPD. This article reviews recent advances in the pharmacological treatment of COPD, encompassing the optimum use of inhaled combination treatments and the evidence to support the use of the novel inhaled phosphodiesterase inhibitor ensifentrine and monoclonal antibodies in patients with COPD.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144109847","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Vonoprazan: A Review in Erosive Esophagitis and Non-Erosive Gastro-Esophageal Reflux Disease.","authors":"Simon Fung","doi":"10.1007/s40265-025-02193-x","DOIUrl":"https://doi.org/10.1007/s40265-025-02193-x","url":null,"abstract":"<p><p>Vonoprazan (Voquezna^®) is a potassium-competitive acid blocker (PCAB) with improved pharmacodynamic and pharmacokinetic properties compared with proton-pump inhibitors (PPIs) that allow for stronger control of gastric acid with once-daily oral administration and no requirement to be taken in relation to timing of meals for optimal efficacy. In the USA, vonoprazan has been approved as a first-in-class treatment for healing and maintenance of healing of erosive esophagitis, and for relief of heartburn in adult patients with erosive esophagitis and non-erosive gastro-esophageal reflux disease (GERD). In pivotal phase III trials, vonoprazan was non-inferior to the PPI lansoprazole in healing and superior to lansoprazole in maintenance of healing of erosive esophagitis, and was superior to placebo for treating heartburn in US patients with non-erosive GERD. Vonoprazan was generally well tolerated; the most common adverse events included abdominal pain, constipation, diarrhea, nausea, and dyspepsia. Vonoprazan is, therefore, a valuable addition to the therapies available for adults with erosive esophagitis and non-erosive GERD.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093123","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment of Advanced HIV in the Modern Era.","authors":"Joseph M Garland, Haim Mayan, Rami Kantor","doi":"10.1007/s40265-025-02181-1","DOIUrl":"https://doi.org/10.1007/s40265-025-02181-1","url":null,"abstract":"<p><p>Antiretroviral therapy has transformed human immunodeficiency virus (HIV) infection from a fatal illness into a manageable chronic condition. However, despite remarkable progress, the HIV epidemic remains a global health challenge, with ambitious targets such as 95-95-95 by 2030 at risk of being unmet. While antiretroviral therapy availability has expanded worldwide, gaps persist, including unawareness of HIV status, inconsistent medication uptake, and limited engagement in care across diverse settings. Advanced HIV represents a particularly challenging yet underexplored aspect of HIV care. Its definition is complex, complicating efforts to address the needs of this vulnerable population. This review characterizes advanced HIV populations, defines them by spectra of immune suppression, antiretroviral therapy exposure, and drug resistance, and explores contemporary approaches to their management, with a particular focus on drug resistance and its clinical implications in modern HIV care. It highlights the unique challenges faced by individuals presenting late to care, those with limited care engagement, and aging populations with long-term exposure to HIV and antiretroviral therapy. By defining these populations, refining our understanding of advanced HIV, and addressing the diverse needs of affected individuals, providers can enhance outcomes and develop strategies to overcome barriers to care. Bridging these critical gaps is essential to advancing global efforts to end the HIV epidemic, both in the USA and worldwide.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143996167","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Navigating Psoriatic Arthritis: Treatment Pathways and Patient-Specific Strategies for Improved Outcomes.","authors":"Rubén Queiro, José Antonio Pinto-Tasende, Carlos Montilla-Morales","doi":"10.1007/s40265-025-02192-y","DOIUrl":"https://doi.org/10.1007/s40265-025-02192-y","url":null,"abstract":"<p><p>Psoriatic arthritis (PsA) is a multifaceted chronic immune-mediated disease characterized by joint, skin, nail and entheseal involvement, affecting approximately 0.3-1% of the global population. In recent years, the treatment options for PsA have expanded from traditional nonsteroidal anti-inflammatory drugs and conventional synthetic disease-modifying antirheumatic drugs (DMARDs) to include biologic DMARDs and targeted synthetic DMARDs. Owing to the heterogeneity of the disease and prevalence of comorbidities, the selection and sequence of treatment are often unclear. In this narrative review, we outline the patient journey from diagnosis through various treatment lines, from conventional therapies to bDMARDS and tsDMARDs, and the considerations for treatment sequencing in patients who do not achieve an adequate response. We examine the factors influencing treatment response, such as disease severity, predominant disease domain, comorbidities, genetic variations, pharmacokinetic and immunogenicity issues. We highlight the importance of identifying robust biomarkers to predict response and the need to determine patient-specific factors, including the contribution of inflammatory mechanisms to disease activity, to inform treatment strategies and improve long-term outcomes. Promising results with more recently marketed biologic and targeted synthetic DMARDs, and the use of combination treatment approaches, offer new options for managing treatment-experienced patients.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144062858","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Role of CPX-351 in the Acute Myeloid Leukemia Treatment Landscape: Mechanism of Action, Efficacy, and Safety.","authors":"Livio Pagano, Romano Danesi, Edoardo Benedetti, Riccardo Morgagni, Luigina Romani, Adriano Venditti","doi":"10.1007/s40265-025-02194-w","DOIUrl":"https://doi.org/10.1007/s40265-025-02194-w","url":null,"abstract":"<p><p>CPX-351 (also known as VYXEOS^® or Vyxeos liposomal) is a dual-drug liposomal encapsulation of cytarabine and daunorubicin in a synergistic 5:1 molar ratio and was the first example that utilized CombiPlex^®, a combination drug technology platform. Superior efficacy with CPX-351 in the pivotal phase 3 randomized clinical trial versus its conventional free-drug counterpart led to its approval for the treatment of newly diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes in multiple countries. Emerging evidence indicates that CPX-351 affords additional benefits compared with conventional chemotherapy, including protection against intestinal dysbiosis and fungal colonization, fewer infectious complications, and a lower incidence of cardiotoxicity. This review examines the mechanisms underlying CPX-351's therapeutic effects and highlights its expanding role in AML treatment by summarizing efficacy and safety data from preclinical models, the pivotal clinical trial, and real-world studies. Particular focus is given to recent findings on CPX-351's intestinal and cardioprotective properties, which together strengthen its safety and efficacy profile compared with conventional chemotherapy.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143973772","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Datopotamab Deruxtecan: First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40265-025-02185-x","DOIUrl":"https://doi.org/10.1007/s40265-025-02185-x","url":null,"abstract":"<p><p>Datopotamab deruxtecan (DATROWAY^®) is a trophoblast cell surface antigen 2 (TROP2)-directed antibody-drug conjugate being developed by Daiichi Sankyo and AstraZeneca for the treatment of solid tumours. On 27 December 2024, datopotamab deruxtecan was approved in Japan for the treatment of adult patients with hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative unresectable or recurrent breast cancer after prior chemotherapy. The drug has since been approved on 17 January 2025 in the USA for the treatment of adult patients with unresectable or metastatic HR positive, HER2 negative breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease. Datopotamab deruxtecan has also been approved in the EU for the treatment of adult patients with unresectable or metastatic HR positive, HER2 negative breast cancer who have received endocrine therapy and at least one line of chemotherapy in the advanced setting. Regulatory review of datopotamab deruxtecan in breast cancer is underway in Canada and China. This article summarizes the milestones in the development of datopotamab deruxtecan leading to this first approval for the treatment of HR positive, HER2 negative breast cancer.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143969071","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Stapokibart: First Approval.","authors":"Matt Shirley","doi":"10.1007/s40265-025-02151-7","DOIUrl":"10.1007/s40265-025-02151-7","url":null,"abstract":"<p><p>Stapokibart (Kangyueda^®; ^®) is a humanised IgG4 monoclonal antibody targeted against the interleukin (IL)-4 receptor alpha subunit (IL-4Rα). By binding IL-4Rα, stapokibart blocks the binding by (and subsequent signalling of) IL-4 and IL-13, two type 2 cytokines. Stapokibart is being developed by KeyMed Biosciences for the treatment of atopic dermatitis and other type 2 inflammatory diseases. In September 2024, stapokibart received its first approval, in China, for use in the treatment of moderate-to-severe atopic dermatitis in adults whose disease is poorly controlled by, or not suitable for, topical medications. Subsequently, stapokibart additionally received approval in China for use in the treatment of chronic rhinosinusitis with nasal polyps (December 2024) and for the treatment of seasonal allergic rhinitis (February 2025). Stapokibart is also under clinical evaluation for use in the treatment of moderate-to-severe asthma and chronic obstructive pulmonary disease, and prurigo nodularis. This article summarises the milestones in the development of stapokibart leading to this first approval for moderate-to-severe atopic dermatitis.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"715-720"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143647633","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tafolecimab, a Third Monoclonal Antibody for PCSK9 as the Novel Lipid-Lowering Drug Around the World: A Narrative Review.","authors":"Hui-Hui Liu, Sha Li, Jian-Jun Li","doi":"10.1007/s40265-025-02167-z","DOIUrl":"10.1007/s40265-025-02167-z","url":null,"abstract":"<p><p>Tafolecimab, a fully human monoclonal antibody targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), represents a significant therapeutic advancement in the management of hypercholesterolemia and has been approved for use in the Chinese population. Elevated low-density lipoprotein cholesterol (LDL-C) is a major risk factor for atherosclerotic cardiovascular disease, and traditional treatments often fall short due to challenges such as statin intolerance. Clinical trials have demonstrated that tafolecimab can effectively reduce LDL-C levels, achieving reductions of over 60% in many patients. It also improves other lipid parameters, including lipoprotein(a) [Lp(a)], non-high-density lipoprotein cholesterol (non-HDL-C), and apolipoprotein B (ApoB). It has a favorable safety profile, primarily characterized by mild to moderate adverse events. The long-acting formulation of tafolecimab enables less frequent dosing, thereby promoting compliance. As cardiovascular diseases continue to escalate globally, tafolecimab holds promise not only for patients in China but also for broader international applications, representing a critical advancement in the evolving landscape of lipid-lowering therapies.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"627-642"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751585","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Potential of RNA Therapeutics in Treating Cardiovascular Disease.","authors":"Gayatri Mainkar, Matteo Ghiringhelli, Lior Zangi","doi":"10.1007/s40265-025-02173-1","DOIUrl":"10.1007/s40265-025-02173-1","url":null,"abstract":"<p><p>Despite significant advances in cardiology over the past few decades, cardiovascular diseases (CVDs) remain the leading cause of global mortality and morbidity. This underscores the need for novel therapeutic interventions that go beyond symptom management to address the underlying causal mechanisms of CVDs. RNA-based therapeutics represent a new class of drugs capable of regulating specific genetic and molecular pathways, positioning them as strong candidates for targeting the root causes of a wide range of diseases. Moreover, owing to the vast diversity in RNA form and function, these molecules can be utilized to induce changes at different levels of gene expression regulation, making them suitable for a broad array of medical applications, even within a single disease context. Several RNA-based therapies are currently being investigated for their potential to address various CVD pathologies. These include treatments aimed at promoting cardiac revascularization and regeneration, preventing cardiomyocyte apoptosis, reducing harmful circulating cholesterols and fats, lowering blood pressure, reversing cardiac fibrosis and remodeling, and correcting the genetic basis of inherited CVDs. In this review, we discuss the current landscape of RNA therapeutics for CVDs, with an emphasis on their classifications, modes of action, advancements in delivery strategies and considerations for their implementation, as well as CVD targets with proven therapeutic potential.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"659-676"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143771718","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficiency and Safety of Noninvasive and Intravesical Therapy for Adult Neurogenic Lower Urinary Tract Dysfunction: A Systematic Review and Network Meta-analysis of Randomized Controlled Trials.","authors":"Qiang Wang, Canyong Li, Yongfu Long, Bin Cao, Yangbo Lu, Gang Luo, Yaqiang Huang, Guiyan Huang, Hongxing Huang","doi":"10.1007/s40265-025-02171-3","DOIUrl":"10.1007/s40265-025-02171-3","url":null,"abstract":"<p><strong>Background: </strong>Neurogenic lower urinary tract dysfunction (NLUTD), a complex abnormality caused by multiple neurologic disorders, is a serious threat to patients' prognosis and quality of life. However, benefit and safety for various treatments are inconsistent. The aim of the current study was to investigate the available trials of adult NLUTD treatments and provide valuable insights for clinical practice.</p><p><strong>Methods: </strong>The data sources were Medline, Embase, and Cochrane databases up to 31 December 2023. A Bayesian network meta-analysis was conducted with randomized controlled trials in patients who were diagnosed with NLUTD, reporting clinical symptoms and urodynamic data. The main outcomes were incontinence episodes frequency (IEF) and maximum cystometric capacity (MCC). Secondary outcomes included frequency, maximum detrusor pressure, bladder compliance, volume of involuntary detrusor contraction, voided volume, incontinence, quality of life, and adverse events.</p><p><strong>Results: </strong>A total of 54 articles were eligible, evaluating 28 treatments and 4478 patients for efficacy and safety. Compared with the control group, the oxybutynin instillation demonstrated a mean reduction in IEF of - 2.65 episodes (95% confidence interval [CI]: - 4.64 to - 0.67), with a surface under the cumulative ranking curve (SUCRA) value of 85.8%. Botulinum toxin trigone-combined injection resulted in a reduction of -2.30 episodes (95% CI: -3.23 to - 1.44; SUCRA 84.2%). Additionally, intravesical therapies significantly increased MCC: oxybutynin instillation (mean 227.75 mL, 95% CI 147.00 to 311.42, SUCRA 99.1%) and BTX300U (mean 147.88 mL, 95% CI 100.45-190.32, SUCRA 83.2%). Botulinum toxin injection emerged as the preferred option for improving most urodynamic outcomes and quality of life. However, the incidence of adverse events associated with intravesical injections was higher compared with oral medications and other noninvasive therapies. The three types of botulinum toxins (onabotulinum toxin, abobotulinum toxin, and incobotulinum toxin) demonstrated consistent efficacy in treating both IEF and MCC. Individual studies were sequentially excluded for analysis of network stability. Most results from the alternative networks were consistent with the original analysis, although specific trials influenced certain therapy rankings.</p><p><strong>Conclusions: </strong>Overall, oxybutynin instillation and intravesical botulinum toxin injection demonstrated significant advantages in improving symptoms and urodynamic parameters. Our findings support intravesical treatment as a safe and effective option, provided that patients are fully informed about their treatment choices. Clinically, intravesical therapies, oral medications, nerve stimulation, and other treatments should be integrated into shared decision-making processes, while some options require further research to bolster the supporting evidence.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"677-693"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751568","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}