Drugs最新文献

{"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}

{"title":"Current Drug Treatment for Acute and Recurrent Pericarditis.","authors":"Aldo Bonaventura, Davide Santagata, Alessandra Vecchié, Antonio Abbate","doi":"10.1007/s40265-025-02169-x","DOIUrl":"10.1007/s40265-025-02169-x","url":null,"abstract":"<p><p>Pericarditis is the most frequent pericardial disease and presents with a relatively benign course when treated according to guideline-directed therapies at first presentation. Recurrence is the most frequent complication and may occur more frequently after a first episode, in patients with autoimmune etiology, in patients who received glucocorticoids, or after rapid (i.e., within 1 month) tapering of anti-inflammatory drugs. The therapeutic armamentarium for pericarditis includes high-dose nonsteroidal anti-inflammatory drugs (NSAIDs) that are tapered rapidly once symptoms are controlled. Colchicine is necessary to both relieve symptoms and reduce the rate of recurrences and is continued for at least 3-6 months. Low- to moderate-dose glucocorticoids are reserved for patients with a first recurrence for which NSAIDs and colchicine failed and/or who have an autoimmune disorder, with a slow tapering. Interleukin-1 blockers-anakinra, rilonacept, and goflikicept-are used as a third-line option in patients who cannot come off glucocorticoids or as second-line therapy after NSAIDs and colchicine in patients with contraindications to glucocorticoids or in those with high-risk features (i.e., multiple episodes, markedly elevated inflammatory markers, or extensive abnormalities at pericardial imaging) in whom treatment with glucocorticoids is unlikely to succeed.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"643-658"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12031812/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143676965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Tailored Biosimilar Approach: Expectations and Requirements.","authors":"Elena Guillen, Sean Barry, Nils Jost, Niklas Ekman, Verena Knippel, Johanna Kuhlmann-Gottke, Julia Maier, Martina Weise, Andrea Laslop, René Anour, Ger van Zandbergen, Nadine Kirsch-Stefan","doi":"10.1007/s40265-025-02168-y","DOIUrl":"10.1007/s40265-025-02168-y","url":null,"abstract":"<p><p>Regulatory approval of biosimilar medicines currently requires a combination of physicochemical and functional testing, pharmacokinetic data, and clinical efficacy studies (CES). In this article, we discuss the tailored biosimilar approach, which represents an evolution in regulatory thinking by moving away from the default requirement for CES in biosimilar approval. We explore how physicochemical and functional data can be predictive for clinical performance and address the limitations of CES for regulatory decision-making. We argue that, in most cases, the combination of a robust package of physicochemical and functional testing, with appropriately designed pharmacokinetic studies provides sufficient evidence to establish biosimilarity. Additionally, we provide our perspective on the requirements, expectations, and exceptions for future biosimilar approvals, outlining specific scenarios where additional clinical evidence may be necessary. These include cases where the mechanism of action is unknown or poorly characterized, when product heterogeneity cannot be adequately characterized, or where relevant safety or immunogenicity concerns arise with the reference product or biosimilar candidate. Finally, we aim to clarify the remaining concerns surrounding the tailored biosimilar approach, providing insights into the potential to streamline biosimilar development and regulatory approval.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"601-608"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12031863/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143763398","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction: From Prospective Evaluation to Practice: Model-Informed Dose Optimization in Oncology.","authors":"Bram C Agema, Birgit C P Koch, Ron H J Mathijssen, Stijn L W Koolen","doi":"10.1007/s40265-025-02174-0","DOIUrl":"10.1007/s40265-025-02174-0","url":null,"abstract":"","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"721-723"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12031969/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143662859","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cosibelimab: First Approval.","authors":"Arnold Lee","doi":"10.1007/s40265-025-02164-2","DOIUrl":"10.1007/s40265-025-02164-2","url":null,"abstract":"<p><p>Cosibelimab (UNLOXCYT; cosibelimab-ipdl) is an anti-PD-L1 antibody developed by Checkpoint Therapeutics for the treatment of advanced cancers, including metastatic or locally advanced cutaneous squamous cell carcinoma (mCSCC or laCSCC). Cosibelimab is the first approved anti-PD-L1 therapy in the USA for the treatment of adults with mCSCC or laCSCC who are not candidates for curative surgery or curative radiation. In contrast to other anti-PD-L1 antibodies, cosibelimab can induce antibody-dependent cell mediated cytotoxicity as it includes a functional F_c domain. This article summarizes the milestones in the development of cosibelimab leading to this first approval for this indication.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"695-698"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751565","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":"Zanidatamab: First Approval.","authors":"Susan J Keam","doi":"10.1007/s40265-025-02163-3","DOIUrl":"10.1007/s40265-025-02163-3","url":null,"abstract":"<p><p>Zanidatamab (ZIIHERA^®; zanidatamab- hrii), a bi-specific antibody targeting two non-overlapping epitopes of the human epidermal growth factor receptor 2 (HER2) protein, is being developed by Jazz Pharmaceuticals and BeiGene Ltd under license agreements from Zymeworks Inc., the developer of the molecule, for the treatment of HER2-expressing solid tumours. This article summarizes the milestones in the development of zanidatamab leading to this first accelerated approval for use in adults with previously treated, unresectable or metastatic HER2+ (IHC3+) biliary tract cancer (BTC), as detected by an FDA-approved test.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"707-714"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143662893","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":"Classic Psychedelics for the Treatment of Depression: Potential Benefits and Challenges.","authors":"Sharmin Ghaznavi, Sarah G Richter","doi":"10.1007/s40265-025-02172-2","DOIUrl":"10.1007/s40265-025-02172-2","url":null,"abstract":"<p><p>There has been a recent resurgence in research on psychedelics as therapeutic agents for psychiatric conditions. This leading article outlines the studies to date of classic psychedelic treatments for treatment-resistant depression and major depression, including psilocybin, ayahuasca, dimethyltryptamine (DMT), and O-methyl-bufotenine (5-Me-O DMT). We discuss the potential of expanding treatment options for depression based on the data available, as well as the difficulties and limitations of research on psychedelics that make assessing that potential more challenging.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"609-626"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143699918","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":"Iparomlimab and Tuvonralimab: First Approval.","authors":"Susan J Keam","doi":"10.1007/s40265-025-02160-6","DOIUrl":"10.1007/s40265-025-02160-6","url":null,"abstract":"<p><p>Iparomlimab and tuvonralimab (^®) is a bifunctional combination of anti-programmed death receptor-1 (PD-1)/anti-cytotoxic T lymphocyte-associated protein-4 (CTLA-4) monoclonal antibodies (mAbs) being developed by Qilu Pharmaceutical Co., Ltd for the treatment of advanced, solid, malignant tumours. In September 2024, iparomlimab and tuvonralimab was granted conditional approval (based on surrogate endpoints) for the treatment of patients with recurrent or metastatic cervical cancer who have failed previous platinum-based chemotherapy. This article summarizes the milestones in the development of iparomlimab and tuvonralimab leading to this first approval for the treatment of patients with recurrent or metastatic cervical cancer who have failed previous platinum-based chemotherapy.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"699-706"},"PeriodicalIF":13.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751583","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}