Drugs最新文献

{"title":"Acoramidis: First Approval.","authors":"Arnold Lee","doi":"10.1007/s40265-025-02159-z","DOIUrl":"10.1007/s40265-025-02159-z","url":null,"abstract":"<p><p>Disassociation of transthyretin (TTR) has been identified as a key step in the pathology of TTR amyloid cardiomyopathy, which is characterised by the presence of amyloid fibrils in cardiac tissue. Acoramidis (ATTRUBY™) is a small molecule TTR stabiliser being developed by BridgeBio Pharma, Inc. and is the first drug to demonstrate near-complete (> 90%) stabilisation of TTR. This article summarizes the milestones in the development of acoramidis leading to its first approval in the USA for the treatment of the cardiomyopathy of wild-type or variant TTR-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization. In the EU, a positive opinion has been adopted for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"833-837"},"PeriodicalIF":13.0,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143779430","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":"Renal Safety Assessment of Lipid-Lowering Drugs: Between Old Certainties and New Questions.","authors":"Daniele Tramontano, Simone Bini, Carlo Maiorca, Alessia Di Costanzo, Martina Carosi, Jacopo Castellese, Ina Arizaj, Daniela Commodari, Stella Covino, Giorgia Sansone, Ilenia Minicocci, Marcello Arca, Laura D'Erasmo","doi":"10.1007/s40265-025-02158-0","DOIUrl":"10.1007/s40265-025-02158-0","url":null,"abstract":"<p><p>Cardiovascular disease (CVD) is the leading cause of morbidity and mortality in patients with chronic kidney disease (CKD). Quantitative and qualitative changes in plasma lipoprotein profiles are frequently associated with CKD and represent a significant risk factor for CVD in patients with CKD. Guidelines from the European Society of Cardiology and the European Atherosclerosis Society classify CKD as a condition with high or very high cardiovascular risk and set specific low-density lipoprotein cholesterol targets. Conventional lipid-lowering therapies (LLTs), such as statins, ezetimibe, and fibrates, can control CKD-associated dyslipidemia and, to some extent, prevent major atherosclerotic events in patients with CKD, but their use in clinical practice presents challenges because of the potential renal safety concerns. In recent years, novel therapies with the ability to lower both low-density lipoprotein cholesterol and triglycerides have been introduced to the market (e.g., proprotein convertase subtilisin/kexin type 9 inhibitors, bempedoic acid, lomitapide, volanesorsen) to improve our ability to control lipid abnormalities. However, their impact on kidney functionality has not been fully elucidated. The aim of this review was to examine the renal safety profiles of various LLTs, with special reference to novel medications, and to highlight important considerations and guidance for the use of these medications in overt CKD or in patients with some degree of renal function impairment. We underscore the lack of a comprehensive understanding of kidney safety, particularly for novel LLT therapies, and strongly emphasize the importance of future dedicated research to fully assess the safety and efficacy of these agents in patients with kidney abnormalities.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"755-775"},"PeriodicalIF":13.0,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12098426/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143662888","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: Current and Emerging Therapies for Lysosomal Storage Disorders.","authors":"Diego Agustín Abelleyra Lastoria, Sophie Keynes, Derralynn Hughes","doi":"10.1007/s40265-025-02175-z","DOIUrl":"10.1007/s40265-025-02175-z","url":null,"abstract":"","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"853"},"PeriodicalIF":13.0,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143987606","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":"Suzetrigine: First Approval.","authors":"Susan J Keam","doi":"10.1007/s40265-025-02178-w","DOIUrl":"10.1007/s40265-025-02178-w","url":null,"abstract":"<p><p>Suzetrigine (JOURNAVX™), an oral, non-opioid analgesic that is highly selective for the voltage-gated sodium channel NaV1.8 relative to other NaV channels, is being developed by Vertex Pharmaceuticals Inc. for the management of acute and neuropathic pain. NaV1.8 is expressed in peripheral pain-sensing neurons but is not expressed in the brain. Suzetrigine prevents transmission of pain signals by blocking NaV1.8 channels located along peripheral pain-sensing neurons, inhibiting the normal action potential. On 30 January 2025, suzetrigine was approved for the treatment of moderate to severe acute pain in adults in the USA. Suzetrigine is the first NaV inhibitor to be approved in this new therapeutic class of non-opioid analgesics. This article summarizes the milestones in the development of suzetrigine leading to this first approval for the treatment of moderate to severe acute pain.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"845-851"},"PeriodicalIF":13.0,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143989757","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":"Garadacimab: First Approval.","authors":"Simon Fung","doi":"10.1007/s40265-025-02180-2","DOIUrl":"10.1007/s40265-025-02180-2","url":null,"abstract":"<p><p>Garadacimab (Andembry^®) is a fully human IgG4/lambda recombinant monoclonal anti-activated Factor XII antibody being developed by CSL Behring for the prevention of hereditary angioedema attacks. In January 2025, garadacimab received its first approval in Australia and the UK for prevention of hereditary angioedema attacks in adult and adolescent patients aged ≥ 12 years. Additionally, in February 2025, garadacimab was approved for the same indication in the EU, Japan and Switzerland. In the USA and Canada, regulatory review of garadacimab is currently underway. This article summarizes the milestones in the development of garadacimab leading to this first approval for prevention of recurrent hereditary angioedema attacks in adult and adolescent patients aged ≥ 12 years.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"827-832"},"PeriodicalIF":13.0,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143977141","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":"Lipid-Lowering Therapy and Cardiovascular Prevention in Elderly.","authors":"Francesco Baratta, Federica Moscucci, Ilaria Lospinuso, Nicholas Cocomello, Alessandra Colantoni, Alessia Di Costanzo, Daniele Tramontano, Laura D'Erasmo, Daniele Pastori, Evaristo Ettorre, Maria Del Ben, Marcello Arca, Giovambattista Desideri","doi":"10.1007/s40265-025-02182-0","DOIUrl":"10.1007/s40265-025-02182-0","url":null,"abstract":"<p><p>The global population aged 80 years and older will reach approximately half a billion in the coming years, and cardiovascular prevention in this group of patients will become a global health challenge. In the era of evidence-based medicine, the use of lipid-lowering therapies (LLTs) in the elderly, particularly in primary and secondary cardiovascular prevention, remains an area of active research. Although there is broad consensus on the use of LLTs in the elderly to prevent recurrent cardiovascular events in secondary prevention, there is considerable debate about their use in primary prevention. Many efforts have been made to improve cardiovascular risk stratification in patients over 75 years of age in primary prevention. In recent years, some specific risk scores have been developed, including the Systematic Coronary Risk Evaluation 2 for Older Persons (SCORE2-OP). While there are very few specific warnings to consider for LLTs in the elderly, an important challenge in this patient population is to identify the turning point at which the disutility risk outweighs the potential benefits. However, despite the widespread recognition of the importance of this issue, there is a lack of guidance on how to identify patients who should be withdrawn from therapy. The aim of this narrative review is to examine the current state of knowledge regarding the indications for LLT in elderly patients, identify outstanding issues, and discuss future developments.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"801-812"},"PeriodicalIF":13.0,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12098413/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143984823","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":"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":"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":"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}