DrugsPub Date : 2025-06-01Epub Date: 2025-05-08DOI: 10.1007/s40265-025-02182-0
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
{"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recognition of the importance of this issue, there is a lack of guidance on how to identify patients who should be withdrawn from therapy.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}
DrugsPub Date : 2025-05-27DOI: 10.1007/s40265-025-02190-0
Sheridan M Hoy
{"title":"Mirdametinib: First Approval.","authors":"Sheridan M Hoy","doi":"10.1007/s40265-025-02190-0","DOIUrl":"https://doi.org/10.1007/s40265-025-02190-0","url":null,"abstract":"<p><p>Mirdametinib (GOMEKLI<sup>TM</sup>) is an oral small molecule inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2) developed by SpringWorks Therapeutics for the treatment of neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PN). It is the first therapy to be approved for the treatment of both adults and children with NF1-associated PN in the USA, where it is indicated for use in adult and paediatric patients 2 years of age and older with NF1 who have symptomatic PN not amenable to complete resection. A marketing authorisation application for mirdametinib in NF1-PN is currently under review in the EU. Clinical studies of mirdametinib for the treatment of paediatric low-grade glioma are ongoing. This article summarizes the milestones in the development of mirdametinib leading to this first approval.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144149786","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}
DrugsPub Date : 2025-05-26DOI: 10.1007/s40265-025-02191-z
Arnold Lee
{"title":"Vimseltinib: First Approval.","authors":"Arnold Lee","doi":"10.1007/s40265-025-02191-z","DOIUrl":"https://doi.org/10.1007/s40265-025-02191-z","url":null,"abstract":"<p><p>Vimseltinib (ROMVIMZA™) is an orally administered kinase inhibitor that targets colony-stimulating factor 1 receptor (CSF1R), which is being developed by Deciphera Pharmaceuticals. As CSF1R activity has been identified as a contributing factor for tenosynovial giant cell tumour (TGCT), treatments targeting CSF1R have been investigated. Vimseltinib received its first approval in February 2025 in the USA for the treatment of TGCT and is under development for the treatment of chronic graft versus host disease. This article summarizes the milestones in the development of vimseltinib leading to this first approval for the treatment of adult patients with symptomatic TGCT for which surgical resection will potentially cause worsening functional limitation or severe morbidity.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144141714","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}
DrugsPub Date : 2025-05-24DOI: 10.1007/s40265-025-02195-9
Pascale C S Rietveld, Niels A D Guchelaar, Sebastiaan D T Sassen, Birgit C P Koch, Ron H J Mathijssen, Stijn L W Koolen
{"title":"A Clinical Pharmacological Perspective on Intraperitoneal Chemotherapy.","authors":"Pascale C S Rietveld, Niels A D Guchelaar, Sebastiaan D T Sassen, Birgit C P Koch, Ron H J Mathijssen, Stijn L W Koolen","doi":"10.1007/s40265-025-02195-9","DOIUrl":"https://doi.org/10.1007/s40265-025-02195-9","url":null,"abstract":"<p><p>Peritoneal metastases (PM), frequently observed in malignancies such as ovarian, colorectal, pancreatic, and gastric cancers, present a significant therapeutic challenge due to poor prognosis and limited effectiveness to systemic chemotherapy. The peritoneal-plasma barrier reduces effective drug transfer from plasma to the peritoneal cavity, reducing cytotoxic effects on PM. Intraperitoneal (IP) chemotherapy offers a locoregional approach, enabling high local drug concentrations that can enhance therapeutic efficacy while limiting systemic toxicity. The three major methods for IP administration-hyperthermic intraperitoneal chemotherapy (HIPEC), pressurized intraperitoneal aerosol chemotherapy (PIPAC), and catheter-based IP (CBIP) chemotherapy-each provide unique pharmacokinetic (PK) advantages for PM treatment. This review provides a comprehensive update on the pharmacological rationale of IP chemotherapy, focusing on drug characteristics that support extended IP retention and effective tumor targeting. The effects of administration variables are discussed, highlighting their role in optimizing IP drug exposure. Additionally, recent PK data on commonly used drugs in IP therapy, including platinum-based agents, taxanes, and novel nanoparticle formulations, will be evaluated. While PK rationale supports the administration of IP chemotherapy, further efficacy results from ongoing clinical trials are still awaited. Innovations in nanoparticle-based formulations and controlled-release systems offer substantial potential for improving both drug retention and targeted delivery, enhancing treatment precision and minimizing systemic toxicity. Continued exploration in these areas, along with optimization of IP administration protocols, is vital for advancing patient outcomes, refining therapeutic strategies, and maximizing the benefits of IP chemotherapy in clinical practice.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144136087","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}
DrugsPub Date : 2025-05-22DOI: 10.1007/s40265-025-02196-8
Wenqin Yin, Dan Luo, Haiqi Mi, Zhimin Ren, Lianling Li, Zhidong Fan, Jingyan Lin
{"title":"Rebound Pain After Peripheral Nerve Block: A Review.","authors":"Wenqin Yin, Dan Luo, Haiqi Mi, Zhimin Ren, Lianling Li, Zhidong Fan, Jingyan Lin","doi":"10.1007/s40265-025-02196-8","DOIUrl":"https://doi.org/10.1007/s40265-025-02196-8","url":null,"abstract":"<p><p>Peripheral nerve block (PNB) is now a commonly used analgesic treatment in clinical anesthesia owing to ongoing advancements in ultrasound imaging technology, which provides clear images of the nerves. Multimodal analgesia based on peripheral nerve blocks is replacing the conventional opioid-based analgesic strategy. However, after the nerve block effect is removed, some patients experience rebound pain (RP), which exacerbates suffering. The benefits of PNB as a perioperative analgesic may be completely negated if RP is discovered and treated too late, even if it can be promptly managed with analgesics. The definitions, clinical signs, risk factors, pathophysiology, and prevention of RP after PNBs are reviewed in this article. At present, the mechanism of RP after PNB is still unclear, but different types of RP may share similar mechanisms in the pain transmission pathway. In this review, we have determined the characteristics of RP and tried to identify the high-risk factors. Among the many means of preventing and reducing the incidence of RP identified, a single block with adjuvant dexamethasone is a reliable regimen, but for the time being, the application of a catheter would be a more reliable method of reducing RP. This review also provides recommendations for the proper use of nerve blocks as supplemental analgesics under clinical anesthesia.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144119244","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}
DrugsPub Date : 2025-05-20DOI: 10.1007/s40265-025-02188-8
Dave Singh, Andrew Higham, Alexander G Mathioudakis, Augusta Beech
{"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}
DrugsPub Date : 2025-05-19DOI: 10.1007/s40265-025-02193-x
Simon Fung
{"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<sup>®</sup>) 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}
DrugsPub Date : 2025-05-12DOI: 10.1007/s40265-025-02181-1
Joseph M Garland, Haim Mayan, Rami Kantor
{"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}
DrugsPub Date : 2025-05-11DOI: 10.1007/s40265-025-02192-y
Rubén Queiro, José Antonio Pinto-Tasende, Carlos Montilla-Morales
{"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<sup>®</sup> 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<sup>®</sup>, 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}