DrugsPub Date : 2025-06-01Epub Date: 2025-03-19DOI: 10.1007/s40265-025-02158-0
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
{"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}
DrugsPub Date : 2025-06-01Epub Date: 2025-04-22DOI: 10.1007/s40265-025-02180-2
Simon Fung
{"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<sup>®</sup>) 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}
DrugsPub Date : 2025-06-01Epub Date: 2025-04-12DOI: 10.1007/s40265-025-02175-z
Diego Agustín Abelleyra Lastoria, Sophie Keynes, Derralynn Hughes
{"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}
DrugsPub Date : 2025-06-01Epub Date: 2025-05-05DOI: 10.1007/s40265-025-02178-w
Susan J Keam
{"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}
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 : 2025-05-30DOI: 10.1007/s40265-025-02189-7
Sebastiano Mercadante
{"title":"QT Prolongation and Risk of Death with the Use of Methadone for Chronic Cancer and Noncancer Pain: Myths or Reality?","authors":"Sebastiano Mercadante","doi":"10.1007/s40265-025-02189-7","DOIUrl":"https://doi.org/10.1007/s40265-025-02189-7","url":null,"abstract":"<p><p>The use of methadone has been associated with corrected QT (QTc) prolongation. However, conclusions about the dangers of methadone are limited by its dual use for narcotic abuse deterrence. All these observations can deter physicians from prescribing methadone in patients with chronic pain, particularly those with cancer pain. The aim of this review was to evaluate the existing data regarding the relevance of QT changes, the risk factors for QTc prolongation, as well as the risk for cardiac events and mortality, in patients receiving methadone for chronic pain. In total, 15 studies were evaluated. They differed greatly in design (prospective, retrospective), levels of QTc ranges, number of patients included, and methadone doses. Data suggest that the relevance of QTc prolongation induced by methadone seems to be minimal, also considering the range of dosages commonly used in both noncancer and cancer pain. Some risk factors for QTc prolongation have been identified. Information regarding a prior history or prolonged QTc interval, a family history of a prolonged QTc interval, or a family history of sudden, unexplained death is crucial. In this population and at clinical methadone dosages, serious cardiac events have not been described. Low doses of methadone commonly used in most chronic patients may not require QTc monitoring. When a patient is receiving various medications that could potentially prolong QTc, clinicians may consider obtaining a manually measured QTc. Early discussions with patients regarding goals of care, risks, and benefits will help avoid QTc measurements at regular intervals.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0,"publicationDate":"2025-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144186766","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 : 2025-05-27DOI: 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}