Drugs最新文献

{"title":"Antifungal Treatment Regimens as Primary Therapy for Invasive Aspergillosis: A Systematic Review and Network Meta-analysis.","authors":"Qiaoling Gu, Yechao Chen, Peng Ding, Juan He, Haixia Zhang, Dayu Chen","doi":"10.1007/s40265-026-02316-y","DOIUrl":"10.1007/s40265-026-02316-y","url":null,"abstract":"<p><strong>Background: </strong>Invasive aspergillosis (IA) is an invasive fungal disease associated with high mortality. Triazoles are the primary therapy for IA, while amphotericin B, echinocandins, and antifungal combined therapy are commonly considered as alternative treatment options although their efficacy and safety remain subjects of debate. We employed Bayesian network meta-analysis (NMA) to synthesize evidence and evaluate the efficacy and safety of different primary antifungal treatment regimens for IA.</p><p><strong>Methods: </strong>A literature search was done in PubMed, EMBASE, Cochrane, and Web of Science databases for clinical studies on primary treatment for patients with IA published before 28 March 2024. We included randomized controlled trials (RCTs) and cohort studies involving patients with proven or probable IA who underwent primary treatment. The primary treatment regimens comprised different types or dosages of monotherapy or combination therapy involving triazoles, echinocandins, and amphotericin B. The primary outcome was cumulative all-cause mortality. Secondary outcomes included all-cause mortality at day 42, day 84, or end of treatment (EOT); the overall response at day 42, day 84, or EOT; invasive aspergillosis-associated mortality on day 42, day 84, and EOT; the incidence of adverse events (AEs), serious adverse events (SAEs), and treatment-related AEs; and treatment discontinuation due to AEs.</p><p><strong>Results: </strong>The primary network meta-analysis for cumulative all-cause mortality (5 RCTs, 6 regimens, n = 1293) revealed no statistically significant differences among treatment regimens compared with voriconazole (VOR; surface under the cumulative ranking curve [SUCRA] 56.3%), including VOR combined with anidulafungin (ANI; risk ratio [RR] 0.74, 95% credible interval [CrI] 0.43-1.28; SUCRA 84.3%; moderate confidence), isavuconazole (ISA; RR 0.79, 95% CrI 0.45-1.40; 79.4%; moderate confidence), and posaconazole (POS; RR 1.20, 95% CrI 0.72-2.02; 34.6%; moderate confidence). Sensitivity analyses confirmed the robustness of these findings. Regarding safety, no significant differences were observed in SAEs for ISA (RR 0.91, 95% CrI 0.75-1.11; 85.7%; moderate confidence), POS (RR 1.03, 95% CrI 0.87-1.23; 40.8%; low confidence), and VOR combined with ANI (RR 1.09, 95% CrI 0.88-1.36; 20.8%; moderate confidence) compared with VOR (52.6%). Integrated efficacy-safety analysis suggested a favorable risk-benefit profile for isavuconazole.</p><p><strong>Conclusions: </strong>ISA, POS, and VOR are the preferred antifungal agents for primary treatment of IA, with ISA demonstrating a higher likelihood of an optimal efficacy-safety balance. The combination of VOR and ANI may be considered for a subset of severe cases.</p><p><strong>Registration: </strong>PROSPERO identifier number CRD42024561215.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"927-941"},"PeriodicalIF":14.7,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147765809","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":"Targeting Inflammation in Bronchiectasis.","authors":"Micheál Mac Aogáin, Amy Gilmour, James D Chalmers, Sanjay H Chotirmall","doi":"10.1007/s40265-026-02314-0","DOIUrl":"10.1007/s40265-026-02314-0","url":null,"abstract":"<p><p>Bronchiectasis is defined by chronic infection, dysregulated inflammation and impaired mucociliary clearance underpinning progressive structural lung injury. While airway infection remains a clinical hallmark, numerous studies demonstrate that excessive neutrophil-dominated inflammation is a key determinant of disease severity, exacerbation risk and quality of life. Recent developments have transformed our understanding of inflammatory drivers uncovering distinct inflammatory endotypes defined by dominant microbial species, pattern-recognition receptor activation, inflammasome signalling, Th17-associated cytokine networks and failures of mucosal immunity. The emerging roles of viral-bacterial interactions, fungi, pathobionts and the broader microbiome challenge the conventional infection-only paradigm and highlight gaps in current therapeutic strategies. Such developments underpin the rationale behind anti-inflammatory strategies in bronchiectasis, ranging from suppression of neutrophil-driven injury through direct neutrophil elastase or upstream dipeptidyl peptidase-1 (DPP-1) inhibition, to immunomodulatory macrolides, toward therapies aimed at recalibrating epithelial and mucosal homeostasis. While several antibacterial and anti-infective trials have produced mixed results, this is likely to reflect unresolved heterogeneity in microbiome composition and host immune signalling. In contrast, emerging anti-inflammatory strategies show strong positive signals, reinforcing the need for better endotyping and biomarker-guided patient selection. Here we synthesize recent mechanistic and clinical insights to propose a more integrated framework for understanding and ultimately targeting airway inflammation in bronchiectasis.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"797-812"},"PeriodicalIF":14.7,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147638352","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":"Depemokimab: First Approval.","authors":"Arnold Lee","doi":"10.1007/s40265-026-02306-0","DOIUrl":"10.1007/s40265-026-02306-0","url":null,"abstract":"<p><p>Depemokimab (depemokimab-ulaa; EXDENSUR) is an anti-IL-5 antibody being developed by GSK for the treatment of asthma, chronic rhinosinusitis with nasal polyps (CRSwNP), chronic obstructive pulmonary disease, eosinophilic granulomatosis with polyangiitis and hypereosinophilic syndrome. Add-on treatment with depemokimab reduced asthma-related exacerbations in patients with severe asthma with an eosinophilic phenotype, in addition to reducing the severity of nasal polyps and nasal obstruction in patients with CRSwNP. This article summarizes the milestones in the development of depemokimab leading to this first approval in the UK as an add-on maintenance treatment of asthma in adult and adolescent patients aged ≥ 12 years with type 2 inflammation characterised by an eosinophilic phenotype who are inadequately controlled on maximum moderate-dose or high-dose inhaled corticosteroids plus another asthma controller; and as add-on therapy with intranasal corticosteroids for the treatment of adults with severe CRSwNP for whom therapy with systemic corticosteroids and/or surgery do not provide adequate control.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"943-948"},"PeriodicalIF":14.7,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147510028","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":"Zoliflodacin: First Approval.","authors":"Aisling McGuigan","doi":"10.1007/s40265-026-02308-y","DOIUrl":"10.1007/s40265-026-02308-y","url":null,"abstract":"<p><p>Zoliflodacin (NUZOLVENCE^®), a first-in-class, single-dose, oral spiropyrimidinetrione bacterial type II topoisomerase inhibitor, has been developed by Innoviva Specialty Therapeutics in collaboration with the Global Antibiotic Research and Development Partnership (GARDP) and the U.S. National Institute of Allergy and Infectious Diseases (NIAID) for the treatment of uncomplicated urogenital gonorrhea (uUGC). In December 2025, zoliflodacin was approved by the US FDA for the treatment of uUGC due to Neisseria gonorrhoeae in adults and pediatric patients 12 years of age and older weighing at least 35 kg. This article summarizes the milestones in the development of zoliflodacin leading to this first approval for the treatment of uUGC.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"963-970"},"PeriodicalIF":14.7,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147722227","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":"Sequential Use of Trastuzumab Deruxtecan and Sacituzumab Govitecan in Patients with Breast Cancer: A Pharmacological Approach to Support the Clinical Rationale.","authors":"Marzia Del Re, Elvira Inglese, Emilio Bria, Erik J Blok, Ron H J Mathijssen, Giuseppe Curigliano, Romano Danesi","doi":"10.1007/s40265-026-02289-y","DOIUrl":"10.1007/s40265-026-02289-y","url":null,"abstract":"<p><p>Antibody-drug conjugates (ADCs) represent a major advance in breast cancer therapy, with trastuzumab deruxtecan and sacituzumab govitecan emerging as leading agents targeting distinct tumor antigens and employing different linker-payload designs. Trastuzumab deruxtecan is a second-generation HER2-directed ADC composed of trastuzumab linked via a cleavable tetrapeptide linker to the camptothecin derivative exatecan, a highly potent topoisomerase I inhibitor. Its high drug-to-antibody ratio (DAR 8:1), membrane-permeable payload, and efficient lysosomal release confer strong antitumor activity, including a robust bystander effect in HER2-low tumors. By contrast, sacituzumab govitecan is a TROP-2-targeted ADC conjugated through a hydrolysable CL2A linker to SN-38, the active metabolite of irinotecan. Sacituzumab govitecan features a high DAR (7.6:1) and allows extracellular as well as intracellular release of SN-38, enhancing bystander killing in tumors with heterogeneous TROP-2 expression, while maintaining a favorable toxicity profile due to the lower intrinsic potency of SN-38 relative to exatecan. Since both these ADCs are conjugated to topoisomerase I inhibitors, concerns about potential cross-resistance regarding their sequencing in clinical practice are being raised, and currently there is a lack of predictive biomarkers providing a rational basis for their sequential administration. Resistance mechanisms exhibit significant heterogeneity: resistance to trastuzumab deruxtecan has been associated with mutations in TOPO I and SLX4, impaired lysosomal function, and efflux via ABC transporters. In contrast, resistance to sacituzumab govitecan is linked to overexpression of multidrug resistance proteins, particularly BCRP-mediated efflux. Clinically, trastuzumab deruxtecan has shown substantial efficacy in both HER2-positive and HER2-low breast cancer, whereas sacituzumab govitecan has demonstrated efficacy across triple-negative and hormone receptor-positive/HER2-negative breast cancers. Collectively, these agents highlight how variations in target antigen, linker chemistry, and payload potency impact ADC activity, therapeutic index, and potential strategies for sequential treatment in advanced breast cancer.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"871-885"},"PeriodicalIF":14.7,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13179276/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147608379","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":"Large Vessel Vasculitis: Recent Advances in Pathophysiology and Targeted Therapies.","authors":"Myriam Reisch, Jens Thiel, Philipp Bosch","doi":"10.1007/s40265-026-02323-z","DOIUrl":"10.1007/s40265-026-02323-z","url":null,"abstract":"<p><p>Large vessel vasculitides (LVV), including giant cell arteritis (GCA) and Takayasu arteritis (TAK), share common features such as inflammation of large sized arteries but differ in several key aspects, including age of onset and pathogenic mechanism. This narrative review gives an update of recent insights into pathogenesis of GCA and TAK, and discusses emerging targeted therapies based on these insights. It highlights omics-based signatures, ULK3 and SLAMF7 in GCA, EGR1 in TAK, alongside genetic and somatic risk factors such as clonal haematopoiesis (DNMT3A/TET2) linked to relapse and ischaemic vision loss in GCA, and the IL6R-p.Asp358Ala variant as a predictor of reduced interleukin (IL)-6 receptor blockade response. Common mechanisms include CD4⁺ T-cell, monocyte/macrophage, and B-cell infiltration with activation of IL-6, JAK/STAT/interferon, and IL-17 pathways. Giant cell arteritis is characterised by GM-CSF-driven macrophages and disrupted programmed cell death (PD)-1/PD-L1 checkpoint regulation, while TAK shows dominance of CD8⁺ T cells and tumour necrosis factor (TNF)-α signalling. Interleukin-6 receptor inhibitors (e.g., tocilizumab) show robust efficacy in GCA but with notable non-responders; the JAK inhibitor upadacitinib demonstrated efficacy in a Phase III study, whereas IL-17 blockade (secukinumab) yielded inconsistent results. In TAK, TNF inhibitors and tocilizumab are comparably effective; early data suggest Janus kinases (JAK) inhibitors promote remission, imaging improvement, and glucocorticoid sparing. Mavrilimumab (GM-CSF receptor blockade) is promising in GCA. Recent studies have increasingly focused on short-term glucocorticoid therapy in combination with biologic agents. Advances in biomarker research, including investigation of the IL-6 receptor and IL-17A gene polymorphisms, may enable more targeted therapeutic strategies.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"909-925"},"PeriodicalIF":14.7,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13179178/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147812330","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":"Tradipitant: First Approval.","authors":"Arnold Lee","doi":"10.1007/s40265-026-02313-1","DOIUrl":"10.1007/s40265-026-02313-1","url":null,"abstract":"<p><p>Tradipitant (NEREUS™) is a small molecule neurokinin-1 (NK-1) receptor antagonist being developed by Vanda Pharmaceuticals. NK-1 receptor activation and substance P release in the central nervous system leads to nausea and vomiting symptoms associated with motion sickness. Tradipitant received its first approval in the USA in December 2025 for the prevention of vomiting induced by motion in adults, and is also being developed for the treatment of gastroparesis and prevention of vomiting induced by GLP-1 receptor agonists. In phase III trials, tradipitant reduced the incidence of vomiting associated with motion sickness. This article summarizes the milestones in the development of tradipitant leading to this first approval for the prevention of vomiting induced by motion in adults.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"957-961"},"PeriodicalIF":14.7,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147627361","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":"Sevabertinib: First Approval.","authors":"Susan J Keam","doi":"10.1007/s40265-026-02299-w","DOIUrl":"10.1007/s40265-026-02299-w","url":null,"abstract":"<p><p>Sevabertinib (HYRNUO^®), an oral, reversible, small molecule tyrosine kinase inhibitor (TKI) of human epidermal growth factor receptor 2 (HER2) that also shows activity against epidermal growth factor receptor (EGFR), is being developed by Bayer for the treatment of solid tumours with mutations in HER2 or EGFR genes. Sevabertinib is derived from Bayer's strategic research alliance with the Broad Institute of MIT and Harvard. In November 2025, sevabertinib was approved under accelerated approval in the USA for use in adults with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumours have HER2 (ERBB2) TK domain (TKD) activating mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy. This article summarizes the milestones in the development of sevabertinib leading to this first approval for the treatment of locally advanced or metastatic NSCLC with HER2 (ERBB2) activating mutations.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"759-764"},"PeriodicalIF":14.7,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147510228","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":"Efficacy and Safety of Anti-nerve Growth Factor Monoclonal Antibodies in Managing Chronic Musculoskeletal Pain: A Systematic Review with Network Meta-analysis.","authors":"Ahmed Abouelella, Qasi Najah, Richard O Day, Lars Arendt-Nielsen, Maria A Lopez-Olivo, Matthew K Bagg, K T Matthew Seah, Mohammad Alhasan, Ahmed Hamoudah, Ola Abuzied, Mansoor Al-Tamimi, Ayman Ibrahim, Eman Basheer, Reham Mahgoub, Muhammed Elhadi","doi":"10.1007/s40265-026-02304-2","DOIUrl":"10.1007/s40265-026-02304-2","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Anti-nerve growth factor (NGF) monoclonal antibodies (mAbs) have emerged as a promising new class of analgesics, offering potential benefits in managing particular painful musculoskeletal (MSK) conditions. However, their long-term safety remains uncertain, leading to regulatory non-approval of these agents. This study aims to evaluate the efficacy and safety of individual anti-NGF mAbs compared to other analgesics when treating chronic MSK pain.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Our literature search included PubMed, Scopus, Embase, Web of Science, Cochrane Library, and ClinicalTrials.gov through April 25th, 2025. Articles eligible for inclusion were randomized controlled trials (RCTs) comparing one of the human anti-NGF mAbs to other interventions in adults with chronic MSK pain. Primary outcomes evaluated were changes from baseline in pain, physical function, and patient global assessment (PGA) scores, as well as risks of adjudicated arthropathies (AAs) and abnormal peripheral sensation (APS). We used the Cochrane Risk of Bias 2 (RoB-2) tool to assess risk of bias. Pairwise and network meta-analyses were performed using random-effects models. Treatments were ranked using the cumulative ranking curve (SUCRA), and a multi-criteria decision analysis with Technique for Order Preference by Similarity to Ideal Solution (TOPSIS) was applied to integrate all efficacy and safety outcomes. Statistical analyses were conducted in R (v4.3.1) using the meta, netmeta, gemtc, and Multi-Criteria Decision Aiding (MCDA) packages.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 29 studies, involving 27,747 patients with osteoarthritis or chronic low back pain, were included in this analysis. Compared to placebo, fasinumab showed the highest improvements in pain (standardized mean difference [SMD] - 0.40, 95% CI [- 0.52, - 0.29], p &lt; 0.001) and physical function (SMD - 0.42, 95% CI [- 0.53, - 0.31], p &lt; 0.001), followed by tanezumab (pain: SMD - 0.36, 95% CI [- 0.44, - 0.28], p &lt; 0.001; function: SMD - 0.39, 95% CI [- 0.47, - 0.31], p &lt; 0.001). For safety, both fasinumab and tanezumab demonstrated a significant risk for AAs (risk ratio [RR] 4.7, 95% CI [3.61, 6.13], p &lt; 0.001; and RR 3.84, 95% CI [2.07, 7.14], p &lt; 0.001, respectively) and APS (RR 1.99, 95% CI [1.49, 2.65], p &lt; 0.001; and RR 2.46, 95% CI [1.93, 3.14], p &lt; 0.001, respectively) relative to placebo. While fulranumab was less effective (pain: SMD - 0.25, 95% CI [- 0.42, - 0.07], p &lt; 0.01; function: SMD - 0.25, 95% CI [- 0.43, - 0.07], p &lt; 0.01), it showed better overall safety against placebo relative to both agents, demonstrating a significant risk only for APS events (RR 1.78, 95% CI [1.09, 2.92], p &lt; 0.05).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Anti-NGF mAbs, particularly fasinumab and tanezumab, are associated with the greatest levels of pain relief and functional improvement over placebo within this analysis. However, these benefits are counterbalanced by significant risks of","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"719-736"},"PeriodicalIF":14.7,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147510055","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":"Plozasiran: First Approval.","authors":"Yahiya Y Syed","doi":"10.1007/s40265-026-02293-2","DOIUrl":"10.1007/s40265-026-02293-2","url":null,"abstract":"<p><p>Plozasiran (Redemplo^®) is a first-in-class GalNAc-conjugated small interfering RNA (siRNA) designed to reduce hepatic apolipoprotein C-III (ApoC3) production. It is being developed by Arrowhead Pharmaceuticals for the treatment of familial chylomicronemia syndrome (FCS), severe hypertriglyceridaemia and mixed hyperlipidaemia. Plozasiran degrades ApoC3 mRNA via RNA interference, reducing hepatic and serum ApoC3 levels and thereby enhancing catabolism and clearance of serum triglycerides. It received its first approval in the USA on 18 November 2025 as an adjunct to diet to reduce triglycerides in adults with FCS. This article summarizes the key milestones in the clinical development of plozasiran leading to its first approval for FCS.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"753-758"},"PeriodicalIF":14.7,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147364438","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}