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Correction: Senaparib: First Approval. 更正:西拿帕尼:首次批准。
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-15 DOI: 10.1007/s40265-025-02245-2
Arnold Lee
{"title":"Correction: Senaparib: First Approval.","authors":"Arnold Lee","doi":"10.1007/s40265-025-02245-2","DOIUrl":"https://doi.org/10.1007/s40265-025-02245-2","url":null,"abstract":"","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":14.4,"publicationDate":"2025-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145291574","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}
引用次数: 0
Sepiapterin: First Approval. sepapterin:第一次批准。
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-15 DOI: 10.1007/s40265-025-02247-0
Yvette N Lamb
{"title":"Sepiapterin: First Approval.","authors":"Yvette N Lamb","doi":"10.1007/s40265-025-02247-0","DOIUrl":"https://doi.org/10.1007/s40265-025-02247-0","url":null,"abstract":"<p><p>An oral formulation of sepiapterin (Sephience™) is being developed by PTC Therapeutics for the treatment of phenylketonuria (PKU). Sepiapterin is a natural precursor of enzyme cofactor tetrahydrobiopterin (BH<sub>4</sub>), which is required for phenylalanine hydroxylase (PAH)-mediated phenylalanine metabolism. Sepiapterin enhances PAH activity, which is deficient in patients with PKU. Sepiapterin received its first approval on 19 June 2025 in the European Union, for use in hyperphenylalaninaemia (HPA) in adult and paediatric patients with PKU. This was followed by approval in the USA on 28 July 2025, for the treatment of HPA in adult and paediatric patients ≥ 1 month of age with sepiapterin-responsive PKU. This article summarizes the milestones in the development of sepiapterin leading to its first approval for HPA in adult and paediatric patients with PKU.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":14.4,"publicationDate":"2025-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145291509","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}
引用次数: 0
Current and Emerging Treatments for Acid Sphingomyelinase Deficiency. 酸性鞘磷脂酶缺乏症的最新治疗方法。
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-12 DOI: 10.1007/s40265-025-02240-7
Tamires Silva Alves, Ana Luíza Fonseca Siqueira, Roberto Giugliani
{"title":"Current and Emerging Treatments for Acid Sphingomyelinase Deficiency.","authors":"Tamires Silva Alves, Ana Luíza Fonseca Siqueira, Roberto Giugliani","doi":"10.1007/s40265-025-02240-7","DOIUrl":"https://doi.org/10.1007/s40265-025-02240-7","url":null,"abstract":"<p><p>Acid sphingomyelinase deficiency is an ultra-rare disease characterised by generalised storage of sphingomyelin, caused by deficiency of the lysosomal enzyme acid sphingomyelinase, owing to the presence of biallelic pathogenic variants in the SMPD1 gene. The main disease manifestations are in the liver, spleen, lung and bone - with some patients having also involvement of the central nervous system. Patients show variable degrees of anaemia, thrombocytopenia and lipid abnormalities, among other findings. Clinically, acid sphingomyelinase deficiency spans from an acute neurovisceral form, with neurological involvement and early death (type A), to a chronic visceral disease, with no or minimal neurological manifestations (type B). An intermediate form, with chronic neurovisceral involvement, is presented by some patients (type A/B). Diagnosis involves the measurement of biomarkers, an assay of enzyme activity and genetic testing. Until a few years ago, treatment was mainly dependent on symptomatic management and bone marrow or solid organ (liver and/or lung) transplantation. In 2022, a specific enzyme replacement therapy with olipudase alfa was approved, and the results available indicate that it changed the therapeutic landscape for patients with acid sphingomyelinase deficiency type B and A/B. Research is being developed to address the needs of patients with acid sphingomyelinase deficiency type A, with gene therapy remaining as a promising approach.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":14.4,"publicationDate":"2025-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145274031","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}
引用次数: 0
Lisaftoclax: First Approval. Lisaftoclax:第一次批准。
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-08 DOI: 10.1007/s40265-025-02251-4
Yahiya Y Syed
{"title":"Lisaftoclax: First Approval.","authors":"Yahiya Y Syed","doi":"10.1007/s40265-025-02251-4","DOIUrl":"https://doi.org/10.1007/s40265-025-02251-4","url":null,"abstract":"<p><p>Lisaftoclax is a B-cell lymphoma 2 (BCL-2) inhibitor developed by Ascentage Pharma for the treatment of haematological malignancies. It is administered using a 5-day ramp-up schedule, reaching the target dose on day 6. Lisaftoclax received its first approval on 10 July 2025 in China for the treatment of chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL) in adult patients who had received at least one prior systemic therapy, including a Bruton's tyrosine kinase (BTK) inhibitor. This article summarizes the milestones in the development of lisaftoclax leading to this first approval for CLL/SLL.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":14.4,"publicationDate":"2025-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145250127","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}
引用次数: 0
The Current Treatment Landscape for Congenital Adrenal Hyperplasia. 目前先天性肾上腺增生症的治疗前景。
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-02 DOI: 10.1007/s40265-025-02216-7
Joseph J Tonge, Irina Bacila, Nils P Krone
{"title":"The Current Treatment Landscape for Congenital Adrenal Hyperplasia.","authors":"Joseph J Tonge, Irina Bacila, Nils P Krone","doi":"10.1007/s40265-025-02216-7","DOIUrl":"https://doi.org/10.1007/s40265-025-02216-7","url":null,"abstract":"<p><p>Congenital adrenal hyperplasia (CAH) is a group of autosomal recessive endocrine disorders caused by impaired steroidogenesis and insufficient cortisol production from the adrenal cortex. The most common form of CAH is 21-hydroxylase deficiency, caused by mutations in the CYP21A2 gene. This enzyme deficiency causes failure to progress through the steroidogenic pathways, consequently there is an increased synthesis of steroid hormone precursors that result in adrenal androgens excess, leading to virilisation in female patients. Over the past two decades, significant progress has been made in optimising corticosteroid replacement strategies, with a focus on reducing androgen excess while minimising glucocorticoid exposure. Modified-release hydrocortisone formulations, such as Efmody<sup>®</sup> and Plenadren<sup>®</sup>, as well as continuous subcutaneous infusion therapies, have been developed to help better mimic physiological cortisol rhythms. In addition, corticotropin-releasing hormone-1 (CRH1) receptor antagonists have been investigated and have now been approved as a novel approach to reducing adrenocorticotropic hormone (ACTH)-driven adrenal hyperplasia and androgen excess. More recently, promising novel approaches have been developed to control androgen excess and reduce glucocorticoid exposure. This review provides an overview of current standard-of-care treatments for CAH and highlights recent advancements in therapeutic strategies. By addressing the limitations of traditional glucocorticoid replacement, these innovations have the potential to improve long-term outcomes and quality of life for patients with CAH.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":14.4,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145205948","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}
引用次数: 0
Avutometinib and Defactinib: First Approval. Avutometinib和Defactinib:首次批准。
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-01 Epub Date: 2025-08-05 DOI: 10.1007/s40265-025-02215-8
Hannah A Blair
{"title":"Avutometinib and Defactinib: First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40265-025-02215-8","DOIUrl":"10.1007/s40265-025-02215-8","url":null,"abstract":"<p><p>Avutometinib and defactinib (AVMAPKI<sup>™</sup> FAKZYNJA<sup>™</sup> CO-PACK) is a co-packaged rapidly accelerating fibrosarcoma (RAF)/mitogen-activated protein kinase kinase (MEK) inhibitor (avutometinib) and focal adhesion kinase (FAK)/proline-rich tyrosine kinase-2 (Pyk2) inhibitor (defactinib) being developed by Verastem Oncology for the treatment of RAS/MAPK pathway-driven cancers. In May 2025, avutometinib and defactinib was approved in the USA for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. This article summarizes the milestones in the development of avutometinib and defactinib leading to this first approval for KRAS-mutated recurrent LGSOC.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"1319-1327"},"PeriodicalIF":14.4,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144783822","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}
引用次数: 0
Update on Medical Treatment of Cushing's Syndrome. 库欣综合征医学治疗进展
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-01 Epub Date: 2025-09-15 DOI: 10.1007/s40265-025-02223-8
Brendan R Dillon, Nidhi Agrawal, Yair Schwarz, Kristen Dancel-Manning, Antoine Tabarin, André Lacroix, Leo J Hofland, Richard A Feelders
{"title":"Update on Medical Treatment of Cushing's Syndrome.","authors":"Brendan R Dillon, Nidhi Agrawal, Yair Schwarz, Kristen Dancel-Manning, Antoine Tabarin, André Lacroix, Leo J Hofland, Richard A Feelders","doi":"10.1007/s40265-025-02223-8","DOIUrl":"10.1007/s40265-025-02223-8","url":null,"abstract":"<p><p>First-line treatment of endogenous Cushing's syndrome (CS) is surgical removal of the tumor responsible for cortisol excess. However, medical therapy has an established role in treatment when patients are not surgical candidates or decline surgery, residual or recurrent disease is present and not amenable to repeat resection, and control of hypercortisolism is needed either preoperatively or while awaiting the effects of radiotherapy. The approach to medical therapy should be tailored based on the etiology, degree of hypercortisolism, and patient characteristics. Currently available medical therapy for all etiologies of CS either blocks adrenal production of cortisol or blocks its action at the level of the glucocorticoid receptor. Currently available medical therapy for Cushing's disease (CD) targets the adrenocorticotropic hormone-secreting pituitary tumor through activation of somatostatin and dopamine receptors, alkylating DNA damage, or immune system activation. More focused therapy with greater efficacy and fewer adverse effects is needed, particularly in the case of CD, with potential targets and drugs identified and in development.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"1207-1230"},"PeriodicalIF":14.4,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12484323/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069266","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}
引用次数: 0
Current Evidence on Celecoxib Safety in the Management of Chronic Musculoskeletal Conditions: An Umbrella Review. 目前关于塞来昔布治疗慢性肌肉骨骼疾病安全性的证据:综述。
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-01 Epub Date: 2025-09-05 DOI: 10.1007/s40265-025-02234-5
Charlotte Beaudart, Christian Brabant, Majed Alokail, Jean-Yves Reginster, Olivier Bruyère
{"title":"Current Evidence on Celecoxib Safety in the Management of Chronic Musculoskeletal Conditions: An Umbrella Review.","authors":"Charlotte Beaudart, Christian Brabant, Majed Alokail, Jean-Yves Reginster, Olivier Bruyère","doi":"10.1007/s40265-025-02234-5","DOIUrl":"10.1007/s40265-025-02234-5","url":null,"abstract":"<p><strong>Objectives: </strong>Our objective was to systematically synthesize and evaluate the existing evidence from meta-syntheses (systematic reviews and meta-analyses) reporting on the safety of celecoxib in adults with chronic musculoskeletal disorders.</p><p><strong>Methods: </strong>We conducted a comprehensive literature search in November 2024 across MEDLINE, Cochrane Central, and Scopus databases, following Preferred Reporting Items for Systematic Reviews and Meta-Analyses 2020 guidelines for umbrella reviews. Only systematic reviews and meta-analyses involving celecoxib safety in osteoarthritis, rheumatoid arthritis, or ankylosing spondylitis were included. We assessed the risk of bias using the AMSTAR-2 tool and graded the certainty of evidence using GRADE.</p><p><strong>Results: </strong>Of 2294 retrieved records, 16 systematic reviews based on randomized controlled trials met the inclusion criteria (14 of 16 were rated as critically low quality). Celecoxib was consistently associated with a lower risk of gastroduodenal ulcers than were non-selective non-steroidal anti-inflammatory drugs (NSAIDs), and some studies also reported fewer gastrointestinal complaints and serious events with celecoxib than with non-selective NSAIDs. Cardiovascular safety outcomes were generally similar to those with non-selective NSAIDs, although one meta-analysis showed a lower risk of cardiovascular mortality with celecoxib. Compared with placebo or non-selective NSAIDs, celecoxib did not increase the risk of renal dysfunction or elevated creatinine and may be associated with fewer renal adverse events. Evidence on all-cause mortality was limited and inconsistent, but one study suggested a lower risk than with non-selective NSAIDs.</p><p><strong>Conclusions: </strong>Celecoxib appears to offer better gastrointestinal safety than non-selective NSAIDs. Although data on cardiovascular, renal, and mortality outcomes suggest possible advantages, the evidence remains limited and of low certainty. Moreover, some real-world evidence raises concerns in specific high-risk populations. Future research should integrate data from both randomized trials and observational studies to better inform long-term safety assessments and guide individualized treatment decisions.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"1289-1306"},"PeriodicalIF":14.4,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12484377/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144999936","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}
引用次数: 0
Acoltremon: First Approval. Acoltremon:第一次批准。
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-01 Epub Date: 2025-07-30 DOI: 10.1007/s40265-025-02218-5
Arnold Lee
{"title":"Acoltremon: First Approval.","authors":"Arnold Lee","doi":"10.1007/s40265-025-02218-5","DOIUrl":"10.1007/s40265-025-02218-5","url":null,"abstract":"<p><p>Acoltremon (TRYPTYR<sup>®</sup>) is a TRPM8 thermoreceptor agonist formulated as eye drops, which has been developed by Alcon for the treatment of dry eye disease. As an insufficient aqueous layer is linked to dry eye disease, the stimulation of tear production by acoltremon can relieve symptoms associated with dry eye disease. During phase III trials in patients with dry eye disease, acoltremon demonstrated increased tear production with a low treatment discontinuation rate. This article summarizes the milestones in the development of acoltremon leading to this first approval for the treatment of the signs and symptoms of dry eye disease.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"1307-1310"},"PeriodicalIF":14.4,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144741639","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}
引用次数: 0
Minimal Residual Disease Negativity as the Primary Goal of Multiple Myeloma Therapy. 最小残留病阴性作为多发性骨髓瘤治疗的首要目标。
IF 14.4 1区 医学
Drugs Pub Date : 2025-10-01 Epub Date: 2025-09-04 DOI: 10.1007/s40265-025-02232-7
Jennifer H Cooperrider, Benjamin A Derman
{"title":"Minimal Residual Disease Negativity as the Primary Goal of Multiple Myeloma Therapy.","authors":"Jennifer H Cooperrider, Benjamin A Derman","doi":"10.1007/s40265-025-02232-7","DOIUrl":"10.1007/s40265-025-02232-7","url":null,"abstract":"<p><p>Measurable (or minimal) residual disease (MRD) testing offers critical prognostic insight in multiple myeloma (MM), surpassing conventional response criteria. While bone-marrow-based assays are most commonly performed, MRD assessment in peripheral blood and advanced imaging may add complementary value. A comprehensive approach, integrating serial MRD testing across compartments, may offer the most accurate appraisal of disease burden. MRD has been validated as a surrogate endpoint for accelerated approval (AA) of MM therapies and is increasingly adopted as a key clinical trial endpoint. Ongoing phase 3 trials are using MRD status to tailor consolidation and maintenance strategies, and emerging evidence supports its role in guiding treatment de-escalation, including discontinuation of maintenance therapy. However, barriers remain to implementing MRD as a treatment goal, including cost, complexity of interpreting results, and uncertainty around the optimal timing for guiding decision-making. Moreover, there is a paucity of data on the use of MRD resurgence to prompt changes in therapy. While MRD negativity represents a compelling endpoint in both clinical practice and research, prospective randomized studies will help to better elucidate how best to incorporate MRD into the MM treatment paradigm.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"1231-1251"},"PeriodicalIF":14.4,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12484384/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144999947","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}
引用次数: 0
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