A Multicentric, Open-label, Non-randomized Study to Evaluate the Pharmacokinetic, Safety and Tolerability of ITF2357 Given as an Oral Single 50 mg Dose in Participants With Chronic Hepatic Impairment Relative to Matched Participants With Normal Hepatic Function

This is a multicentric, open-label, non-randomized study to evaluate the pharmacokinetic, safety and tolerability of ITF2357 in participants with chronic hepatic impairment relative to matched participants with normal hepatic function.

开始日期2025-05-28

申办/合作机构

Italfarmaco SpA

[+1]

NCT06769633

/ Recruiting临床2期

A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old

This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients.
* Planned screening duration: approximately 4 weeks
* Planned Core Treatment duration: approximately 48 weeks
* Planned Extension Treatment duration: approximately 96 weeks
* Planned Follow Up duration: approximately 4 weeks (? 7 days)
* Total duration of study participation: up to 151 weeks (ie, 37-38 months)

开始日期2025-01-02

申办/合作机构

Italfarmaco SpA

[+1]

CTIS2024-511823-32-00

/ Recruiting临床2期

A Phase 2 Open label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old - DSC/14/2357/52

开始日期2025-01-02

申办/合作机构

Italfarmaco SpA

100 项与吉维诺司他相关的临床结果

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100 项与吉维诺司他相关的转化医学

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100 项与吉维诺司他相关的专利（医药）

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261

项与吉维诺司他相关的文献（医药）

2025-10-01·BRAIN & DEVELOPMENT

Duchenne muscular dystrophy: Evolving therapeutic strategies and multidimensional evaluation approaches

Review

作者: Komaki, Hirofumi

Duchenne muscular dystrophy (DMD) is a severe X-linked muscular disorder caused by the absence of dystrophin. Standard therapies prolong survival; however, there is an increasing need for disease-modifying approaches and sensitive evaluation tools. This review presents a summary of the recent advances in DMD therapeutic strategies and multidimensional evaluation approaches, emphasizing their clinical applicability and future perspectives. In this study, a comprehensive review of the current literature was conducted focusing on mutation-specific therapies (exon skipping and gene therapy), non-mutation-specific pharmacological interventions (steroids, vamorolone, and histone deacetylase inhibitors), and emerging modalities such as cell-based therapy. The clinical outcome measures used across the disease stages were also examined, ranging from functional motor tests and imaging biomarkers to digital endpoints and quality of life (QOL) indices. Exon skipping and microdystrophin gene therapies have shown promising results in restoring partial dystrophin expression. Vamorolones and givinostat are alternative drugs with improved safety profiles. Time-based motor tests (such as time to sand, 10 m run) and digital biomarkers (Stride Velocity 95th Centile) enable the earlier detection of disease progression. Natural history data, including large registry-based datasets, enhance the interpretation of clinical trials. Patient-reported outcomes and disease-specific QOL measures, such as the DMD-QoL, are increasingly being incorporated to assess meaningful treatment benefits. The therapeutic landscape of DMD is evolving toward combination and personalized approaches. Multidimensional stage-specific evaluations are essential to capture functional changes and their therapeutic effects. Ongoing research on gene-, pharmacological-, and cell-based therapies, coupled with robust outcome assessments, may further improve patient care and QOL.

2025-09-01·Molecular Therapy-Nucleic Acids

Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy

Article

作者: Farr, Gist H ; Louie, Ke'ale W ; Maenza, Alyssa ; Paquette, Alison G ; Paguio, Alison ; Hasegawa, Eva H ; Henry, Clarissa A ; Ignacz, Amanda C ; Maves, Lisa

Duchenne muscular dystrophy (DMD) is the most common inherited muscle disease. There are currently few effective therapies to treat the disease, although many approaches are being pursued. Certain histone deacetylase inhibitors (HDACi) have been shown to ameliorate DMD phenotypes in mouse and zebrafish models, and the HDACi givinostat has recently gained FDA approval for DMD. Our goal was to identify additional HDACi, or other classes of epigenetic small molecules, that are beneficial for DMD. Using an established animal model for DMD, the zebrafish dmd mutant strain sapje, we screened a library of over 800 epigenetic small molecules. Our screening identified a new HDACi, SR-4370, that ameliorated dmd mutant zebrafish skeletal muscle degeneration, as well as additional HDACi that have previously been shown to improve dmd zebrafish. We find that a single early treatment of HDACi can ameliorate the muscle phenotype and increase lifespan in dmd zebrafish. Furthermore, we find that HDACi treatments that improve dmd muscle also cause increased histone acetylation in zebrafish larvae. Our results add to the growing evidence that HDACi are promising candidates for treating DMD. Our study also provides further support for the effectiveness of small molecule screening in dmd zebrafish.

2025-09-01·BIOCHEMICAL PHARMACOLOGY

Givinostat rescues folding of cystathionine beta-synthase and ameliorates murine homocystinuria

Article

作者: Philipp, Thilo Magnus ; Petrosino, Maria ; Ascenção, Kelly ; Bremer, Olivier ; Majtan, Tomas ; Szabo, Csaba ; Mijatovic, Ela ; Zuhra, Karim

Homocystinuria (HCU) is an inherited metabolic disorder caused by missense mutations in the cystathionine beta-synthase (CBS) gene, leading to protein misfolding and degradation. Pharmacological chaperones, which stabilize native protein conformations, offer a promising therapeutic strategy, but tools for their identification are lacking. We developed a cell-based CBS folding reporter assay using split-fluorescent protein complementation, focusing on the common HCU-causing CBS I278T variant. In addition to proteasome inhibitors investigated as potential treatment for HCU, screening of chemical libraries identified several histone deacetylase inhibitors, with givinostat showing the highest recovery of CBS I278T folding and activity. Givinostat binds CBS, but also acts indirectly by modulating the proteostasis network and protein degradation pathways. Short-term treatment of HCU mice expressing CBS I278T partially restored hepatic CBS expression and reduced serum homocysteine levels. This study presents a novel tool, which lead to identification of new class of potential pharmacological chaperones for HCU, paving the way for personalized assays targeting different pathogenic variants and adaptations for other protein misfolding disorders.

项与吉维诺司他相关的新闻（医药）

2025-09-17

·GlobeNewswire-Health Care

Italfarmaco Announces Exclusive Distribution Agreement with Multicare Pharma for DMD Treatment Givinostat in Brazil

September 16, 2025 – Italfarmaco S.p.A. today announced an exclusive agreement with Multicare Pharma, a prominent pharmaceutical company in Latin America. Multicare Pharma will support the regulatory approval in Brazil and distribute givinostat (Duvyzat®), Italfarmaco’s novel histone deacetylase (HDAC) inhibitor to treat Duchenne muscular dystrophy (DMD). According to the terms of the agreement, Multicare will manage supply and distribution operations for the drug within Brazil. No financial terms were disclosed. “At Italfarmaco, our commitment to rare diseases drives every decision we make. Access to givinostat can have a meaningful impact for patients in Brazil living with DMD,” said Antonio Nardi, Vice President and Head of Business & Portfolio Development of Italfarmaco. “Partnering with Multicare, a leader in the field of rare and highly complex diseases with extensive reach across Brazil, allows us to bring this innovative treatment to those who need it most.” Duchenne muscular dystrophy (DMD) is a rare, progressive neuromuscular disorder caused by mutations in the DMD gene. Mutations in the DMD gene prevent the production of functional dystrophin, causing the dystrophin-associated protein complex (DAPC) to break down. This makes muscle fibres more vulnerable to damage and increases histone deacetylase (HDAC) levels in the muscle cells, blocking the activation of important genes needed for muscle maintenance and repair. As a result, muscle fibres experience ongoing damage, leading to chronic inflammation and poor regeneration. Over time, muscle cells die and are replaced by scar tissue and fat.1-4 DMD primarily affects males, with symptoms typically appearing between the ages of two and five. As the condition progresses, muscle weakness worsens, leading to difficulty walking and eventually to loss of ambulation. Over time, the heart and respiratory muscles are also affected, which are the leading causes of premature death.5 DMD is one of the most severe and common forms of childhood muscular dystrophy, with a global birth incidence of approximately 1 in 5,050 boys.6 Givinostat (Duvyzat®) was discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Givinostat is an orally administered histone deacetylase (HDAC) inhibitor that regulates the excessive HDAC activity characteristic of DMD muscles. By doing so, it helps restore the expression of key genes and biological processes essential for muscle maintenance and repair. Its mechanism of action is independent of the specific dystrophin gene mutation causing the disease.7,8 Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production and commercialisation with proven success in many therapeutic areas including immuno-oncology, gynaecology, neurology, cardiovascular disease and rare diseases. Italfarmaco's rare disease unit includes programmes in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythaemia vera. Multicare Pharma is a leading pharmaceutical company with over 40 years of experience in Latin America. Specializing in the licensing, marketing authorization, promotion, importation, and distribution of innovative treatments, Multicare Pharma focuses on addressing the unmet needs of patients with rare and complex diseases. The company offers a comprehensive range of products and services, from regulatory evaluation to drug distribution, ensuring timely access to essential therapies across the region. With a commitment to improving patient outcomes, Multicare Pharma partners with global pharmaceutical companies to enhance healthcare delivery in Latin America. References: Sandonà M, Cavioli G, Renzini A, et al. Histone Deacetylases: Molecular Mechanisms and Therapeutic Implications for Muscular Dystrophies. Int J Mol Sci. 2023;24(5):4306. https://doi-org.libproxy1.nus.edu.sg/10.3390/ijms24054306. Consalvi S, Saccone V, Giordani L, Minetti G, Mozzetta C, Puri PL. Histone Deacetylase Inhibitors in the Treatment of Muscular Dystrophies: Epigenetic Drugs for Genetic Diseases. Mol Med. 2011;17(5):457–465. https://doi-org.libproxy1.nus.edu.sg/10.2119/molmed.2011.00049. Bez Batti Angulski A, Hosny N, Cohen H, et al. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies. Front Physiol. 2023;14:1183101. https://doi-org.libproxy1.nus.edu.sg/10.3389/fphys.2023.1183101. Giuliani G, Rosina M, Reggio A. Signaling pathways regulating the fate of fibro/adipogenic progenitors (FAPs) in skeletal muscle regeneration and disease. FEBS J. 2022;289(21):6484–6517. https://doi-org.libproxy1.nus.edu.sg/10.1111/febs.16080. Walter MC, Reilich P. Recent developments in Duchenne muscular dystrophy: facts and numbers. J Cachexia Sarcopenia Muscle. 2017;8(5):681–685. https://doi-org.libproxy1.nus.edu.sg/10.1002/jcsm.12245. Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifirò G. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis. 2020;15(1):141. https://doi-org.libproxy1.nus.edu.sg/10.1186/s13023-020-01430-8. Comi G, Bertini E, Vita G, et al. S22.008: Development of the histone deacetylases inhibitor Givinostat in Duchenne Muscular Dystrophy. Poster. Neurology. 2018;90(15 (Supplement)). Licandro SA, Crippa L, Pomarico R, et al. The pan HDAC inhibitor Givinostat improves muscle function and histological parameters in two Duchenne muscular dystrophy murine models expressing different haplotypes of the LTBP4 gene. Skelet Muscle. 2021;11(1):19. https://doi-org.libproxy1.nus.edu.sg/10.1186/s13395-021-00273-6. The content above comes from the network. if any infringement, please contact us to modify.

上市批准引进/卖出

2025-09-06

·药明康德

致敬时代 | 将致命绝症变为“慢性病”：医药创新如何让这类患者寿命延长近一倍？

编者按：2025年，药明康德迎来创立25周年的重要里程碑。值此契机，我们向所有与我们共同书写产业变革篇章的科学家、医药人和投资者致以衷心感谢与诚挚敬意，也特别推出“致敬时代”系列，回顾全球同仁如何借助科学与合作的力量，不断拓展治疗边界、改善患者命运。四分之一个世纪的坚守，只为加速每一款新药的诞生。下一个25年，我们将继续心怀感恩与敬畏，依托独特的CRDMO模式，与全球伙伴携手同行，共赴健康未来。杜氏肌营养不良症（Duchenne Muscular Dystrophy，DMD）是一种致命的、进行性的肌肉萎缩性疾病，由编码抗肌萎缩蛋白（dystrophin）的基因发生变异导致。全球范围内，大约每3500-5000名男性新生儿中就有1名DMD患儿，每年有2万名儿童确诊DMD。这些患者的肌无力症状在2-3岁时就会显现，并随着年龄逐渐加重。在缺乏有效治疗的情况下，疾病进展通常导致患者在12岁左右便需借助轮椅行动，多数人的生命会因心肺并发症在青少年时期或二十岁出头时就走到尽头。过去30多年来，随着皮质类固醇的使用、无创呼吸支持以及DMD相关并发症的主动监测和管理，该病患者的运动能力、身体功能、生活质量和预期寿命均已获得显著改善。近十年来，多种针对DMD根本病因——抗肌萎缩蛋白缺失的创新疗法相继涌现，为延缓、甚至逆转疾病进展带来了前所未有的希望。在这场迎战DMD的攻坚征程中，药明康德深感荣幸能够与全球合作伙伴并肩前行，持续赋能DMD领域创新疗法的开发与落地，将前沿科学突破转化为患者的生命希望。图片来源：123RF 跨越百年揪出肌肉无力的病因杜氏肌营养不良症这个疾病名称，来自一位19世纪的法国医生——纪尧姆-本杰明-阿曼德·杜兴（Guillaume-Benjamin-Amand Duchenne）。杜兴医生一生痴迷于探索神经与肌肉疾病，常年穿梭于巴黎各大医院的病房之间，执着地寻找并记录那些罕见的病例。凭借对每一位患者的细致观察和系统记录，杜兴医生精准描述了许多当时未知的神经肌肉病症。1861年，他在著作中记录了一种特殊的进行性肌无力疾病，并将其命名为“假性肥大型肌营养不良”——患者最显著的特征是小腿的腓肠肌看似粗壮发达，实则肌力显著减弱。1868年，他通过对13例同类病例的系统总结，提供了当时对该病最全面的临床描述，为其作为独立疾病的认知奠定了基础。因此，尽管后世发现杜兴医生可能并非最早注意到该病的人，医学界仍然决定用他的名字来命名这种疾病，以此表彰和纪念他的重要贡献。此后，科学家们曾提出各种假说试图解释这类患者的肌肉为何会逐渐坏死，但均未获证实。直至一个多世纪后，基因研究的突破才最终揭示：这种疾病的根源，在于编码抗肌萎缩蛋白的基因发生了变异。 “治标”措施显著延长患者预期寿命在健康的肌肉组织中，抗肌萎缩蛋白就像一个减震器，能在肌肉细胞收缩和舒张时起稳定作用。而患有DMD的患者产生的抗肌萎缩蛋白很少或者根本就不生产，这使得肌肉在收缩过程中不断受损、发炎，最终被脂肪和瘢痕组织替代，丧失功能。 ▲DMD患者和健康人的肌肉横断面染色对比图（图片来源：参考资料[1]） DMD的病情进展通常始于下肢肌肉，尤其是腓肠肌、胫骨前肌和趾长伸肌等肌群。随着肌无力向全身蔓延，该病后期还会累及呼吸肌和心肌，导致呼吸功能衰竭和心力衰竭，这是DMD患者的两大主要致死原因。在很长一段时间内，DMD由于缺乏有效的治疗手段，患者的中位预期寿命不足26岁。然而，过去三十多年来，DMD综合治疗与系统性护理的发展显著改善了患者的预后。尤其是在心肺功能支持方面，通过机械辅助咳嗽和无创通气技术优化呼吸管理，以及早在10岁左右起推荐患者使用血管紧张素转换酶抑制剂（ACEI）进行心功能保护，有效延缓了患者心肺并发症的进展。据2013年的一项研究显示，1970年后出生的DMD患者的中位生存期已延长至超过40岁。除了这两方面，皮质类固醇的使用在DMD临床治疗中也占据着重要地位。国际指南强烈建议在患儿运动功能进入平台期或开始下降时（通常为4-5岁，不早于2岁）启动激素治疗，并建议长期使用。这类药物已被证实可有效延缓肌力衰退、维持行走能力、保护心肺功能并减少脊柱侧弯的发生。目前，经FDA批准用来治疗DMD的皮质类固醇药物包括Emflaza（deflazacort）和Agamree（vamorolone）。除了类固醇这类基础抗炎药物外，2024年3月，美国FDA批准了一款新型机制药物——Duvyzat（givinostat）。作为首个获批可用于治疗携带任何DMD基因变异患者的非类固醇疗法，Duvyzat旨在通过抑制DMD患者中异常升高的组蛋白去乙酰化酶（HDAC）活性，缓解肌肉组织的损害，保护肌肉超细纤维的功能，从而为此类患者提供一种全新的治疗策略，也与类固醇激素形成了重要的协同与补充。然而，上述方法对于DMD患者来说还是“治标不治本”。尽管许多患者的生存时间得到了延长，其肌肉功能的持续下降依然会严重影响生活质量。患者家庭和医疗界都迫切期待能够找到“根治”DMD的新疗法，以期真正改善患者的运动功能与生活品质。针对DMD疾病根源的新治疗模式对于如何“根治”DMD的问题，科学家们的思路是，既然DMD的产生源于抗肌萎缩蛋白的缺失，那么提高抗肌萎缩蛋白的表达，就有望恢复肌肉细胞的功能，减少肌肉细胞的损伤。实现这一目标的关键在于修正发生变异的DMD基因，或直接向细胞内递送一个完整的健康基因。 1990年发表的一例特殊病例报告给科学家们提供了一个关键启示。在该案例中，一名男子的抗肌萎缩蛋白基因缺失了将近一半，却没有出现肌肉无力的症状，并且还活到了61岁。这个案例表明：即使基因不完整，机体仍有可能产生具备足够功能的截短蛋白，以维持正常的生理活动。这为开发DMD治疗策略开辟了全新思路：如果完全修复DMD基因存在巨大的技术挑战，不如“弃车保帅”。比如，科学家们可以跳过那些出问题的、非关键的基因片段，合成仍有功能的“简化版”蛋白质。针对DMD的外显子跳跃疗法应运而生。这种新型疗法的作用机制是，通过让寡核苷酸疗法与编码抗肌萎缩蛋白的mRNA前体结合，改变mRNA前体的剪接过程，跳过携带基因突变的DMD外显子，从而生成具有大部分抗肌萎缩蛋白功能的截短蛋白。 2016年，由Sarepta Therapeutics公司研发的首个外显子跳跃疗法Exondys 51（eteplirsen）获FDA批准上市，适用于适合外显子51跳跃治疗的DMD患者。临床试验数据显示，接受180周治疗后，患者肌肉中的抗肌萎缩蛋白水平达到了对照组的11.6倍。此后，多款类似药物相继问世，包括Sarepta Therapeutics公司的Vyondys 53（golodirsen），Amondys 45（casimersen），以及Nippon Shinyaku公司的Viltepso（viltolarsen），分别针对不同的外显子跳跃类型，惠及更广泛的DMD患者群体。 ▲以Viltepso（又名NS-065）为例展示的寡核苷酸疗法作用机制（图片来源：Nippon Shinyaku公司官网）值得一提的是，所有4款获批的寡核苷酸疗法均采用了磷二酰胺吗啉寡聚物（PMO）技术。PMO是一类基于RNA天然框架设计的合成分子，其碱基与吗啉环相连，并通过磷酰二胺键进行连接，而非天然RNA中的核糖环与磷酸二酯键。这一特殊结构使PMO能够高效、稳定地与靶标mRNA前体结合，同时在体内表现出优异的抗降解能力，为外显子跳跃策略提供了坚实的技术基础。首款DMD基因疗法诞生对于DMD这类单基因遗传病，理论上的治愈方法之一是通过基因疗法向患者体内递送一个功能正常的基因拷贝，从而持久地表达所需蛋白。然而，DMD基因疗法的开发面临两大严峻挑战：第一，常用作递送载体的病毒天然倾向感染肝脏和肺，难以高效靶向肌肉组织；第二，DMD基因尺寸巨大，远超常用病毒载体的装载容量，导致全长基因无法被递送。转机出现在1996年。当时，北卡罗来纳大学的裘德·萨穆尔斯基（Jude Samulski）博士团队成功设计出了能够感染肌肉细胞的腺相关病毒（AAV）。随后，一些科学家也相继开发出了大小适合病毒载体的精简版抗肌萎缩蛋白——微抗肌萎缩蛋白（micro-dystrophin）。2006年，6名肌肉萎缩症患者接受了一种局部注射的DMD基因疗法，证实了萨穆尔斯基博士的AAV病毒能够安全地将微抗肌萎缩蛋白基因递送到人类的肌肉细胞中。这次关键性的成功，极大地激励了研究团队向终极目标发起挑战：开发出一种能够实现全身给药的基因疗法。历过十年的不懈探索和反复优化，一款代号为SRP-9001的新型基因疗法终于诞生。该疗法在临床试验中取得了令人振奋的效果。例如，一名4岁的DMD患儿在接受治疗后仅3天，运动能力就有了明显提升——从之前上楼困难，变为可以双脚交替上下楼梯；治疗11天后，他甚至能够尝试骑自行车。这些积极的早期信号，为疗法的后续推进注入了强大信心。图片来源：123RF 2023年6月，这款由Sarepta Therapeutics公司与罗氏（Roche）联合开发的基因疗法Elevidys（delandistrogene moxeparvovec）获得了美国FDA的加速批准，用于治疗4-5岁、携带特定DMD基因突变的患儿。Elevidys也是首个获FDA批准上市的用于治疗DMD的一次性基因疗法，它的获批标志着DMD治疗领域的又一重大进展，从此，医护人员可以为DMD患者提供针对疾病根本成因的治疗方法，而不仅仅是控制症状。 2024年6月，美国FDA批准Elevidys扩展适应症，适用于所有4岁及以上、经确诊携带DMD基因突变的患者。这是该疗法自首次获批后的一个重要进展，意味着更多DMD患者有机会接受这种一次性治疗。 CRDMO平台护航新药研发行稳致远回望DMD领域的发展历程，近十年来美国FDA批准的8款疗法中，有5款属于针对疾病根本病因的寡核苷酸疗法与基因疗法，标志着该方向已成为DMD新药研发的重要领域。公开资料显示，目前全球范围内有超过40个DMD在研项目，其中约半数聚焦于基因疗法和寡核苷酸疗法，展现出该领域持续的创新活力与发展潜力。其中，寡核苷酸疗法凭借其高度靶向性、灵活可编程和相对较短的开发周期，吸引了产业界的广泛关注。在这一进程中，偶联技术的进步成为推动疗法突破的关键驱动力。然而，诸如PMO等类型的寡核苷酸分子在合成与生产方面极为复杂，也为企业带来了显著的技术与产业化挑战。作为全球赋能寡核苷酸药物研发的一体化、端到端的CRDMO平台，药明康德依托在化学业务领域二十年的深厚积累，为此类创新疗法的开发与产业化提供了坚实支撑。药明康德化学业务平台的WuXi TIDES团队目前具备PMO单体、PMO、多肽、多肽-PMO（PPMO）偶联及制剂开发的综合能力。公开信息显示，WuXi TIDES在多肽和寡核苷酸等新分子的开发和生产方面持续精进平台能力，通过不断优化工艺、内部整合流程提供一体化服务，在3年内将PMO类药物活性成分（API）成本降低60%。在寡核苷酸的偶联方面，WuXi TIDES全面的平台能力可支持寡核苷酸与多肽、脂质、小分子、毒素/药物等组件的偶联，为复杂的寡核苷酸偶联药物提供一体化解决方案。以2024年药明康德投资者开放日上展示的赋能案例为例：针对将多肽与PMO偶联，生产技术极为复杂的PPMO疗法，WuXi TIDES团队寡核苷酸、多肽、制剂、分析等6个团队平行推进、无缝衔接，仅用11个月将其推进至IND申报阶段。这也得益于药明康德在CMC一体化解决方案方面的专长。成立二十五年来，药明康德始终致力于赋能全球合作伙伴加速创新疗法开发，助力更多突破性疗法早日惠及患者。药明康德也期待与业界同仁继续携手，以CRDMO平台之力为新药研发“护航”，共同推动人类健康事业行稳致远。参考资料： [1] Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021 Feb 18;7(1):13. doi: 10.1038/s41572-021-00248-3. [2] Yiu EM, Kornberg AJ. Duchenne muscular dystrophy. J Paediatr Child Health. 2015 Aug;51(8):759-64. doi: 10.1111/jpc.12868. [3] Guillaume-Benjamin-Amand Duchenne Biography (1806-1875). Retrieved August 28, 2025, from http://www.faqs.org/health/bios/99/Guillaume-Benjamin-Amand-Duchenne.html [4] Maranhão-Filho P, Vincent M. Guillaume-Benjamin Duchenne: a miserable life dedicated to science. Arq Neuropsiquiatr. 2019 Jul 15;77(6):442-444. doi: 10.1590/0004-282X20190044. 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

2025-08-20

·PRNewswire

HDAC Inhibitors Market Sees Upward Trajectory Across the 7MM During the Forecast Period (2025-2034) Driven by Innovation in Cancer Therapies | DelveInsight

The HDAC inhibitor pipeline remains active, with several next-generation agents advancing in clinical trials. Abexinostat (Xynomic Pharmaceuticals) is in Phase III development for renal cell carcinoma, representing a potential expansion into solid tumors. Quisinostat (Viriom) is in Phase II for uveal melanoma, and Entinostat, partnered between EOC Pharma and Syndax, is being explored in Hodgkin lymphoma. LAS VEGAS, Aug. 20, 2025 /PRNewswire/ -- DelveInsight's HDAC Inhibitors Market Size, Target Population, Competitive Landscape & Market Forecast report includes a comprehensive understanding of current treatment practices, addressable patient population, which includes top indications such as Multiple Myeloma, Peripheral T-cell Lymphoma (PTCL), Cutaneous T-cell Lymphoma (CTCL), Amyotrophic Lateral Sclerosis (ALS), and others. The selected indications are based on approved therapies and ongoing pipeline activity. The report also provides insights into the emerging HDAC inhibitors, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM. Key Takeaways from the HDAC Inhibitors Market Report As per DelveInsight's analysis, the total market size of HDAC inhibitors in the 7MM is expected to surge significantly by 2034. The report provides the total potential number of patients in the indications, such as Multiple Myeloma, Peripheral T-cell Lymphoma (PTCL), Cutaneous T-cell Lymphoma (CTCL), Amyotrophic Lateral Sclerosis (ALS), and others. Leading HDAC inhibitor companies, such as Xynomic Pharmaceuticals, Viriom, EOC Pharma, Syndax Pharmaceuticals, Jubilant Therapeutics, and others, are developing novel HDAC inhibitors that can be available in the HDAC inhibitors market in the coming years. Some of the key HDAC inhibitors in the pipeline include Abexinostat, Quisinostat, Entinostat, JBI-802, and others. Discover which indication is expected to grab the major HDAC inhibitors market share @ HDAC Inhibitors Market Report HDAC Inhibitors Overview Histone deacetylases (HDACs) remove acetyl groups from both histone and non-histone proteins. Because acetylation of histones is linked to an open chromatin structure and active gene transcription, HDAC activity leads to histone deacetylation and epigenetic suppression of gene expression. Since HDACs influence many pathways involved in cancer development, HDAC inhibitors (HDACis) show clinical effects through several mechanisms, including changes in gene expression of cell cycle and apoptotic proteins, acetylation of other proteins involved in cell growth and survival, effects on angiogenesis, aggresome formation, and DNA repair. There are 18 known HDACs, divided into two major groups and four classes based on their cellular location, homology, enzyme mechanism, sequence of discovery, and histone substrate specificity. The first group includes zinc-dependent HDACs, which all share a similar catalytic core for deacetylating acetyl-lysine residues. These comprise Class I (HDAC1, -2, -3, -8), Class II (IIa: HDAC4, -5, -7, -9; IIb: HDAC6, -10), and Class IV (HDAC11). The second group includes Class III HDACs (sirtuins, SIRT1–7), which rely on NAD+ for their enzymatic activity. HDAC Inhibitors Epidemiology Segmentation The HDAC inhibitors market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Cases of Selected Indications for HDAC Inhibitor Total Eligible Patient Pool of Selected Indications for HDAC Inhibitor Total Treated Cases of Selected Indications for HDAC Inhibitor HDAC Inhibitors Treatment Market Several HDAC inhibitors are currently available on the market. Recent entrants include DUVYZAT (givinostat), while earlier approved drugs include BELEODAQ (belinostat), HIYASTA (tucidinostat), among others. DUVYZAT is a histone deacetylase inhibitor approved for treating Duchenne muscular dystrophy (DMD) in patients aged 6 and above. While it is categorized as an HDAC inhibitor, its exact mechanism of action in DMD is not fully understood. In April 2025, Italfarmaco announced that the CHMP of the European Medicines Agency (EMA) issued a positive opinion on DUVYZAT. The drug is also being evaluated in other indications: Phase III trial in Becker muscular dystrophy (BMD) and Phase II trial in polycythemia vera. HIYASTA (HBI-8000) is an epigenetic immunomodulator approved for several indications, including use as monotherapy for certain subtypes of relapsed or refractory T-cell non-Hodgkin's lymphoma, specifically adult T-cell leukemia-lymphoma (ATLL) and peripheral T-cell lymphoma (PTCL) in Japan. HUYABIO International is conducting a multinational Phase III trial evaluating HIYASTA in combination with nivolumab for previously untreated metastatic or unresectable malignant melanoma. HIYASTA is an oral HDAC Class I inhibitor that induces cell cycle arrest and apoptosis in tumor cells, which underlies its efficacy in lymphoma. In September 2020, the Japanese Ministry of Health, Labour and Welfare (MHLW) granted orphan drug designation (ODD) to HIYASTA for relapsed/refractory ATL, and in December 2015 for PTCL. In June 2021, HUYABIO also received regulatory approval in Japan for HBI-8000 as monotherapy for relapsed/refractory ATL. Key Emerging HDAC Inhibitors and Companies Xynomic Pharmaceuticals (Abexinostat), Viriom (Quisinostat), EOC Pharma/Syndax Pharmaceuticals (Entinostat), Jubilant Therapeutics (JBI-802), and several other companies are currently engaged in the development and production of HDAC inhibitors, which have the potential to significantly impact and enhance the HDAC inhibitors market. Abexinostat, an oral, broad-spectrum HDAC inhibitor originally developed by Pharmacyclics and later licensed by Xynomic Pharmaceuticals in 2017, has demonstrated encouraging activity in both blood cancers and solid tumors. The drug is currently being studied in several Phase I to III clinical trials worldwide. In September 2019, Xynomic announced that the FDA had granted Fast Track Designation (FTD) to abexinostat as a monotherapy for fourth-line treatment of relapsed or refractory follicular lymphoma. The FDA had previously granted FTD for abexinostat in combination with pazopanib for first- or second-line therapy in renal cell carcinoma. Additional Phase II trials are underway in indications such as Diffuse Large B-cell Lymphoma (DLBCL) and Follicular Lymphoma. Quisinostat is another potent, selective oral pan-HDAC inhibitor, initially developed by Janssen Pharmaceuticals. HDAC inhibitors work by inducing cell cycle arrest, promoting differentiation, and triggering apoptosis in various human cancer cell lines. By inhibiting histone deacetylase, they increase acetylation of histone proteins, resulting in a more transcriptionally active chromatin state and altered gene expression involved in proliferation, apoptosis, and differentiation. Multiple preclinical studies and Phase I/II trials have evaluated quisinostat's safety and effectiveness, showing promising anti-tumor effects in several advanced and treatment-resistant cancers — including platinum-resistant ovarian cancer (in combination with paclitaxel and carboplatin), uveal melanoma, and synovial sarcoma. The anticipated launch of these emerging therapies are poised to transform the HDAC inhibitors market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the HDAC inhibitors market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about HDAC inhibitors clinical trials, visit @ HDAC Inhibitors Treatment Recent Developments in the HDAC Inhibitors Space In May 2025, Amylyx Pharmaceuticals announced positive Week 48 data from the Phase II open-label HELIOS clinical trial of AMX0035 in adults living with Wolfram syndrome. In May 2025, the University of Miami, in collaboration with Viriom, initiated a Phase II clinical trial in the US (NCT06932757) to evaluate an oral therapy for patients with uveal melanoma who have undergone at least one prior line of treatment. In April 2025, Xynomic Pharmaceuticals concluded a Phase I/II clinical trial in the United States (NCT03939182), evaluating a combination oral therapy for patients with Diffuse Large B-cell Lymphoma and Mantle Cell Lymphoma who had received at least one prior line of treatment. Discover more about HDAC inhibitors in development @ HDAC Inhibitors Clinical Trials HDAC Inhibitors Market Dynamics HDAC inhibitors are a class of epigenetic drugs that modify gene expression by altering chromatin structure. Initially developed as anticancer agents, they have shown significant therapeutic potential in various malignancies, especially hematologic cancers like T-cell lymphoma and multiple myeloma. The market initially gained traction with FDA approvals of drugs such as Vorinostat, Romidepsin, and Panobinostat. Their unique mechanism of action targeting epigenetic regulation drew interest as precision medicine and oncology research grew, driving pharmaceutical investment in this segment. The HDAC inhibitors market continues to expand largely due to the rising prevalence of cancer worldwide and the need for more targeted therapies with better safety profiles. Additionally, ongoing clinical trials are exploring HDAC inhibitors in combination with other therapies, such as immune checkpoint inhibitors, proteasome inhibitors, and traditional chemotherapeutics. These combination approaches aim to overcome resistance and enhance efficacy, creating more opportunities for market growth. As more data emerges demonstrating synergistic benefits, partnerships between biotech startups and large pharmaceutical firms are accelerating further development. Despite this positive outlook, the market faces several challenges. Many HDAC inhibitors are associated with toxicity issues such as fatigue, gastrointestinal disturbances, and thrombocytopenia, which limit broader adoption. Competition from newer targeted therapies and immuno-oncology drugs also puts pressure on the HDAC inhibitor segment. Moreover, several compounds failed in late-stage trials due to a lack of efficacy, leading to cautious investor sentiment and prioritization of combination rather than monotherapy approaches. Looking ahead, the market dynamics are expected to be shaped increasingly by precision oncology trends and biomarker-driven patient selection, allowing more effective targeting of responders. If safety profiles can be improved and combination regimens continue to show strong results, HDAC inhibitors will retain a meaningful role in the evolving oncology landscape. Novel formulations, selective HDAC isoform inhibitors, and expansion into non-oncology indications such as neurodegenerative or inflammatory diseases may further diversify the market and drive its next phase of growth. Learn more about the HDAC inhibitors @ HDAC Inhibitors Analysis Scope of the HDAC Inhibitors Market Report HDAC Inhibitors Therapeutic Assessment: HDAC Inhibitors' current marketed and emerging therapies HDAC Inhibitors Market Dynamics: Conjoint Analysis of Emerging HDAC Inhibitor Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, HDAC Inhibitors Market Access and Reimbursement Table of Contents Related Reports Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Sanofi, Karyopharm Therapeutics, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., among others. 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临床2期临床3期快速通道孤儿药免疫疗法

100 项与吉维诺司他相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
-	吉维诺司他	-	DB12645

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适应症	国家/地区	公司	日期
杜氏肌营养不良症	美国	Italfarmaco SpA	2024-03-21

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适应症	最高研发状态	国家/地区	公司	日期
真性红细胞增多症	临床3期	美国	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	奥地利	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	保加利亚	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	克罗地亚	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	法国	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	德国	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	匈牙利	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	爱尔兰	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	以色列	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	意大利	Italfarmaco SpA	2024-03-26

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03373968 (EPIDYS, Pubmed \| Ann Clin Transl Neurol)	临床3期	杜氏肌营养不良症	194	Givinostat oral suspension + Givinostat oral suspension	鑰構鑰鑰遞築網壓網糧(蓋膚醖蓋憲網廠膚廠遞) = 餘艱獵鏇鑰衊範構鹽醖範築夢獵製衊窪齋簾鑰 (齋繭繭餘範鬱獵鏇衊憲 )	积极	2025-08-19
NCT02851797 (EPIDYS, GlobeNewswire) 人工标引	临床3期	杜氏肌营养不良症	179	Givinostat	窪窪廠壓願鹽觸築獵醖(襯齋襯選淵鬱鏇鏇衊憲) = Post hoc analysis from the completed Phase 3 EPIDYS trial showed less decline in cardiac function throughout the study, compared to those who received placebo. 衊獵齋窪願鏇鑰獵選衊 (遞糧構衊鑰製遞鏇蓋淵 ) 更多	积极	2025-06-11
NCT03238235 (CTgov)	临床2期	贝克型肌营养不良	51	Givinostat (Givinostat)	衊製蓋廠廠範鹹願膚鏇(醖範鏇鑰觸網範衊遞壓) = 淵廠壓構艱觸廠積鑰願範餘繭製餘襯淵鬱鑰壓 (鹹選膚鑰鏇獵獵齋壓構, 膚憲糧築衊壓構憲齋構 ~ 衊蓋遞襯簾衊顧積醖願) 更多	-	2024-11-18
				Placebo (Placebo)	衊製蓋廠廠範鹹願膚鏇(醖範鏇鑰觸網範衊遞壓) = 膚醖顧願簾鬱鹽餘製範範餘繭製餘襯淵鬱鑰壓 (鹹選膚鑰鏇獵獵齋壓構, 衊觸積築夢餘壓醖積夢 ~ 選醖廠醖艱選膚範鏇衊) 更多
NCT02851797 (EPIDYS, Literature \| Pubmed \| Lancet Neurol) 人工标引	临床3期	杜氏肌营养不良症	120	givinostat	網淵淵夢憲積鬱糧襯膚(廠簾鑰顧襯蓋獵鬱遞構) = 齋鹽製壓繭憲醖膚遞鏇糧築繭鏇繭顧積膚獵淵 (壓壓鹽膚繭蓋壓齋鬱鑰, 1.17 ~ 1.37)	积极	2024-04-01
				Placebo	網淵淵夢憲積鬱糧襯膚(廠簾鑰顧襯蓋獵鬱遞構) = 觸壓築艱蓋糧觸夢鹽遞糧築繭鏇繭顧積膚獵淵 (壓壓鹽膚繭蓋壓齋鬱鑰, 1.17 ~ 1.37)
NCT02851797 (FDA_CDER) 人工标引	临床3期	杜氏肌营养不良症	179	DUVYZAT	築構獵鹽積網憲簾廠襯(壓願壓簾製憲淵製醖遞) = 窪夢膚廠顧顧醖繭繭淵鬱憲夢網願糧繭夢顧選 (餘選齋壓製齋築製淵蓋 )	积极	2024-03-21
NCT05845567 (CTgov)	临床1期	药物相互作用	20	Givinostat (Givinostat Alone (Day 1))	繭觸艱構膚襯鑰壓艱願(範壓簾衊餘積鹹積餘構) = 艱築淵醖膚憲艱廠觸憲鬱夢網蓋蓋衊鏇範積憲 (蓋夢鹽簾範憲膚願蓋製, 24.2) 更多	-	2024-02-09
				Givinostat+Clarithromycin (Givinostat Co-Administered With Clarithromycin (Day 8))	繭觸艱構膚襯鑰壓艱願(範壓簾衊餘積鹹積餘構) = 廠餘衊觸醖鬱遞築膚窪鬱夢網蓋蓋衊鏇範積憲 (蓋夢鹽簾範憲膚願蓋製, 21.9) 更多
NCT00792740 (CD, CTgov)	临床1/2期	克罗恩病	51	Placebo (Placebo)	鹹鹹製觸築蓋鏇夢鬱範 = 製窪獵鹹憲積鬱積遞淵壓壓醖壓醖網選壓獵鹽 (餘顧觸襯鏇製遞鏇觸鏇, 鑰鏇積蓋繭糧願遞製襯 ~ 餘繭範齋願鏇簾襯築齋) 更多	-	2023-07-19
				ITF2357 (ITF2357)	鹹鹹製觸築蓋鏇夢鬱範 = 鏇壓廠廠觸繭鬱醖窪夢壓壓醖壓醖網選壓獵鹽 (餘顧觸襯鏇製遞鏇觸鏇, 簾顧構襯餘壓窪顧繭憲 ~ 壓網膚顧醖構襯衊蓋鹹) 更多
NCT02851797 (Epidys, MDA2023)	临床3期	杜氏肌营养不良症 fat fraction of the vastus lateralis (VL)	179	Givinostat	簾衊夢選餘鹽製壓憲構(窪淵鏇繭鹹鏇齋衊壓顧) = 廠鹹窪壓顧構網憲鏇構淵構膚築築憲夢選艱構 (鏇鏇醖襯獵獵醖壓遞醖 )	积极	2023-03-19
				Placebo	簾衊夢選餘鹽製壓憲構(窪淵鏇繭鹹鏇齋衊壓顧) = 選鑰顧獵顧鬱醖廠淵顧淵構膚築築憲夢選艱構 (鏇鏇醖襯獵獵醖壓遞醖 )
NCT02851797 (Epidys Study, MDA2023)	临床3期	杜氏肌营养不良症	179	Givinostat	築觸顧糧築觸廠製餘構(網廠窪顧襯憲淵窪蓋糧) = reversible and manageable 蓋積範壓範構遞夢選簾 (餘膚壓鏇選鏇築積鑰獵 ) 更多	积极	2023-03-19