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]

NCT06093672

/ Recruiting临床3期

Randomized, Open-label, Multicenter Phase 3 Study to Assess the Efficacy and Safety of GIVinostat Versus Hydroxyurea IN JAK2V617F-positive High-risk Polycythemia Vera Patients: the GIV-IN PV TRIAL

The goal of this clinical trial is to compare the efficacy and safety of givinostat to hydroxyurea in Jak2V617F-positive high risk polycythemia vera patients.

开始日期2024-03-26

申办/合作机构

Italfarmaco SpA

[+1]

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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221

项与吉维诺司他相关的文献（医药）

2026-06-01·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications

Review

作者: Owens, Katie ; Yu, Jingjing ; Argon, Sophie ; Wang, Yan ; Ragueneau-Majlessi, Isabelle

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

2025-12-01·ARCHIVES DE PEDIATRIE

Corticosteroid treatment in Duchenne muscular dystrophy

Review

作者: Fontaine-Carbonnel, Stéphanie

Duchenne muscular dystrophy (DMD) is a severe degenerative disease that remains incurable. Its prognosis has been transformed by multidisciplinary care, which has significantly improved the life expectancy of patients. Corticosteroids have been used for over 20 years and have modified the care of young boys with DMD: They delay the loss of ambulation, support the gradual introduction of technical mobility aids, and reduce the frequency of scoliosis surgery. Corticoids are started and maintained at the theoretical dose of 0.75 mg/kg/day of prednisone/prednisolone or 0.9 mg/kg/day of deflazacort, adjusting the dose to weight if the benefit-risk ratio favors treatment. If introduced early enough, when muscle degeneration is not too advanced, they prolong the ability to climb stairs, rise from the floor, walk, and bring the hands to the mouth. They have been suggested to contribute to delaying the onset of respiratory failure and cardiomyopathy. The well-known side effects of corticosteroids require specific management (to prevent obesity and osteoporosis) and/or adjustment of their dosage. The only reasons to discontinue treatment when the patient and family are adhering to it are the uncontrolled side effects. Thus, in many countries including France, as of late 2025, the only routinely prescribed symptomatic treatment for DMD remains conventional corticosteroids, which will be discussed in this article, followed by a description of vamorolone, a dissociative steroidal compound, and givinostat, a histone deacetylase inhibitor.

2025-11-28·BIOLOGICAL & PHARMACEUTICAL BULLETIN

Comparative Study of Histone Deacetylase Inhibitors for Radiation Protection Using Survival Outcomes in a Mouse Model

Article

作者: Watanabe, Shinichi ; Tanaka, Mamoru ; Yano, Takaaki ; Yamaguchi, Takumi ; Hidaka, Noriaki ; Kurokawa, Yukiro

Histone deacetylases (HDACs) regulate chromatin structure and gene expression, and their inhibition has been proposed as a radioprotective strategy. However, few studies have compared multiple HDAC inhibitors (HDACis) under identical conditions. This study evaluated the efficacy of seven HDACis in a mouse total body irradiation (TBI) model. Male ICR mice received 7.5 Gy TBI followed by a single administration of valproic acid (VPA; 300 or 600 mg/kg), sodium butyrate (NaB; 500 or 1000 mg/kg), trichostatin A (TSA; 0.5 or 1.0 mg/kg), vorinostat (10 or 50 mg/kg), panobinostat (25 or 50 mg/kg), givinostat (5 or 10 mg/kg), or entinostat (25 or 50 mg/kg). Survival was monitored for 20 d. Only VPA at 600 mg/kg significantly improved survival compared with vehicle (overall p = 0.00241; p = 0.0039 vs. vehicle), while all other HDACis showed no significant benefit. VPA's efficacy may reflect a combination of effects on DNA repair, inflammation, and redox regulation rather than HDAC inhibition alone. These findings suggest VPA to be a promising candidate for radioprotection and emphasize the need for further studies to optimize dosing and explore underlying mechanisms.

100

项与吉维诺司他相关的新闻（医药）

2026-02-23

·PRNewswire

ITF Therapeutics LLC Announces Multiple Data Presentations at 2026 MDA Clinical and Scientific Conference

Presenting ten abstracts including observations on long-term safety, disease progression, and decline in muscle contractile area with givinostat Encore oral presentation also highlights analyses from the givinostat clinical development program on weight-based flexible dosing CONCORD, Mass., Feb. 23, 2026 /PRNewswire/ -- ITF Therapeutics LLC, the U.S. rare disease affiliate of Italfarmaco, today announced that one oral and nine poster presentations have been accepted at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference to be held March 8-11, 2026, in Orlando, Florida. Poster presentations will include long-term safety observations, as well as new findings on disease progression and decline in muscle contractile area in patients with Duchenne muscular dystrophy (DMD) treated with givinostat. In addition, an encore oral presentation delivered by Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital, includes analyses of functional outcomes with weight-based flexible dosing in the pivotal Phase 3 EPIDYS trial. Functional outcomes were evaluated based on time to complete the 4-stair climb test and North Star Ambulatory Assessment scores. Encore poster presentations will also cover findings related to cardiac safety data in ambulant patients from the EPIDYS study, additional insights on the simplified givinostat dosing regimen, and the characterization of thrombocytopenia and gastrointestinal adverse events. Please see the Indication and Important Safety Information for DUVYZAT® (givinostat) below. "Since the launch of ITF Therapeutics just over two years ago, our progress has been shaped by the voices and experiences of people living with Duchenne and their families whom we are privileged to serve," said Matt Trudeau, President, ITF Therapeutics. "The MDA Clinical and Scientific Conference offers an essential opportunity for us to hear directly from community members to better understand their evolving needs and priorities. At this year's meeting, we look forward to presenting new data — including a range of perspectives on the safety and efficacy of DUVYZAT — that reflect our ongoing dialogue with the Duchenne community." Encore Oral Presentation Title: Givinostat Weight-Based Flexible Dosing: Rationale and Efficacy at the Different Doses Presenter: Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital Date and time: Wednesday, March 11, 2026, at 1:30 p.m. ET De Novo P oster Presentations Title: Long-Term Safety of Givinostat in Patients with Duchenne Muscular Dystrophy: Interim Results from an Open-Label Extension Study Presenter: John F. Brandsema, M.D., Pediatric Neurologist, Division of Neurology, Children's Hospital of Philadelphia Poster number: 51S Title: Givinostat Reduces the Decline of Contractile Cross-Sectional Area and Decreases Fat Infiltration in Patients with Duchenne Muscular Dystrophy Presenter: Sara Cazzaniga, R&D Senior Manager, Neuromuscular Area, Italfarmaco Poster number: 37S Title: Open Label Extension Analysis Shows Potential Delay in Age at Loss of Ambulation in Patients with Duchenne Muscular Dystrophy Treated with Givinostat Presenter: Paolo Bettica, M.D., Ph.D., Chief Medical Officer, Italfarmaco Poster number: 64S Title: Givinostat Reprograms TGFβ-Induced Fibrotic Network in DMD Stromal Cells Presenter: Monica Forino, M.D., Molecular Biologist, Italfarmaco Poster number: 276T Encore Poster Presentations Title: A Simplified Givinostat Dosing Regimen Minimizes Exposure Differences and Dose Reductions in Higher-Weight Patients Presenter: Paolo Bettica, M.D., Ph.D., Chief Medical Officer, Italfarmaco Poster number: 36S Title: Cardiac Safety Data for Givinostat in Ambulant Patients with Duchenne Muscular Dystrophy: Results from the EPIDYS Study Presenter: Han C. Phan, M.D., Principal Investigator and Head of Research, Rare Disease Research Poster number: 42S Title: Characterizing Thrombocytopenia in Patients with Duchenne Muscular Dystrophy Treated with Givinostat: Results from the Phase 3 EPIDYS Trial Presenter: John F. Brandsema, M.D., Pediatric Neurologist, Division of Neurology, Children's Hospital of Philadelphia Poster number: 50S Title: Characterizing Platelet Count Reductions Below 75 × 109/L in Patients with Duchenne Muscular Dystrophy Treated with Givinostat from the Phase 3 EPIDYS Trial and LTSE Study Presenter: Paolo Bettica, M.D., Ph.D., Chief Medical Officer, Italfarmaco Poster number: 67S Title: Characterizing Gastrointestinal Adverse Events of Interest from a Phase 3 Study of Givinostat in Patients with Duchenne Muscular Dystrophy Presenter: John F. Brandsema, M.D., Pediatric Neurologist, Division of Neurology, Children's Hospital of Philadelphia Poster number: 49S ITF Therapeutics is also sponsoring the following educational program taking place during the conference: Industry Forum: Clinical Perspectives on HDAC Inhibition in Duchenne Muscular Dystrophy Presenters: Omer A. Abdul Hamid, M.D., Neuromuscular Specialist, Pediatric Neurology, Nemours Children's Hospital, joined by a caregiver from the DMD community Date and time: Tuesday, March 10, 2026, at 12:00-1:30 p.m. ET Room: Florida 5-7 During the forum, Dr. Hamid will share perspectives on unmet needs in the DMD treatment landscape, the potential role of histone deacetylase (HDAC) inhibition in the treatment of DMD, and expanded findings from the Phase 3 clinical trial evaluating the safety and efficacy of givinostat including the latest open-label extension data. A caregiver will also share their personal experiences with DUVYZAT. This is not an official event of the 2026 MDA Clinical and Scientific Conference. This event is not sponsored, endorsed, or accredited by MDA. About DUVYZAT® (givinostat) DUVYZAT is a U.S. FDA-approved histone deacetylase (HDAC) inhibitor indicated for the treatment of patients six years of age and older with Duchenne muscular dystrophy (DMD). The therapy was discovered through the research and development efforts of Italfarmaco in collaboration with Telethon and Duchenne Parent Project (Italy). HDACs are enzymes located in the body's cells that play a key role in maintaining and repairing muscles. In DMD, the HDAC enzymes become overactive, leading to chronic muscle inflammation, decreased muscle repair, and replacement of muscle with fat and scar tissue. DUVYZAT inhibits HDAC overactivity and is thought to help reduce inflammation, increase the body's ability to repair muscles, and slow muscle loss. For more information visit . About ITF Therapeutics LLC ITF Therapeutics was established in January 2024 as the United States affiliate of Italfarmaco Group to develop and commercialize treatments for rare diseases. The company combines end-to-end execution with close listening to learn directly from advocates, researchers, clinicians, and families, ensuring their perspectives help shape the design of innovative and potentially life-changing solutions. With deep expertise across regulatory, commercial operations, access, and community partnerships, ITF Therapeutics is focused on delivering proven therapies with urgency, ambition, and compassion to each rare disease community it serves. For more information visit . About Italfarmaco S.p.A. 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 commercialization with proven success in many therapeutic areas including immuno-oncology, gynecology, neurology, cardiovascular disease, and rare diseases. Italfarmaco's rare disease unit includes programs in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis, and polycythemia vera. For more information visit . Indication and Important Safety Information for DUVYZAT® (givinostat) INDICATION DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. IMPORTANT SAFETY INFORMATION Warnings and Precautions Hematological Changes: DUVYZAT can cause dose-related thrombocytopenia and other signs of myelosuppression. Monitor blood count every 2 weeks for the first 2 months, at month 3, and every 3 months thereafter. Modify the dosage for confirmed thrombocytopenia. Discontinuation may be needed if abnormalities worsen. Increased Triglycerides: DUVYZAT can cause elevations in triglycerides. Monitor triglycerides at 1 month, 3 months, 6 months, and then every 6 months thereafter. Modify the dosage if fasting triglycerides are verified >300 mg/dL. Treatment with DUVYZAT should be discontinued if triglycerides remain elevated despite adequate dietary intervention and dosage adjustment. Gastrointestinal Disturbances: Gastrointestinal disturbances, including diarrhea, nausea/vomiting, and abdominal pain were common adverse reactions in DUVYZAT clinical trials. Antiemetics or antidiarrheal medications may be considered during treatment with DUVYZAT. Modify the dosage of DUVYZAT in patients with moderate or severe diarrhea and discontinue treatment if significant symptoms persist. QTc Prolongation: DUVYZAT can cause prolongation of the QTc interval. Avoid use of DUVYZAT in patients who are at an increased risk for ventricular arrhythmias (including torsades de pointes), such as those with congenital long QT syndrome, coronary artery disease, electrolyte disturbance or in patients taking concomitant medicinal products known to cause QT prolongation. Obtain ECGs prior to initiating treatment with DUVYZAT in patients with underlying cardiac disease or in patients who are taking concomitant medications that cause QT prolongation. Adverse Reactions The most common adverse reactions reported in >5% of patients treated with DUVYZAT are diarrhea (37%), abdominal pain (34%), thrombocytopenia (33%), nausea/vomiting (32%), hypertriglyceridemia (23%), pyrexia (13%), myalgia (9%), rash (9%), arthralgia (8%), fatigue (8%), constipation (7%), and decreased appetite (7%). Drug Interactions Closely monitor when DUVYZAT is used in combination with an oral CYP3A4 sensitive substrate or a sensitive substrate of the OCT2 transporter, for which a small change in substrate plasma concentrations may lead to serious toxicities. Avoid concomitant use with other drugs that prolong the QTc interval; monitor ECG if concomitant use cannot be avoided. If concomitant use cannot be avoided, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold DUVYZAT if the QTc interval is >500 ms or the change from baseline is >60 ms. To report SUSPECTED ADVERSE REACTIONS, contact ITF Therapeutics LLC at 1-833-582-4312 or FDA at 1-800-FDA-1088 or . Please see full Prescribing Information for additional safety information. DUVYZAT is a registered trademark of Italfarmaco S.p.A. U.S.A. Media inquiries: Emily Steinhauer | [email protected] Other inquiries: Martha McKennitt | Communications Lead U.S.A.| [email protected] Global Media inquiries: [email protected] | +49 (0) 151 106 199 05 Other inquiries: Samantha Parker | Patient Advocacy and Communications Lead| [email protected] C-ITF-US-0022 02/2026 SOURCE ITF Therapeutics LLC 21% more press release views with Request a Demo

临床3期

2026-02-04

·类器官大师兄

肿瘤类器官：免疫治疗的新利器，从疾病建模到临床转化

免疫治疗已成为肿瘤治疗的重要方向，但肿瘤异质性导致患者治疗响应差异显著，传统 2D 细胞培养和动物模型难以模拟肿瘤微环境（TME）的复杂性，限制了免疫治疗研究的推进。肿瘤类器官作为源自患者肿瘤样本的 3D 自组装结构，能精准复刻肿瘤的组织学和遗传异质性，以及 TME 的关键特征，为解析肿瘤 - 免疫相互作用提供了生理相关性更强的研究平台。来自浙江大学、深圳大学、爱丁堡大学的团队在《Journal for ImmunoTherapy of Cancer》期刊上发表了题为Tumor organoids in immunotherapy: from disease modeling to translational research的综述，系统综述了肿瘤类器官的构建方法、特性及在肿瘤 - 免疫相互作用研究中的应用，明确其在免疫治疗药物研发、个性化治疗及克服耐药性等方面的转化潜力。实验方法 1.肿瘤类器官制备：通过机械或酶解方式解离患者肿瘤组织，获得细胞碎片或单细胞，分别采用基质胶包埋法、微流控芯片法、气液界面法或 3D 生物打印法进行培养，添加 EGF、FGF 等生长因子维持类器官生长和干细胞特性。 2.类器官功能表征：运用活体成像（如高内涵成像）和多光谱成像观察类器官动态变化；结合流式细胞术、质谱流式、单细胞 RNA 测序、TCR 测序及空间转录组学等技术，解析类器官的基因表达、免疫细胞组成及异质性特征。 3.肿瘤 - 免疫相互作用模型构建：将肿瘤类器官与免疫细胞（如 CD8+T 细胞、NK 细胞、PBMCs）或基质细胞（如 CAFs）共培养，模拟 TME 中的细胞相互作用；通过基因编辑技术（如 CRISPR-Cas9）修饰类器官基因，探究关键分子在免疫逃逸、免疫抑制中的作用。 4.免疫治疗相关实验：在类器官模型中测试免疫检查点抑制剂、CAR-T 细胞、新抗原疫苗、双特异性抗体等免疫治疗方法的 efficacy；开展高通量药物筛选，优化联合治疗方案；结合临床样本验证类器官预测患者治疗响应的准确性。 5.临床转化验证：建立肿瘤类器官生物样本库，关联患者临床数据，评估类器官指导个性化治疗的效果；开展临床 trials，验证类器官在免疫治疗方案选择中的实用性。关键结果图.1：肿瘤类器官的培养方法与分析技术基质胶包埋法操作简便，可快速获得肿瘤类器官，但对 TME 模拟不完全；微流控芯片法通过流动介质模拟血流，支持血管化类器官构建，适用于高通量药物筛选；气液界面法能保留免疫细胞和基质细胞，适合肺、表皮等需气体交换的组织类器官培养；3D 生物打印法可精准控制细胞空间分布，构建结构复杂的类器官。成像技术中，活体成像可实时观察 T 细胞浸润等动态过程，多光谱成像能识别类器官内不同细胞亚群；单细胞分析技术（如 CyTOF、scRNA-seq）可深度解析免疫细胞表型和基因表达特征，为肿瘤 - 免疫相互作用研究提供高分辨率数据。图.2：肿瘤类器官模拟肿瘤微环境及肿瘤 - 免疫相互作用肿瘤类器官可有效复刻 TME 的异质性，与 CAFs 共培养后，结直肠癌和胰腺癌类器官的增殖能力增强、药物耐药性提升，重现患者肿瘤的病理特征；通过引入内皮细胞和间充质祖细胞，成功构建血管化类器官，对血管内皮生长因子受体靶向药物和缺氧环境具有响应性。在免疫相互作用研究中，类器官与 CD8+T 细胞共培养可模拟 MHC - 肽介导的 T 细胞激活，NK 细胞能通过肿瘤细胞表面应激配体被激活；基因修饰类器官可分泌 CXCL9、CXCL10 等趋化因子，介导 T 细胞募集；黑色素瘤类器官与 TILs 共培养时，IL-2 和 αPD-1 刺激可最大化 CD8+T 细胞扩增，且能评估 TIL 的迁移和细胞毒性。同时，类器官可模拟免疫抑制微环境，如 HER2 过表达会上调 PD-L1 表达，促进肿瘤免疫逃逸，敲低 HER2 后可增强免疫检查点抑制剂敏感性；肿瘤类器官分泌的 GM-CSF 能诱导 MDSCs 扩增，抑制 T 细胞功能，且可观察到 T 细胞耗竭、巨噬细胞 M2 极化等免疫抑制表型。图.3：肿瘤类器官在免疫治疗研究中的应用免疫治疗方法验证中，气液界面法培养的类器官与 TILs 共培养，经 PD-1/PD-L1 抗体激活后可诱导肿瘤细胞杀伤；CAR-T 细胞在表达 HER2、EGFR 等靶点的类器官模型中，能展现特异性细胞毒性，MesoFAP CAR-TEAM 细胞对胰腺癌类器官的杀伤效果显著；新抗原刺激的 CD8+T 细胞在肝胆肿瘤类器官中表现出增强的肿瘤杀伤作用，低新抗原表达的结直肠癌类器官经新抗原疫苗联合免疫检查点抑制剂治疗后，免疫逃逸可被逆转。药物发现方面，BEHAV3D 系统可追踪类器官中 T 细胞行为，助力免疫治疗药物筛选；胰腺癌类器官模型筛选出 ITF2357 与 I- BET151 联合抗 PD-1 疗法，能显著增强 T 细胞毒性。个性化治疗中，膀胱癌类器官生物样本库的药物响应与患者临床结果高度吻合；结直肠癌类器官中 DKK1 高表达和 NLR 高表达分别与 PD-1 抑制剂耐药相关，可作为生物标志物指导治疗方案选择。此外，类器官可用于疾病建模、细胞毒性研究，结合 CRISPR-Cas9 技术能精准探究肿瘤发生、耐药相关基因功能，为联合治疗方案优化和耐药机制研究提供可靠平台。全文总结肿瘤异质性和 TME 复杂性是免疫治疗研究的主要挑战，传统模型难以满足临床转化需求。本研究系统阐述了肿瘤类器官的多种构建方法（基质胶包埋、微流控、3D 生物打印等）及其在复刻肿瘤异质性和 TME 方面的优势，通过与免疫细胞、基质细胞共培养及基因编辑修饰，类器官可精准模拟肿瘤 - 免疫相互作用，包括免疫激活、细胞毒性、免疫抑制和免疫逃逸等关键过程。在应用层面，肿瘤类器官已成功用于免疫治疗药物筛选、个性化治疗预测、疾病建模和耐药机制研究，多项临床案例验证了其指导治疗方案选择的有效性。尽管目前仍面临 TME 模拟不完全、培养方案标准化不足、伦理规范待完善等挑战，但随着血管化类器官构建、多组学分析等技术的发展，肿瘤类器官有望成为连接基础研究与临床应用的关键工具，推动免疫治疗向精准化、个性化方向发展，为改善患者预后提供新路径。参考文献 Qi Si, Shuhan Tao, Jizhun Wu, et al. Tumor organoids in immunotherapy: from disease modeling to translational research[J]. Journal for ImmunoTherapy of Cancer, 2025, 13:e011733. doi:10.1136/jitc-2025-011733

免疫疗法疫苗细胞疗法

2026-01-31

·肌ying养不良

紧跟 2026 步伐！新上市药物开启肌营养不良治疗新时代

点击蓝字关注我们肌营养不良是一组遗传性肌肉疾病，主要表现为进行性的肌肉无力和萎缩。长期以来，这种疾病的治疗手段相对有限，但随着医学研究的不断进步，2026年，多款创新药物陆续上市，为患者带来了新的希望。本文将详细介绍这些新药的特点、作用机制以及临床应用前景。一、吉维诺司他（Givinostat）：开创非激素治疗新时代图 01 吉维诺司他吉维诺司他是一种口服组蛋白脱乙酰酶抑制剂，于2024年首次在美国获得批准用于治疗杜氏肌营养不良症（DMD），并在2026年进一步扩展其适应症范围至其他类型的肌营养不良。该药物通过调节基因表达，促进肌肉纤维再生，并减少炎症反应。在为期两年的跨国III期EPIDYS试验中，吉维诺司他显著减缓了患者的运动功能下降速度，改善了生活质量。特点与优势非类固醇药物：避免了长期使用激素带来的副作用。广泛适用性：适用于多种基因突变导致的肌营养不良。易于服用：每日两次口服悬浮液，方便患者日常管理。二、DYNE-251：突破性疗法重新定义DMD治疗 DYNE-251是Dyne Therapeutics公司开发的一种外显子跳跃疗法，专为适合外显子51跳跃的杜氏肌营养不良症患者设计。该药物通过特异性地靶向外显子51，促使肌营养不良蛋白mRNA的正常剪接，从而生成近乎全长的功能性肌营养不良蛋白。DYNE-251已获得美国FDA授予的突破性疗法称号，并在正在进行的DELIVER临床试验中显示出显著的行走能力改善效果。特点与优势精准靶向：针对特定基因突变，提高治疗效果。快速通道：FDA快速通道、孤儿药及罕见儿科疾病认定加速审批过程。长期疗效：旨在产生持久的肌营养不良蛋白，改善功能。三、安迦利®（伐莫洛龙口服混悬液）：中国首款获批DMD治疗药物图 02 伐莫洛龙安迦利®（通用名：伐莫洛龙口服混悬液）是中国曙方医药于2024年底推出的一款创新药物，专门用于治疗四岁及以上的杜氏肌营养不良症患者。伐莫洛龙通过抑制炎症反应和保护肌肉细胞，延缓病情进展。该药物在中国国家药品监督管理局（NMPA）的批准下，成为国内首个获批上市的DMD治疗药物，填补了国内市场的空白。特点与优势本土创新：中国自主研发，更适合中国患者。安全性高：多项临床试验证明其良好的安全性和耐受性。便捷给药：口服混悬液形式，便于儿童患者服用。四、辅酶Q10软胶囊与维生素E：基础营养支持的重要性图 03 维生素E 虽然辅酶Q10软胶囊和维生素E并非新上市药物，但它们在肌营养不良治疗中的重要性不容忽视。辅酶Q10作为一种脂溶性抗氧化剂，能够激活人体细胞能量，增强肌肉活力；而维生素E则能促进脑细胞的新陈代谢，改善血液循环，对维持肌肉健康具有积极作用。这两种补充剂通常作为辅助治疗手段，帮助患者缓解症状，提高生活质量。特点与优势天然成分：基于天然维生素E和辅酶Q10，安全性高。多重功效：不仅限于肌肉保护，还能改善整体健康状况。易获取：无需处方即可购买，便于患者长期使用。五、未来展望：干细胞与基因编辑技术的潜力除了上述已经上市或即将上市的新药，干细胞治疗和基因编辑技术也在肌营养不良领域展现出巨大潜力。例如，信念医药集团研发的BBM-D101注射液，利用工程化衣壳提高AAV基因治疗的肌肉组织递送效率，已在2025年初获美国FDA批准进入临床试验阶段。此外，士泽生物医药有限公司的人异体通用“现货型”神经前体细胞注射液XS-228也获得了中国IND受理，有望在未来几年内为渐冻症等神经系统疾病提供新的治疗选择。特点与优势根本性治疗：直接修复受损基因或替换病变细胞，实现根治可能。个体化定制：根据患者具体情况制定个性化治疗方案。长效作用：一次治疗可能带来长期甚至终生的效果。 2026年，随着吉维诺司他、DYNE-251、安迦利®等创新药物的上市，肌营养不良的治疗迎来了前所未有的机遇。这些新药不仅提高了治疗效果，还减少了传统治疗方法的副作用，为患者提供了更多选择。同时，干细胞与基因编辑技术的发展也为未来的治疗开辟了新的方向。我们有理由相信，在科学家和医疗工作者的共同努力下，肌营养不良这一顽疾终将被攻克，患者的生活质量将得到显著提升。六、确诊肌营养不良如何治疗？图 04 王世龙中医师中医治疗肌营养不良有其独特的优势。王世龙医师采用中西医结合的综合疗法，其中核心是中药治疗。他会根据您的具体病情和体质，精心选配多种中草药，通过调整人体的气血、阴阳平衡，达到改善病情、缓解症状的目的。比如他的“五龙荣肌汤”，是他凭借多年临床经验和深厚的中医理论功底调配出来的，很多患者服用后症状都有明显改善，而且中药的副作用相对较小。在西药治疗效果不稳定、副作用大的情况下，中医是一个很好的选择。王世龙中医师指出："中医调理肌营养不良必须遵循'辨证论治'原则，每位患者的体质和症状表现各不相同，必须通过详细问诊和舌脉辨证，才能制定个性化的调理方案。我们采用'一人一方'精准治疗模式，根据患者具体证型（如肝肾阴虚、气血两虚等）配伍相应中药。现提供两种就诊方式：1.北京面诊（可现场把脉问诊）；2.远程视频看诊（适合异地患者），两种方式均能提供精准、有效的治疗。” 扫描二维码咨询治疗微信号 : 18534938512

突破性疗法信使RNA孤儿药快速通道加速审批

100 项与吉维诺司他相关的药物交易

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外链

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

研发状态

批准上市

10 条最早获批的记录,

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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

未上市

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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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适应症

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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


NCT06093672 (ASH2025)	临床3期	真性红细胞增多症	904	Givinostat	積糧鏇夢糧淵製齋鏇構(憲簾選鹹積網夢糧築願) = 淵鏇獵選蓋夢窪淵範膚憲淵構廠鹹積夢簾選構 (淵衊淵製夢鏇衊獵獵齋 )	积极	2025-12-06
NCT06093672 (GIV-IN-PV, ASH2025)	临床3期	真性红细胞增多症 JAK2V617F mutation	220	Givinostat	選淵簾鹹淵餘鏇膚淵網(蓋淵獵齋壓襯積繭膚積) = 網製積憲醖鏇淵廠襯簾壓網憲網鏇觸簾簾遞窪 (壓廠衊鏇鏇鏇積網顧夢 ) 更多	积极	2025-12-06
				Hydroxyurea	艱鹽膚築醖選構繭築鹽(鹹鬱構艱顧簾鬱衊構夢) = 積構齋鑰糧繭願網繭簾淵鏇餘鹹遞選壓餘觸醖 (餘淵製窪繭網築窪顧鬱 ) 更多
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	範繭襯網蓋繭憲膚淵簾(鬱餘遞醖繭願糧糧鬱憲) = 繭積齋膚糧繭壓構構醖糧製繭衊憲糧憲醖鬱構 (鹹醖醖廠鹹蓋憲齋觸觸 )	积极	2025-11-01
NCT02851797 (EPIDYS, GlobeNewswire) 人工标引	临床3期	杜氏肌营养不良症	179	Givinostat	淵夢齋蓋廠鬱鹽鬱鑰鏇(衊築遞鹽鬱觸繭艱衊築) = 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. 齋鏇膚壓鹹簾簾鏇觸窪 (築窪範網遞壓淵築鹹築 ) 更多	积极	2025-06-11
NCT02851797 (EPIDYS, MDA2025)	临床3期	杜氏肌营养不良症	179	Givinostat (off-target group)	憲壓願鑰鏇衊製構淵繭(鬱顧鑰鹹觸壓遞選積糧) = 願膚獵窪壓簾顧憲選鹽觸鑰膚艱鹹壓鑰積築襯 (簾鹹廠觸襯範網窪壓襯, 7.9)	积极	2025-03-16
				Placebo (off-target group)	憲壓願鑰鏇衊製構淵繭(鬱顧鑰鹹觸壓遞選積糧) = 蓋積鏇築鑰夢廠醖繭襯觸鑰膚艱鹹壓鑰積築襯 (簾鹹廠觸襯範網窪壓襯, 13.3)
NCT03373968 (MDA2025)	临床2/3期	杜氏肌营养不良症	194	Givinostat	鏇範鬱鏇憲構窪願齋範(鹽襯廠鹹壓窪選膚夢蓋) = 憲糧憲範鹽網夢顧觸淵淵築襯願醖糧製夢夢簾 (憲膚鹽餘齋觸範艱顧憲 ) 更多	积极	2025-03-16
				Givinostat (Prior Placebo)	鏇範鬱鏇憲構窪願齋範(鹽襯廠鹹壓窪選膚夢蓋) = 淵憲構憲衊網繭願築艱淵築襯願醖糧製夢夢簾 (憲膚鹽餘齋觸範艱顧憲 ) 更多
NCT02851797 (EPIDYS, MDA2025)	临床3期	杜氏肌营养不良症	284	Givinostat	網醖齋襯廠積獵網製夢(淵夢鬱齋蓋夢醖願壓鹽) = 糧艱願衊鬱蓋襯鏇網觸醖窪觸願壓餘齋繭簾餘 (糧遞廠艱衊製壓餘淵衊 ) 更多	积极	2025-03-16
				Steroids	網醖齋襯廠積獵網製夢(淵夢鬱齋蓋夢醖願壓鹽) = 夢憲構簾醖蓋網齋蓋顧醖窪觸願壓餘齋繭簾餘 (糧遞廠艱衊製壓餘淵衊 ) 更多
NCT02851797 (EPIDYS, MDA2025)	临床3期	杜氏肌营养不良症	56	Givinostat in addition to steroids	膚壓鹽蓋鹽壓簾願鏇製(艱餘顧餘範艱鬱夢糧餘) = 淵鑰願艱壓憲簾鹹鹹鑰觸憲範襯構願築鑰齋鏇 (願艱選艱顧獵醖鏇壓糧, 2.2) 更多	积极	2025-03-16
				Steroids only (PRO-DMD-01)	膚壓鹽蓋鹽壓簾願鏇製(襯衊積齋夢衊鏇網獵獵) = 膚齋膚壓製範繭觸窪選蓋蓋憲繭壓構構觸壓願 (鏇顧衊衊膚製鹹衊願網, 2.2) 更多
NCT05860114 (CTgov)	临床1期	药物相互作用	8	Givinostat (Givinostat)	鬱艱製衊網艱選範製顧(繭艱淵艱觸顧獵觸齋淵) = 艱簾艱築繭衊願糧醖鬱糧網蓋艱範襯餘糧鹹餘 (製積構簾網範選鏇網願, 25.8) 更多	-	2025-01-24
				Givinostat (ITF2374)	鬱艱製衊網艱選範製顧(繭艱淵艱觸顧獵觸齋淵) = 壓製鬱網艱鏇憲憲餘構糧網蓋艱範襯餘糧鹹餘 (製積構簾網範選鏇網願, 45.7) 更多
NCT03238235 (CTgov)	临床2期	贝克型肌营养不良	51	Givinostat (Givinostat)	鬱遞餘夢範艱膚壓淵顧(獵網壓網製選糧廠憲糧) = 構範鑰淵積顧鏇繭夢鑰顧夢製夢網鬱衊鬱遞願 (選網顧製積積遞憲鹽鹽, 醖鹽窪壓窪鑰遞鬱鑰繭 ~ 遞鏇壓醖鏇齋製鹽鹽鹽) 更多	-	2024-11-18
				Placebo (Placebo)	鬱遞餘夢範艱膚壓淵顧(獵網壓網製選糧廠憲糧) = 餘獵淵獵齋膚窪壓廠簾顧夢製夢網鬱衊鬱遞願 (選網顧製積積遞憲鹽鹽, 艱餘夢遞蓋觸鑰鑰餘構 ~ 窪糧選鑰築衊鏇鬱糧憲) 更多