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 was 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2025-05-29

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]

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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260

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 ; Argon, Sophie ; Yu, Jingjing ; Ragueneau-Majlessi, Isabelle ; Wang, Yan

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.

2026-06-01·BIOCHEMICAL PHARMACOLOGY

Corrigendum to “Givinostat rescues folding of cystathionine beta-synthase and ameliorates murine homocystinuria” [Biochem. Pharmacol. 239 (2025) 117079]

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

2026-04-01·JOURNAL OF MEDICAL GENETICS

Targeting autophagy in Duchenne muscular dystrophy: mechanistic insights and emerging therapeutic strategies

Review

作者: Karnik, Medha ; Anand, Rhea ; Prashant, Akila ; Dharmashekar, Chandan ; Rao, Anagha ; Kumar Honnavalli, Yogish ; Krishna, Lakshmi ; Vishwanath, Prashant ; Srivathsa, Ananyashree

Duchenne muscular dystrophy (DMD) is a severe X-linked myopathy characterised by progressive skeletal and cardiac muscle degeneration, loss of ambulation, respiratory failure and premature mortality. Although corticosteroids and gene therapies have improved disease management, they are limited by significant side effects, mutation specificity and delivery challenges, underscoring the need for an alternative or an adjunctive strategy. Emerging evidence identifies autophagy dysregulation as a critical secondary pathological mechanism in DMD, contributing to impaired clearance of damaged organelles and toxic protein aggregates, exacerbating muscle atrophy and fibrosis.This review aims to acknowledge current insights into autophagy regulation in healthy muscle and its disruption in DMD, explore its crosstalk with key pathological pathways such as nuclear factor kappa B signalling, mitochondrial dysfunction and endoplasmic reticulum stress and critically evaluate emerging therapeutic strategies targeting autophagy.Autophagy, a fundamental cellular recycling process, is suppressed in DMD by hyperactivation of the Akt-mTOR pathway and dysregulated calcium homeostasis. This leads to mitochondrial dysfunction, oxidative stress and activation of inflammatory cascades. Recent preclinical studies highlight the therapeutic potential of pharmacological and dietary autophagy modulators, including rapamycin, 5-aminoimidazole-4-carboxamide ribonucleotide, low protein diets, SRT2104 and Givinostat, which improve autophagic flux, restore mitochondrial integrity and attenuate fibrosis. Lifestyle interventions and combinatorial approaches further underscore the importance of integrating multimodal strategies.Further research should focus on longitudinal studies to optimise therapeutic timing, validate dynamic biomarkers (LC-II, p62, miRNAs) and leverage artificial intelligence with multiomics integration for precision therapies. Targeting autophagy and its interconnected pathways holds promise for transforming DMD management and improving patient outcomes.

119

项与吉维诺司他相关的新闻（医药）

2026-09-02

·必诺达-注册通

第 006 期新药批准深度解读：Mimrylo（rusfertide） ——首款铁调素模拟肽改写真性红细胞增多症治疗范式

北京必诺达 · 注册科学观察 / 栏目二 / 重点药品 / 第 006 期新药批准深度解读：Mimrylo（rusfertide）——首款铁调素模拟肽改写真性红细胞增多症治疗范式 FDA 2026-08-28/31 批准 · 成人真性红细胞增多症（PV）· first-in-class 铁调素模拟肽 2026-09-02 · 整理：北京必诺达生物医药科技有限公司本文为"重点药品"专栏。Mimrylo（rusfertide）由 Takeda 与 Protagonist Therapeutics 联合开发，于 2026-08-28/31 获 FDA 批准，是首个模拟铁调素（hepcidin）的肽类疗法，用于现有治疗未能充分控制的成人真性红细胞增多症（PV）。以下从机制、数据、CMC、监管、竞争、中国策略与解读边界等维度展开，所有数值均来自 FDA 新闻稿、企业新闻稿与权威血液学媒体（A/B 级来源），缺失项一律标注"资料未涉及"。一、药物与机制定位 Mimrylo（通用名 rusfertide）是一款 first-in-class 的铁调素模拟肽（hepcidin mimetic peptide），由 Takeda 与 Protagonist Therapeutics 联合开发。它的核心机制并非直接杀伤克隆性造血，而是"绕道"——通过模拟人体天然的铁调素（hepcidin）来调节铁稳态：限制可用于红细胞生成的铁供给，从而抑制红细胞过度增殖、把红细胞比容（Hct）稳定控制在目标范围。 PV 是一种 BCR-ABL1 阴性的骨髓增殖性肿瘤，特征为红细胞过度生成，使血液黏稠度升高、血栓与心血管事件风险增加。JAK2 突变见于近 100% 的 PV 患者，是疾病驱动的核心分子事件。现行治疗的核心目标，是把 Hct 维持在 45% 以下以降低血栓风险，而这一目标的实现长期高度依赖放血（phlebotomy）——一种需反复就医、耐受性差且常被现实世界研究证明难以稳定达标的手段。 rusfertide 的机制意义，正在于它首次提供了"针对红细胞过度增殖本身"而非"压制克隆"的特异性疗法：通过铁稳态这一上游开关，从根源减少红细胞生成需求，从而有望把患者从反复放血的循环中解放出来。这一点与 JAK 抑制剂、干扰素等"广谱抑制造血/信号"的思路形成机制层级上的根本差异。二、关键临床试验与人群支持获批的关键研究为 VERIFY（NCT05210790）：一项多中心、随机、双盲、安慰剂对照的 III 期研究，纳入 293 例尽管接受标准治疗仍依赖频繁放血的 PV 成人。患者按 1:1 随机接受 rusfertide 或安慰剂，观察 32 周。研究人群中位年龄 57 岁、73% 为男性，约 56% 在试验期间合并细胞减灭治疗（如羟基脲、干扰素或 ruxolitinib），说明入组的是现实世界中"标准治疗已用上、但仍未控"的难治人群。 rusfertide 起始剂量 19 mg、皮下注射、每周一次，并按需滴定以维持 Hct<45%。研究的主要终点定义为 20–32 周期间"无放血资格"（phlebotomy eligibility，即未达需放血标准）的应答比例；放血资格本身被严格界定为 Hct≥45% 且较基线升高≥3%，或 Hct≥48%。这一终点设计直接锚定 PV 的治疗目标（控 Hct、减放血），而非替代性血液学指标，使临床获益与监管判定高度一致。除 VERIFY 外，证据包还整合了 II 期 REVIVE（NCT04057040）与长期延展 THRIVE（NCT06033586）的数据，提供了从剂量探索、确证到最长 196 周真实暴露的连续性证据链。三、疗效数据（关键终点与统计口径）下表汇总已公开的核心疗效终点。需说明统计口径：主要终点"临床应答"定义为 20–32 周无放血资格，属二分类应答（非生存类终点），其显著性检验为组间比较（p<0.0001）；Hct 维持率同理。各数值均为已发表顶线/最终分析结果，置信区间资料未涉及。终点指标 rusfertide 组安慰剂+标准治疗组统计学含义临床应答（20–32 周无放血资格） 76.9% 32.9% p<0.0001 0–32 周平均放血次数 0.5 次 1.8 次 p<0.0001 维持 Hct<45% 的患者比例 62.6% 14.4% p<0.0001 需接受放血的患者比例 27% 78% 放血负担大幅下降 52 周持久临床应答 61.9% — 应答具持久性 REVIVE：无需治疗性放血的 Hct 控制 69.2% 14.8% p<0.0001 统计口径说明：上述疗效指标为应答率/均值比较，未以风险比（HR）或中位生存形式报告（与肿瘤药不同，PV 为慢性非恶性血液病，主要终点为放血依赖的消除）。样本量、随访时长与截点日期资料未涉及，故不举例外推算。临床意义：在 PV 这类"控 Hct、减放血"即等于改善预后与生活质量的开销型慢病中，应答率从约三分之一翻倍至近四分之三、平均放血次数从 1.8 降至 0.5、仅 27% 患者仍需放血——意味着绝大多数患者有望摆脱反复就医放血的负担。更值得注意的是，应答在合并/不合并细胞减灭治疗、高/低风险患者中一致出现，提示其获益不依赖于既有治疗背景，具有 broad-spectrum 适用潜力。四、安全性谱、停药率与重要警示 VERIFY 中 rusfertide 总体耐受性良好：最常见治疗期不良事件为注射部位反应（47.4%）、贫血（25.6%）与疲劳（19.6%），多为 1–2 级；严重不良事件发生率 8.1%。II 期 REVIVE 中 3 级治疗期不良事件为 25.7%，严重不良事件 3.4% 对 4.8%（安慰剂），且长期延展 THRIVE 中未见判定与药物相关的严重不良事件。需提示的重要警示：① 贫血（25.6%）是铁限制疗法的机制性预期事件，临床需监测血红蛋白并适时调整剂量/输血策略；② 注射部位反应虽高发（长期数据达 87%，多为 1–2 级）但多为自限性，与每周皮下给药方式相关；③ 作为铁调素模拟剂，其安全性信号集中于铁代谢相关（铁蛋白、转铁蛋白饱和度、血红蛋白），具体黑框警告与重点关注信号以 FDA 说明书为准（资料未涉及）。停药率解读：需明确，本品种公开数据未以"因不良事件停药率"作为主要安全性指标报告（不同于肿瘤药常规）。其安全性结论以"多为低级别、无相关严重不良事件"表述，提示长期用药的中断风险可控；但确切停药率数值资料未涉及，不应据推测填入。五、CMC 与药学特征（仅锚定已公开信息）已公开的药学信息仅为：皮下注射肽类、每周一次自我注射、起始 19 mg 并按需滴定至维持 Hct<45%。以下常被问及的药学维度，证据层均无数据，依规标注为"资料未涉及"，不据推测：处方组成、缓冲体系、冻干/液体剂型与稳定性数据 ——资料未涉及；原料药合成路线、纯度谱、有关物质与工艺验证细节 ——资料未涉及；药代动力学（吸收、分布、代谢、排泄）、蛋白结合率、半衰期——资料未涉及；与铁剂、其他造血调节药物的相互作用 ——资料未涉及。对国内同类的启示：作为肽类皮下注射剂，若国内企业以 rusfertide 为对标开发同类铁调素模拟肽，CMC 文件应把"每周给药的暴露-效应关系""滴定方案的剂量-Hct 响应模型""肽类原料药工艺与杂质控制""长期稳定性与给药便利性（自我注射）"作为申报核心。需特别强调：境外已获批绝不意味着其 CMC 证据已"完备"——制造挑战本身被列为该品种的商业化风险点，国内若技术引进或对标开发，须把完整的处方工艺、质量标准与稳定性研究作为尽调与申报的第一优先级，不能因境外先批而默认其药学表征齐全。六、监管路径与全球同步性 FDA 于 2026-08-28/31 以标准批准（非加速批准）放行 Mimrylo，并曾授予 Priority Review、Breakthrough Therapy、Orphan Drug 与 Fast Track 四项资格。值得注意，尽管拥有四项加速/激励资格，最终仍走的是基于 VERIFY 主要终点（放血依赖消除）的完整批准路径——这反映出监管对该终点临床意义的认可，也提示同类品种在 PV 这类以"治疗负担消除"为核心获益的慢病中，可用替代终点支撑常规批准。对国内注册而言，rusfertide 的获批提供了"铁稳态新机制 + 慢性血液病 + 以治疗负担为终点"的参照范式：国产同类若计划中美双报，应提前规划"境外关键试验 + 中国桥接/验证"的证据组合，而非等到境外获批后再单独设计国内研究。监管沟通的前移，是把"全球同步开发"落到申报资料的具体动作。七、竞争格局与国内外竞品进度 PV 已上市治疗包括羟基脲、阿那格雷、JAK 抑制剂 ruxolitinib（Jakafi）以及长效干扰素 ropeginterferon alfa-2b（Besremi，本观察周期亦获 FDA 扩展批准用于 ET）。rusfertide 的差异化在于机制层级：它不抑制 JAK-STAT 信号、也不直接压制克隆，而是从铁供给这一上游开关切入，专门解决"红细胞过度增殖 + 放血依赖"这一 PV 最核心的治疗痛点。但随着 rusfertide 首批落地，国内外竞品管线已全面提速，竞争窗口正在快速收窄。国际在研竞品进度：竞品（企业）机制/给药全球最高阶段关键数据与下一步 divesiran（Silence Therapeutics） siRNA 沉默 TMPRSS6、上调内源 hepcidin；皮下 Q6W/Q12W II 期完成（SANRECO，48 例）应答 88% vs 19%（p<0.0001）；超 90% 患者全程免放血 vs 安慰剂 12.5%；计划 2027 上半年启动 Q12W III 期 bomedemstat / MK-3543（Merck/Imago）口服 LSD1 抑制剂 II 期完成（开放标签，20 例） 36 周 Hct 控制不伴放血为主要终点之一；数据已于 2025 年 12 月 ASH 年会公布 idasanutlin / givinostat 等 MDM2 抑制剂 / HDAC 抑制剂早期临床研究 idasanutlin 显示降低 JAK2 V617F 突变负荷但胃肠道反应常见；givinostat 单药/联用羟基脲显示疗效与长期安全性其中对 rusfertide 威胁最直接的是 divesiran：SANRECO II 期应答率 88%（Q6W 组 93.8%、Q12W 组 81.3%）、人均放血 0.2 次 vs 安慰剂 2.1 次，且仅 2 例 1 级贫血、无新安全性信号；公司同期完成 1.75 亿美元融资，计划 2026 年底前完成 FDA 沟通、2027 上半年启动每 12 周给药一次的 III 期，完整数据拟于 2026 年 12 月 ASH 发布。若 III 期维持疗效，divesiran 凭借"每季给药"便利性，将对 rusfertide 的"每周皮下注射"形成直接给药频次竞争；而口服的 bomedemstat 若在更大样本中确证，则可能在给药便利性上再下一城。国内竞品进度：竞品（企业）机制国内进度对 rusfertide 的意义邦瑞替尼 / OB-756（杭州邦顺制药/广良医药） JAK2 抑制剂 2026-04-29 已在中国获批（MF/PV 后 MF/ET 后 MF）；羟基脲耐药/不耐受 PV 的 III 期 BEWELL-302 进行中若 BEWELL-302 阳性，将率先卡位国内"PV 二线"市场马来酸氟诺替尼 / FM（成都赜灵生物） JAK2/FLT3/CDK6 三靶点抑制剂 2026-01 获 CDE 突破性治疗认定（适应症含 PV）；PV II 期已启动 BTD 加持下 PV 适应症开发提速，国内 PV 治疗进入多靶点竞争 XH-5102（上海勋和医药） JAK2 抑制剂 MPN（含 PV）I/II 期招募中早期梯队，尚不足以构成近期威胁竞争格局解读：国内管线集中于 JAK2 通路（信号抑制路线），与 rusfertide 的铁稳态路线、divesiran 的 siRNA 路线形成三条并行的机制赛道——这意味着国内"跟随式"立项尚未进入铁-hepcidin 轴这一新机制层面。对国内企业而言，立项 iron-hepcidin 轴药物时需尽早明确自身是在"肽类每周给药"还是"核酸类低频给药"路线上差异化——两条路线的 CMC、给药便利性与专利布局截然不同；而邦瑞替尼 PV III 期的推进速度，也将决定 rusfertide 类品种进入中国时面对的是"空白市场"还是"JAK2 先行"的格局。八、中国注册策略要点境外数据接受 PV 为罕见/孤儿病种，中国已建立境外已上市药品境内外数据接受的沟通机制。rusfertide 类的 PV 关键试验若以全球多中心形式纳入中国受试者，可争取以境外数据为主、境内数据为辅的支持路径；但需确认中国亚组样本量与代表性是否满足技术指导原则对"种族因素"的要求。桥接若境外试验未充分纳入中国人群，应规划正式桥接研究——以 PK/PD 桥接为主、必要时补充小样本疗效验证，而非简单沿用境外剂量。需明确桥接的统计学接受标准（等效/非劣的界值）并在沟通会上与 CDE 锁定。监测与终点取舍（本药无强制伴随诊断）与 RAS 抑制剂不同，rusfertide 不依赖某一驱动突变作为用药前提（PV 的 JAK2 V617F 为诊断性而非预测性标志物），因此无强制伴随诊断。真正的"取舍"在于终点与监测策略：以 Hct<45% 为治疗目标、以"放血资格"为替代终点，这一终点设计能否被 CDE 接受为关键获益证据，以及长期铁代谢指标（铁蛋白、转铁蛋白饱和度）的监测方案如何设定，需在沟通中前置锁定。绿色通道对标对照 CDE"关爱计划-延伸"与"先锐计划"等加速通道，评估 rusfertide 类品种是否满足罕见/难治、临床急需的纳入标准；若满足，应在早期即提交试点申请，把沟通节点前移，避免重复境外已解决的 CMC 与临床设计问题。九、解读边界本文所有数值来自 FDA/企业/权威血液学媒体公开报道，未获原始临床研究报告（CSR），不替代说明书与监管文件。本品种疗效以"放血依赖消除/ Hct 控制"等二分类或均值终点报告，未以 HR 或中位生存形式呈现（与肿瘤药不同），解读时不应套用生存类统计框架。安全性具体不良事件谱、分级与发生率资料未涉及完整清单，不以推测补充；确切停药率数值资料未涉及。 CMC 维度中处方工艺、稳定性、PK、相互作用均为资料未涉及项，不得据境外未载信息反推写入申报或解读。中国亚组的具体疗效与 PK 数据资料未涉及，境内桥接的必要性须以实际纳入情况判定。竞争格局与国内外竞品进度基于已上市/公开在研信息（含 divesiran、bomedemstat II 期数据与国内 JAK2 管线公开登记信息），不预示未来审批或商业结果。药学数据缺口（重点）：该品种公开信息中 CMC 披露极为有限（仅给药途径、频次与起始剂量），国内若开展对标开发或技术引进，须把完整的药学表征（处方工艺、质量标准、稳定性、暴露-效应与给药便利性）作为尽调与申报的第一优先级，不能因境外已获批而默认其 CMC 证据完备。来源：FDA 新闻稿（A）、Takeda/Protagonist 新闻稿（B）、MPN Hub / Pharmacy Times / FirstWord / CancerNetwork（B）、Silence Therapeutics SEC 公告（A）、ClinicalTrials.gov（A）、智慧芽新药情报库（B）。本期整理：北京必诺达生物医药科技有限公司。事实核查：PASS（未溯源数值：无）。

2026-08-30

·地平汪style

DMD/BMD最新行业资讯｜国产疗法迎来临床突破，全球新药研发多点开花

对于杜氏与贝氏肌营养不良（DMD/BMD）患者家庭而言，每一项临床研究的进展，每一款新药的动态，都承载着沉甸甸的希望。2026年，国内外在基因编辑、RNA编辑、创新药物、综合康复管理多个赛道捷报频传，一大批国产原创技术走到临床台前，全球管线也持续迎来新的审批与试验节点。 DMD和BMD同属于假性肥大型肌营养不良，绝大多数发病于男性儿童。DMD一般在2‑5岁起病，病情进展较快，大多在13岁前后运动能力出现明显衰退；BMD发病时间更晚，病程进展相对缓和，不少患者可以保留行走能力直至成年，但心肌病是BMD患者最主要的健康威胁。二者的干预方向也有所区别，DMD更多依靠药物联合、RNA以及基因治疗，BMD则把心脏保护、科学康复放在管理的核心位置。在基因治疗与RNA编辑领域，国产原创技术取得了令人振奋的实质性突破。我国科研团队自主开发的LEAPER RNA编辑技术已经进入临床阶段，该技术能够促使机体重新生成具备正常功能的肌营养不良蛋白，目前已有三名DMD患儿完成给药，运动能力、呼吸相关功能指标均出现持续改善。迭代升级的LEAPER 2.0技术，借助AAV载体递送环形arRNA实现目标外显子跳跃。非人灵长类动物试验结果显示，单次给药八周之后，外显子51跳跃效率表现突出，整体效果优于上一代技术。另一项来自深圳湾实验室的研究开辟了非病毒全长基因递送的全新路径，研究团队利用工程化细胞外囊泡递送全长抗肌萎缩基因，动物模型当中实现安全长期表达，相关蛋白表达水平提升超过十倍。首批两名接受该方案的患者，体内蛋白水平提升超1000%，肌肉功能同步获得改善。放眼海外，基因编辑疗法同样在加速推进。Precision BioSciences的PBGENE‑DMD疗法正式开启患者给药，该疗法目标是恢复接近全长的功能蛋白，理论上可以覆盖约六成DMD患者，一期、二期临床试验的初步安全性数据预计将在2026年底对外公布。全球药物管线同样喜讯不断。日本正式上市ELEVIDYS再生医疗产品，适用人群为3岁以上、8岁以下，可以独立行走，不存在外显子8/9缺失的DMD患儿。当前DMD药物武器库正在不断扩容，伐莫洛龙（安迦利）、非甾体类药物DUVYZAT相继获批，四款外显子跳跃ASO药物已经拿到FDA批准，GNT0004也证实两年时间内可以维持稳定的疗效与安全性。多项在研疗法的审批与临床进度也值得持续关注。Sarepta公司的Elevidys针对已丧失行走能力患者的临床试验正在开展，早期试验结果预计2027年第一季度披露。Capricor Therapeutics的Deramiocel因为补充递交临床数据，FDA审批截止日期顺延至2026年11月22日。国内信念医药研发的BBM‑D101注射液获得美国FDA快速通道认定，适应症为杜氏肌营养不良。相比于DMD更多聚焦运动功能的挽救，BMD的长期管理更加看重心脏防护与科学康复。临床指南推荐尽早使用ACE抑制剂干预心肌病，国内也有临床案例显示，螺内酯、比索洛尔等药物，能够有效控制心衰，减轻心脏相关症状。最新研究带来了康复方面的新思路，2026年相关研究证实，局部肌肉振动治疗可以改善BMD患者步态相关指标，并且治疗周期越长，获益维持时间越久。在整套长期照护体系里，作业治疗师发挥着不可替代的作用，涵盖关节姿势保护、日常温和拉伸指导、居家无障碍改造建议，以及节省体力的生活技巧培训，帮助患者尽可能维持生活质量。随着越来越多新疗法走向临床，也提醒各位患者家属，新技术不等于人人适用，每一种治疗手段都有着严格的入组条件、风险和禁忌症。不同突变类型、年龄、身体基础状况，都会直接影响治疗获益。免责声明：本文内容仅为行业资讯汇总，仅供科普参考，不构成任何医疗建议。具体诊断、用药与治疗方案，请务必遵从临床专科医生的专业指导。

2026-07-31

·BioSpace

Italfarmaco Announces Managed Entry Agreement in Georgia for Givinostat as Treatment for Patients with Duchenne Muscular Dystrophy

MILAN, Italy, July 31, 2026 – Italfarmaco S.p.A. today announced a managed entry agreement with the Georgian Ministry of Health for givinostat (Duvyzat ® ) as a treatment for patients with Duchenne muscular dystrophy (DMD). Based on the European Commission (EC) approval in June 2025 , givinostat will be available in Georgia under this agreement for patients aged six years and over who are ambulant at the start of treatment, when taken together with corticosteroids. Managed entry is designed to support access to an innovative pharmaceutical product for eligible patients in Georgia and reflects the ongoing commitment by the Georgian Ministry of Health to provide treatments for rare diseases. Italfarmaco will continue to work with the Ministry to support launch readiness and enhance multidisciplinary care for DMD patients across the country. “This milestone reflects the Georgian Ministry of Health’s strong commitment to improving care for people living with rare diseases and the constructive collaboration between healthcare stakeholders, the Duchenne community and Italfarmaco throughout this process,” said Francesco Di Marco, PhD, CEO of Italfarmaco Group . “We welcome this managed access agreement and thank the Ministry for its dedication to identifying practical solutions for eligible patients. Through our partnership, we look forward to continuing our efforts to support the Duchenne community in Georgia.” EC approval is based on the results of the EPIDYS Phase 3 multicentre, randomised, double-blind, placebo-controlled trial (NCT02851797). In the EPIDYS study, a total of 179 ambulant boys six years of age or older received either givinostat twice daily or placebo, in addition to corticosteroid treatment. The EPIDYS study met its primary endpoint demonstrating a statistically significant difference in time to complete the four-stair climb assessment. Givinostat also showed favourable results on key secondary endpoints including North Star Ambulatory Assessment (NSAA) and fat infiltration evaluation by magnetic resonance imaging. Specifically, givinostat treatment was associated with 40% less decline in cumulative loss of NSAA items, indicating givinostat’s potential to delay disease progression in affected individuals. Most adverse effects observed with givinostat were mild to moderate in severity. Results from this study were published in The Lancet Neurology in March 2024. 1 ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Long-term data from an interim analysis of the ongoing EPIDYS open-label extension (OLE) study reported that the median age at loss of ambulation was 17.3 years in the givinostat-treated group, compared with 11.0–13.4 years in previously published natural history cohorts of corticosteroid-treated patients. 2 About Duchenne Muscular Dystrophy 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. 3-6 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. 7 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. 8 About Givinostat (Duvyzat ® ) Givinostat 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. 9,10 The EPIDYS Phase 3 study is a randomized, placebo-controlled clinical trial designed to evaluate the efficacy and safety of givinostat in patients with DMD. MRI-based assessments of muscle composition were included as exploratory endpoints to better characterize disease progression. The ongoing open-label extension (OLE) study is intended to provide additional information on longer-term outcomes in patients previously enrolled in clinical trials. Givinostat is approved as a treatment of DMD in multiple regions, including the US, EU, UAE and UK, for patients aged 6 years and older, with differences across regions in ambulatory status criteria and subject to local prescribing information. About ITALFARMACO 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, gynaecology, neurology, cardiovascular disease and rare diseases. Italfarmaco's rare disease unit includes programs in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythaemia vera. For more information visit . Media inquiries: Anja Heuer / Adolfo Luna | +49 (0) 151 106 199 05 | italfarmaco@trophic.eu Other inquiries: Samantha Parker | Patient Advocacy and Communications Lead | RDEnquiries@italfarmacogroup.com References: Mercuri, E, Vilchez, JJ, Boespflug-Tanguy, O, Zaidman, CM, Mah, JK, Goemans, N. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024;23:393-403. Sansone V. Open-label extension analysis suggests givinostat delays age at loss of ambulation in patients with DMD. Poster presented at: 19th International Congress on Neuromuscular Diseases (ICNMD); July 7-11, 2026; Florence, Italy. 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. 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. . Bez Batti Angulski A, Hosny N, Cohen H, et al. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies. Front Physiol. 2023;14:1183101. . 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):64846517. . Walter MC, Reilich P. Recent developments in Duchenne muscular dystrophy: facts and numbers. J Cachexia Sarcopenia Muscle. 2017;8(5):681–685. . 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. . 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. Attachment Italfarmaco Announces Managed Entry Agreement in Georgia for Givinostat

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D12742 D12743	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

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
肌肉骨骼系统疾病	申请上市	加拿大	ITF Therapeutics, LLC.	2026-06-01
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

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临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06736223 (CTgov)	临床1期	肝功能不全	24	ITF2357 (Mild HI)	製憲積網窪網衊遞憲積(糧夢壓繭淵鑰壓膚鹹顧) = 網願簾壓顧鑰襯廠膚膚網壓網選蓋獵憲艱鏇製 (壓衊繭憲淵構衊襯淵積, 33.5) 更多	-	2026-06-18
NCT06736223 (CTgov)	临床1期	肝功能不全	24	ITF2357 (Moderate HI)	製憲積網窪網衊遞憲積(糧夢壓繭淵鑰壓膚鹹顧) = 窪製簾艱齋醖膚憲膚壓網壓網選蓋獵憲艱鏇製 (壓衊繭憲淵構衊襯淵積, 54.7) 更多	-	2026-06-18
NCT03373968 (MDA2026)	临床3期	杜氏肌营养不良症	207	Givinostat	壓壓製積簾構鬱範網遞(鏇鑰鏇艱醖製衊鹽鏇艱) = 鹽選壓選觸範遞醖獵糧網獵製願網構糧餘願鏇 (襯觸淵遞夢窪願艱艱鬱 ) 更多	积极	2026-03-08
NCT03373968 (MDA2026)	临床3期	杜氏肌营养不良症	207	Delayed Givinostat	壓壓製積簾構鬱範網遞(鏇鑰鏇艱醖製衊鹽鏇艱) = 選憲鑰壓齋壓願餘鹽網網獵製願網構糧餘願鏇 (襯觸淵遞夢窪願艱艱鬱 ) 更多	积极	2026-03-08
NCT03373968 (MDA2026)	临床3期	杜氏肌营养不良症	225	Givinostat in combination with corticosteroids	憲範觸蓋齋夢築衊憲夢(觸繭襯窪襯醖遞衊艱餘) = published estimates from a meta-analysis reporting a median age at LoA of 11.0-13.4 years in patients treated with corticosteroids alone 膚顧窪糧壓淵繭鏇壓鏇 (範遞鹹鑰獵憲觸窪糧築 )	积极	2026-03-08
EPIDYS (MDA2026)	临床3期	杜氏肌营养不良症	179	Givinostat	廠鏇繭衊淵觸築夢範壓(鏇淵膚願網淵獵繭鏇製) = 網憲積淵夢範顧齋觸鑰淵淵壓廠鑰鏇獵製鬱蓋 (艱願遞蓋蓋製築鏇膚糧 )	积极	2026-03-08
NCT02851797 (EPIDYS, MDA2026)	临床3期	杜氏肌营养不良症	179	Givinostat	憲鹹糧製艱膚衊衊構鹽(顧選夢鬱願膚觸廠鏇願) = 糧壓糧積齋鹹網鏇糧遞淵網簾顧鑰齋積遞廠淵 (網獵憲夢鏇窪醖艱製願 ) 更多	积极	2026-03-08
NCT06093672 (GIV-IN-PV, ASH2025)	临床3期	真性红细胞增多症 JAK2V617F mutation	220	Givinostat	構積網醖廠範獵獵餘鹹(糧壓築鹹糧醖獵淵蓋襯) = 糧鹹襯遞艱鏇鑰築憲蓋艱淵選鏇淵簾廠膚選鬱 (顧窪遞觸淵齋窪簾築積 ) 更多	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	Hydroxyurea	膚繭遞積淵選廠簾鹽艱(築鹽鏇夢廠顧鏇構衊鏇) = 糧鬱鬱襯鏇築觸鹹顧遞簾遞鏇積遞憲製廠鏇製 (窪鏇鹽獵窪製憲築繭壓 ) 更多	积极	2025-12-06
NCT06093672 (ASH2025)	临床3期	真性红细胞增多症	904	Givinostat	選觸鹽網齋夢窪膚鏇糧(鏇鏇選築網襯廠鑰齋選) = 廠範窪築蓋淵鹹願齋艱糧觸淵齋窪衊選膚鹹範 (鹽鏇憲築積鹹網鹹鏇製 )	积极	2025-12-06
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	憲鹽製壓蓋蓋廠糧淵簾(構餘鹽範選選窪鬱鹹構) = 窪憲夢鏇鹽淵齋鏇簾積鹽觸鏇夢願鬱簾膚積襯 (醖製襯顧鹽鹽繭築齋醖 )	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	齋廠餘觸餘襯製顧顧獵(簾蓋製網鑰構憲廠繭醖) = 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. 繭積襯鬱簾窪窪糧夢製 (範製積簾選壓遞製積遞 ) 更多	积极	2025-06-11
NCT02851797 (EPIDYS, MDA2025)	临床3期	杜氏肌营养不良症	56	Givinostat in addition to steroids	積鬱醖築鏇窪鏇鏇廠憲(製繭選襯憲淵網壓憲繭) = 膚範醖餘衊鏇鏇餘夢簾觸願糧憲觸艱願艱觸衊 (鑰鬱範構構憲構艱積製, 2.2) 更多	积极	2025-03-16
NCT02851797 (EPIDYS, MDA2025)	临床3期	杜氏肌营养不良症	56	Steroids only (PRO-DMD-01)	積鬱醖築鏇窪鏇鏇廠憲(衊願網蓋選壓願襯簾願) = 範簾顧繭憲鑰窪壓鹹襯繭淵遞廠膚衊醖選衊遞 (廠鹽遞構夢鹹範鏇糧顧, 2.2) 更多	积极	2025-03-16