A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - ESSENCE

开始日期2022-10-05

申办/合作机构

Sarepta Therapeutics, Inc.

[+1]

NCT04708314 / Terminated临床4期

An Open-Label Study to Evaluate the Safety of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

This is an open-label study to evaluate the safety and tolerability of golodirsen injection in Non-ambulant DMD patients with confirmed genetic mutations amenable to treatment by exon 53 skipping (Golodirsen).
Golodirsen 30 mg/kg will be administered as an intravenous (IV) infusion over approximately 35 to 60 minutes once a week during the treatment period (up to 96 weeks). After the treatment period, patients can go into a safety extension period (not to exceed 48 weeks) until the patient is able to transition to commercially available drug or a separate golodirsen study.
Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. Exploratory assessments, including pulmonary function tests (PFTs), upper extremity testing, and other measurements of functional status, will occur at functional assessment visits every 12 weeks over the first year of treatment and approximately every 24 weeks over the second year of treatment.

开始日期2020-10-31

申办/合作机构

Rare Disease Research, LLC

[+1]

NCT04179409 / Completed临床2期IIT

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

This is an 48-week open-label study to determine the efficacy and safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12.

开始日期2020-02-18

申办/合作机构

Nationwide Children's Hospital

[+1]

100 项与 Golodirsen 相关的临床结果

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100 项与 Golodirsen 相关的转化医学

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100 项与 Golodirsen 相关的专利（医药）

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项与 Golodirsen 相关的文献（医药）

2023-08-28·Biomolecules

Advances in Dystrophinopathy Diagnosis and Therapy.

Review

作者: Siciliano, Gabriele ; Saad, Fawzy A ; Angelini, Corrado

Dystrophinopathies are x-linked muscular disorders which emerge from mutations in the Dystrophin gene, including Duchenne and Becker muscular dystrophy, and dilated cardiomyopathy. However, Duchenne muscular dystrophy interconnects with bone loss and osteoporosis, which are exacerbated by glucocorticoids therapy. Procedures for diagnosing dystrophinopathies include creatine kinase assay, haplotype analysis, Southern blot analysis, immunological analysis, multiplex PCR, multiplex ligation-dependent probe amplification, Sanger DNA sequencing, and next generation DNA sequencing. Pharmacological therapy for dystrophinopathies comprises glucocorticoids (prednisone, prednisolone, and deflazacort), vamorolone, and ataluren. However, angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blockers (ARBs), and β-blockers are the first-line to prevent dilated cardiomyopathy in dystrophinopathy patients. Duchenne muscular dystrophy gene therapy strategies involve gene transfer, exon skipping, exon reframing, and CRISPR gene editing. Eteplirsen, an antisense-oligonucleotide drug for skipping exon 51 from the Dystrophin gene, is available on the market, which may help up to 14% of Duchenne muscular dystrophy patients. There are various FDA-approved exon skipping drugs including ExonDys-51 for exon 51, VyonDys-53 and Viltolarsen for exon 53 and AmonDys-45 for exon 45 skipping. Other antisense oligonucleotide drugs in the pipeline include casimersen for exon 45, suvodirsen for exon 51, and golodirsen for exon 53 skipping. Advances in the diagnosis and therapy of dystrophinopathies offer new perspectives for their early discovery and care.

2023-05-09·Neurology

Increased Dystrophin Production With Golodirsen in Patients With Duchenne Muscular Dystrophy

Article

2023-01-01·Methods in molecular biology (Clifton, N.J.)

Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes

Article

作者: Echigoya, Yusuke ; Yokota, Toshifumi

Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is a therapeutic approach that applies to many Duchenne muscular dystrophy (DMD) patients harboring out-of-frame deletion mutations in the DMD gene. In particular, PMOs for skipping exon 44 have been developing in clinical trials, such as the drug NS-089/NCNP-02. Two exon 53 skipping PMOs, golodirsen and viltolarsen, have received conditional approval for treating patients due to their ability to restore dystrophin protein expression. Although promising, further development of exon-skipping technology is needed for patients to have more therapeutic benefit. This chapter describes evaluation methods of exon 44 and 53 skipping PMOs in immortalized DMD patient-derived skeletal muscle cells. We introduce how to quantify exon-skipping efficiencies and dystrophin rescue levels represented by RT-PCR and western blotting, respectively. The screening methods using immortalized patient myotubes can serve to find exon-skipping PMO drug candidates.

项与 Golodirsen 相关的新闻（医药）

2024-06-22

·药融圈

首款DMD基因疗法Elevidys获FDA完全批准！

▲8月15-16日 NDC2024生物医药创新者峰会扫码立即报名注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。 6月20日，小核酸龙头Sarepta Therapeutics发布公告：美国FDA批准其杜氏肌营养不良（DMD）基因疗法Elevidys扩展适应症，将年龄至少为4岁的患者纳入其中。FDA对可行走的患者授予了传统批准，对于不可行走的患者，则授予加速批准。值得注意的是， Elevidys禁用余DMD基因中涉及外显子8和/或外显子9的确实缺失突变的患者，因为他们最容易出现“免疫介导性肌炎”。早在2023年6月，美国FDA加速批准DMD基因疗法Elevidys 用于治疗4至5岁患有杜氏肌营养不良症的儿科患者，定价为320万美元/剂，这是全球i首款获批上市的杜氏肌营养不良AAV基因疗法。如今，Elevidys 获批用于≥4岁DMD患者，无论其是否能够行走，这对DMD患者的治疗来说具有重要意义。受此影响，Sarepta Therapeutics股价上涨5%，目前市值为111亿美元。杜氏肌营养不良症（Duchenne muscular dystrophy，DMD）是一种X染色体连锁的隐性遗传疾病，由DMD基因突变引起。该基因是目前人类中已知最大的基因，包含79个外显子，编码含有3685个氨基酸的抗肌萎缩蛋白，其突变类型复杂且异质性高，因此其治疗药物的开发也十分困难。国内外常用类固醇激素结合多学科诊疗延缓DMD患者运动能力丧失，提高患者的生存质量；但类固醇激素治疗伴有肥胖、矮小、骨质疏松等副作用，且不能改变疾病的最终结局。而Elevidys（delandistrogene moxeparvovec，SRP-9001）是一款重组基因疗法，将表达微抗肌萎缩蛋白（micro-dystrophin）的转基因包装在AAV病毒载体中，通过单次静脉注射，使得患者肌肉生成具有部分抗肌萎缩蛋白功能的重组蛋白解决DMD的根本病因，可以对携带任何类型DMD致病基因变异的患者生效。 Elevidys 2023年销售额达2.004亿美元；2024Q1 销售额约1.34亿美元，都远超预期。根据加速批准途径，Sarepta承诺进行并提交一项随机对照试验的结果，以验证和确认Elevidys在不可行走DMD患者中的临床益处。除了Sarepta，还有多家公司进行DMD基因疗法，但是进展不太顺利。 6月12日，辉瑞宣布其DMD基因疗法PF-06939926的III期临床CIFFREO失败，与安慰剂相比，PF-06939926在III期试验中未达到主要和关键次要终点。PF-06939926是一款在研静脉注射基因疗法，使用重组腺相关病毒9 (rAAV9)衣壳在人类肌肉特异性启动子的控制下传递缩短的人类肌营养不良蛋白基因。本以为Elevidys并未达到主要终点，只达到了次要终点，PF-06939926有望成为了业内希望。但是试验结果不尽人意。 5月份，NS Pharma宣布其DMD小核酸疗法Viltepso 3期试验失败，经过48周治疗后，与安慰剂相比，接受Viltepso治疗的患者未出现统计学上的显著差异。Viltepso是一种反义寡核苷酸，通过跳过第53号外显子发挥作用。它于2020年3月在日本获批用于治疗确诊发生53外显子跳跃基因突变的DMD患者。2020年8月，Viltepso获得美国FDA加速批准上市，成为第2款用于治疗外显子53跳跃DMD的反义寡核苷酸(ASO)疗法。随着辉瑞DMD基因疗法的失败，Elevidys扩展适应症获批，意味着Elevidys市场份额进一步扩大。除了辉瑞、NS Pharma，还有多款DMD基因疗法处于临床早期阶段，国内金唯科、新芽基因就有布局：关于Sarepta Therapeutics Sarepta Therapeutics公司是一家专注于治疗罕见、传染病和其他疾病的生物技术公司。公司针对广泛的疾病，同时专注于其候选药物的快速发展。Sarepta的战略包括专有的RNA靶向技术平台，用于开发新型药物产品，以治疗广泛的疾病并解决关键的未满足的医疗需求。作为小核酸龙头，Sarepta Therapeutics还开发3款DMD小核酸疗法，分别是2016年获批上市的Eteplirsen、2019年获批上市的Golodirsen和2021年获批上市的Casimersen，均获得FDA加速审批。2023年，Eteplirsen销售额5.4亿美元（+6%）；Golodirsen 销售额1.3亿美元（+11%）；Casimersen 销售额2.74亿美元（+28%）。研发管线参考资料： https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-expanded-us-fda-approval-elevidys?_ga=2.185028521.1002736062.1718930498-723160938.1718930498 08/15 专题一 XDC领域的差异化创新和国际化策略 ◎ 09:00-09:20 XDC偶联药物研发进展及挑战 ◎ 09:20-09:40 ADC药物创新要点及与挑战 ◎ 09:40-10:00 Nectin-4 ADC最新临床研究策略 ◎ 10:00-10:20 双抗ADC早期设计和靶点选择 ◎ 10:20-10:40 新型偶联技术bi-XDC的设计及优势 ◎ 10:40-11:00 XDC药物国际注册策略的思考及实例分享 ◎ 11:00-11:20 偶联药物BD合作与投融资策略 ◎ 11:20-11:40 ADC药物出海之--海外临床开发策略和经验 ◎ 11:40-12:00 偶联药物CMC难点及商业化策略 08/16 专题二核素药物开发专题 ◎ 高通量筛选技术与放射性新药物发现与优化 ◎ 靶向GPC3放射性药物的筛选与开发 ◎ 靶向放射性核素疗法的迭代与升级的展望 ◎ 放射性药物CMC的全面解析 ◎ 临床CRO助力中国放射性新药研发--挑战与应对 ◎ 放射性药物注册策略与实施 ◎ 医用放射性同位素的国产化及其应用 ◎ 从投资角度看待放射性药物赛道 ◎ 临床经验分享：靶向放射性药物对多发转移肿瘤治疗的优势 ◎ 创新核药研发与商业化的新模式与案例解析拟邀嘉宾高长寿信达生物，CSO 刘翠华百奥泰生物高级副总裁刘大涛迈威生物，CEO 吕明尚健生物董事长秦刚启德医药，CEO 唐华东植德律所，合伙人田文志宜明昂科，创始人、董事长兼总经理王春河达石药业，董事长王忠民康方生物，副总裁衣宪华苏州普灵生物，CEO 张富尧弼领生物，董事长&总经理赵永新多禧生物CEO 周清诗健生物，CEO 朱义百利药业，董事长朱永红岸迈生物，CMO 限时扫码报名！版权声明：本文转自生物药大时代，如不希望被转载的媒体或个人可与我们联系，我们将立即删除【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。

基因疗法临床3期加速审批

2024-06-21

·佰傲谷BioValley

尽管主要终点未达到，但是FDA批准

目前来看，Sarepta/罗氏的Elevidys仍然将会是DMD唯一的治愈希望。尽管Elevidys价格昂贵、难以获得。随着辉瑞的同类基因治疗产品宣告III期临床失败、Solid重新设计新一代管线、Regenxbio即将启动关键试验。Elevidys再拿下监管批准——FDA批准扩展Elevidys的适应症，允许用于治疗4岁及以上的DMD患者。以及FDA对可行走的患者授予了传统批准；对于不可行走的患者授予了加速批准。细胞与基因治疗（CGT）开发难度高，业内常有“九死一生”的评价，在CGT产品的全生命周期内，常常会遇到各种各样的挑战和阻碍。Thermo Fisher聚焦CGT，铸就全流程新范式，助力CGT开发。有分析师认为，本次监管批准，将会加速Elevidys的销售。Elevidys已经在过去的销售中证实了其销售潜力和市场需求，自2023年6月上市至2024年3月底，累积销售3.34亿美元。延伸阅读：大卖2个亿美元！尽管主要终点未达到 Elevidys是一款AAV基因疗法，利用AAV作为载体将表达微抗肌萎缩蛋白（micro-dystrophin）的转基因递送至患者的肌肉细胞中，使得患者能够自主生成微抗肌萎缩蛋白，从而缓解疾病。DMD患者有望通过一次性给药，实现终身受益，不过DMD基因第8和/或第9外显子有缺失的患者禁用Elevidys。 Elevidys于2023年6月首次获得FDA加速批准上市，用于治疗4至5岁的DMD儿童患者；是首款获得美国FDA批准上市的DMD基因治疗。 2024年6月20日，Elevidys获得FDA批准扩大治疗范围，用于治疗4岁以上的DMD患者。以及FDA对可行走的患者授予了传统批准；对于不可行走的患者授予了加速批准。 Elevidys最新的批准是基于全球III期研究EMBARK（Study 9001-301）的顶线结果，但是Sarepta公司于2023年10月披露的数据显示，Elevidys在研究中未达到主要终点。不过，EMBARK研究的关键次要终点，包括Time to rise (TTR)测试、10米步行测试等，在所有年龄组中都显示出具有临床意义的治疗益处。延伸阅读：主要终点未达到，却支持提交疗效补充申请 FDA受理DMD基因治疗的疗效补充申请主要终点未达到的确引发了些许怀疑，但是FDA生物制品评价与研究中心（CBER）主任Peter Marks肯定了Elevidys的益处，推翻了其他FDA官员对Elevidys的否定意见。市场竞争 DMD是一种特征明确、罕见、进行性的、致命的X连锁神经肌肉单基因疾病，患者通常会在青少年时期逐渐失去行走能力，并可能在30岁左右死于心脏或肺部并发症。DMD在美国发病率约为5000分之一。 DMD目前尚无治愈方法，基因治疗被视为治愈的希望，目前也仅有Elevidys获得监管批准，并在未来几年内不会有同类的竞争产品上市。这是由于辉瑞的同类基因治疗产品宣告III期临床失败、Solid放弃后期DMD管线，重新设计新一代管线、Regenxbio还未启动关键试验，这都为Elevidys争取到了未来几年的市场独占时间。延伸阅读：辉瑞基因疗法III期临床失败，此前已致2人死亡明星管线失败，Solid再出发 2023年6月，美国FDA对Elevidys的批准，仅用于4-5岁的DMD患儿。Sarepta公司预计每年约有400名患儿接受Elevidys治疗。此外，Sarepta公司的发言人表示，Elevidys新的批准，将覆盖80%的DMD患者。除了基因治疗Elevidys，美国FDA还批准了几款DMD药物，包括Sarepta公司的两款外显子跳跃药物，以及一种非甾体药物，不过这些药物都需要进行长期服药。另外，Elevidys已经成为Sarepta公司的核心业绩增长驱动，超过该公司另外两款DMD疗法Amondys 45和Vyondys 53。参考资料： 1.https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-expanded-us-fda-approval-elevidys?_ga=2.191694697.2122024712.1718933897-1175645299.1688607373 2.https://www.biopharmadive.com/news/sarepta-fda-elevidys-expand-approval-duchenne-gene-therapy/716778/ 本周好文推荐如需转载请联系佰傲谷并在醒目位置注明出处 · · · · · · · ·

上市批准临床3期基因疗法

2024-06-17

·PRNewswire

Pfizer's Setback in Duchenne Muscular Dystrophy Treatment Trial Clears Path for Sarepta, and Many Other Pharma Companies | DelveInsight

Pfizer's mini-dystrophin gene therapy fordadistrogene movaparvovec failure in the Duchenne muscular dystrophy treatment opens new opportunities for several pharmaceutical companies such as Sarepta Therapeutics (SRP-5051), Santhera Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), and others, to advance their therapies and potentially dominate the DMD market. LAS VEGAS, June 17, 2024 /PRNewswire/ -- Duchenne muscular dystrophy is the most prevalent type of muscular dystrophy found in children. This genetic condition leads to ongoing muscle weakening and degeneration. It is one among nine different forms of muscular dystrophy. According to DelveInsight's estimates, the US accounted for around ~17K total prevalent cases of Duchenne muscular dystrophy in 2023. The highest cases were found in the children of the 5-9 age group, these numbers are expected to grow by the year 2034. As per DelveInsight's analysis, the most commonly associated comorbidities accompanied with DMD included the highest cases of scoliosis accounting for around ~20% of cases followed by ADHD, cardiomyopathy, and obsessive-compulsive disorders accounting for around ~19%, ~18%, and ~15% cases respectively in the US in the year 2023. The standard of care for DMD typically involves a combination of corticosteroids and surgical interventions. Prednisone and deflazacort are commonly prescribed glucocorticoids that have been utilized for over two decades to increase muscular strength in DMD patients. Surgery may be necessary to address contractures, spinal curvature, or scoliosis, which can affect breathing and overall mobility. Additionally, interventions such as pacemakers or other cardiac devices may be utilized to improve heart function. Sarepta Therapeutics is a key player in the DMD market, boasting three approved assets and two in the current pipeline. Despite its dominance, emerging competition is evident. The historical success rate for market entry varies across the 7MM. Notably, the DMD market exhibits geographic disparities in regulatory approvals, with instances of products approved in one jurisdiction and rejected in another, despite identical clinical data, reflecting the distinct standards of regulatory bodies such as EMA and FDA. Learn more about the FDA-approved DMD drugs @ Drugs for Duchenne Muscular Dystrophy Treatment The current US DMD treatment market possesses approved products, EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), and VILTEPSO (viltolarsen), ELEVIDYS (delandistrogene moxeparvovec) in patients with DMD. In January 2024, Santhera Pharmaceuticals announced the introduction of its AGAMREE (vamorolone) to treat DMD patients aged four years and above in Germany. Santhera Pharmaceuticals has officially entered the commercial stage of its biopharma journey with the launch of this drug in Germany. ELEVIDYS, the first FDA-approved gene therapy for DMD, received accelerated approval in June 2023. However, ICER recently challenged the potential conversion of Sarepta's gene therapy to full approval, citing concerns about the surrogate endpoint used in the approval process. In the EU4 and the UK, the current market is dominated by steroid therapies along with an approved medication targeting DMD patients with the nonsense mutation, TRANSLARNA (ataluren). In Japan, the only approved treatment is VILTEPSO (viltolarsen). ELEVIDYS is a prescription gene therapy designed for ambulatory children aged 4 to 5 years with DMD and a confirmed dystrophin gene mutation. It received accelerated approval. Patients will take oral corticosteroids before and after receiving ELEVIDYS and will have weekly blood tests to monitor liver enzyme levels for three months post-treatment. In June 2023, the USFDA approved ELEVIDYS as the first gene therapy for treating pediatric patients aged 4-5 years with Duchenne Muscular Dystrophy and a confirmed DMD gene mutation. VYONDYS 53, developed by Sarepta Therapeutics, is prescribed for treating DMD in patients with a specific mutation of the DMD gene that can be addressed by exon 53 skipping. This approval is under the accelerated approval pathway, based on the observed increase in dystrophin production in the skeletal muscle of patients treated with VYONDYS 53. The FDA approved VYONDYS 53 (golodirsen) in December 2019. Additionally, the FDA granted VYONDYS 53 (golodirsen) New Chemical Entity (NCE) exclusivity until December 2024 and Orphan Drug Exclusivity until December 2026. To know more about DMD treatment options, visit @ New Treatment for Duchenne Muscular Dystrophy The DMD market is crowded with so many companies including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, and others are running clinical trials to improve the treatment space. The DMD pipeline possesses potential drugs in mid-stage developments to be launched shortly. Some of the promising drugs in the pipeline include Pizuglanstat (TAS-205), SRP-5051, Allogeneic Cardiosphere-derived Cells (CAP-1002), ITF2357 (Givinostat), and others that hold the potential to create a significant positive shift in the DMD market size. Discover which therapies are expected to grab major DMD market share @ Duchenne Muscular Dystrophy Market Report Italfarmaco is developing Givinostat (ITF2357), an oral small molecule that functions as a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic properties. ITF2357 increases the production of follistatin protein in muscle cells by inhibiting the HDAC enzyme, leading to increased muscle mass and prevention of muscle degeneration. This action counters the effects of myostatin, helping to alleviate the symptoms of DMD. Givinostat is currently in Phase III trials. The FDA has granted it Orphan Drug, Rare Pediatric Disease, and Fast Track designations for DMD treatment. Additionally, the European Commission has designated Givinostat as an Orphan Medicinal Product for DMD treatment. TAS-205 (pizuglanstat) is a selective inhibitor of hematopoietic prostaglandin D synthase (HPGDS), developed by Taiho Pharmaceutical. It is being advanced as a treatment for Duchenne muscular dystrophy (DMD), effective irrespective of the type of dystrophin gene mutation. By inhibiting HPGDS, which worsens the inflammatory response in the muscles of DMD patients, it helps control the decline in motor function. Currently, it is in Phase III of clinical trials. Discover more about drugs for DMD in development @ Duchenne Muscular Dystrophy Clinical Trials The anticipated launch of these emerging therapies for Duchenne muscular dystrophy are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Duchenne muscular dystrophy market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for DMD is expected to grow from USD 2.1 billion in 2023 with a significant CAGR by 2034. The growth of the DMD market is expected to be mainly driven by increased diagnosed incidence, patient awareness, and a robust clinical pipeline during the forecast period (2024–2034). DelveInsight's latest published market report titled as Duchenne Muscular Dystrophy Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the DMD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The DMD market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Duchenne Muscular Dystrophy Age-specific Cases of Duchenne Muscular Dystrophy Ambulatory and Non-ambulatory Cases of Duchenne Muscular Dystrophy Mutation-specific Cases of Duchenne Muscular Dystrophy Associated Comorbidities in Duchenne Muscular Dystrophy The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM DMD market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this DMD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the DMD market. Also, stay abreast of the mitigating factors to improve your market position in the DMD therapeutic space. Related Reports Duchenne Muscular Dystrophy Epidemiology Forecast Duchenne Muscular Dystrophy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted Duchenne muscular dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key nonsense mutation Duchenne muscular dystrophy companies, including Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, among others. Becker Muscular Dystrophy Pipeline Becker Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key becker muscular dystrophy companies, including Epirium Bio, Ultragenyx, Strykagen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

临床3期上市批准孤儿药快速通道

100 项与 Golodirsen 相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
D11707	-	J05AB	DB15593

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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	Sarepta Therapeutics, Inc.	2019-12-12

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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


NCT02310906 (Study 4053-101, MDA2024)	临床1/2期	杜氏肌营养不良症 exon 53	20	Golodirsen	範遞構鹽夢襯簾糧築鹹(選選築簾齋糧鏇鹽夢襯) = 鏇壓獵窪鹽憲襯顧鏇鑰鹽襯蓋壓夢衊糧築選願 (壓糧觸繭憲齋簾衊餘製, 1.8) 更多	积极	2024-03-03
				Golodirsen	範遞構鹽夢襯簾糧築鹹(選選築簾齋糧鏇鹽夢襯) = 獵繭齋鑰齋觸糧獵襯夢鹽襯蓋壓夢衊糧築選願 (壓糧觸繭憲齋簾衊餘製, 1.3) 更多
MDA2024	N/A	杜氏肌营养不良症	-	Golodirsen	窪積窪願鹽鑰窪蓋構廠(襯鏇鹹觸積築鹽鏇壓築) = treatment-emergent adverse events were generally mild, nonserious, and unrelated to treatment; there were no treatment-related discontinuations, kidney abnormalities, or port-related infections in the OLE 艱繭構淵網鏇遞築選鏇 (積鑰鏇鑰範範顧艱鹽艱 )	-	2024-03-03
				Golodirsen
NCT04179409 (CTgov)	临床2期	杜氏肌营养不良症	3	Amondys 45 (AMONDYS 45)	衊窪憲遞餘壓網網願鹹(鏇鹽鬱蓋簾衊醖衊選廠) = 願觸廠齋繭醖製蓋衊淵齋鬱窪夢蓋餘選齋憲餘 (積願積壓鹹齋範壓淵窪, 壓遞齋齋憲遞願糧選築 ~ 衊繭網膚淵壓襯醖繭獵) 更多	-	2023-10-12
				Exondys 51 (EXONDYS 51)	網積獵淵築遞選範衊鹹(醖窪糧繭築鏇網積獵鹹) = 鹹構選壓憲鏇衊鑰築鹽襯膚遞範艱糧鹹選衊夢 (夢窪憲廠鑰製鬱淵壓壓, 製遞觸簾淵觸蓋獵獵壓 ~ 淵憲淵鏇簾觸網餘製夢) 更多
NCT02310906 (4053-101, Pubmed \| Nucleic Acid Ther) 人工标引	临床1/2期	杜氏肌营养不良症	25	golodirsen	鑰膚醖鬱構膚選觸範選(築窪膚糧製鏇膚蓋築齋) = 觸積簾憲壓憲艱網願壓襯糧壓獵網獵顧製醖製 (積遞網廠餘製夢範鹹餘 ) 更多	积极	2022-02-01
				external controls	鑰膚醖鬱構膚選觸範選(築窪膚糧製鏇膚蓋築齋) = 蓋醖憲願願艱窪鏇艱鹽襯糧壓獵網獵顧製醖製 (積遞網廠餘製夢範鹹餘 )
NCT02310906 (Pubmed \| Ann Neurol)	临床1/2期	杜氏肌营养不良症	39	Golodirsen	齋築鏇淵齋蓋網鏇觸艱(襯襯壓窪憲築衊夢繭鏇) = 齋艱齋膚淵遞餘繭顧淵遞築構廠鬱膚醖糧顧網 (網鑰鏇願齋艱衊艱顧餘 )	-	2020-05-26
CNS2018	N/A	genetic mutations amenable to exon 53 skipping	-	Golodirsen 30 mg/kg	鏇願齋獵築淵齋鹹願糧(艱積壓觸獵觸網憲鏇淵) = 壓醖鑰觸淵餘膚簾糧膚糧鬱醖憲獵淵艱鹹壓餘 (鹹鏇鹹廠遞築鹽鏇憲齋 )	-	2018-10-05
				Placebo	鏇願齋獵築淵齋鹹願糧(艱積壓觸獵觸網憲鏇淵) = 選廠衊鑰積獵壓鏇餘願糧鬱醖憲獵淵艱鹹壓餘 (鹹鏇鹹廠遞築鹽鏇憲齋 )
AAN2018	N/A	genetic mutation amenable to exon 53 skipping	25	Golodirsen 30 mg/kg	襯鏇淵觸願繭淵選壓鬱(壓鑰選遞壓網壓獵築顧) = 構鹽顧窪衊積築壓醖襯願製顧繭襯蓋衊廠鬱憲 (顧繭顧窪窪淵壓製蓋壓 )	-	2018-04-10