更新于：2024-06-24
Casimersen
更新于：2024-06-24
概要
基本信息
药物类型 反义寡核苷酸 |
别名 Casimersen (USAN/INN)、SRP-4045、Amondys 45 |
作用机制 DMD exon 45调节剂(DMD 外显子45调节剂) |
在研适应症 |
非在研适应症- |
非在研机构- |
最高研发阶段批准上市 |
首次获批日期 美国 (2021-02-25), |
最高研发阶段(中国)- |
特殊审评快速通道 (美国)、加速批准 (美国)、孤儿药 (美国) |
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序列信息
Sequence Code 29924134
来源: *****
关联
5
项与 Casimersen 相关的临床试验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
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.
Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
100 项与 Casimersen 相关的临床结果
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100 项与 Casimersen 相关的转化医学
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100 项与 Casimersen 相关的专利（医药）
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17
项与 Casimersen 相关的文献（医药）2023-12-28Cureus
AMONDYS 45 (Casimersen), a Novel Antisense Phosphorodiamidate Morpholino Oligomer: Clinical Considerations for Treatment in Duchenne Muscular Dystrophy
Review
作者: Shekoohi, Sahar ; Davis, Brice A ; Varrassi, Giustino ; Vasterling, Megan E ; Patel, Hirni ; Maitski, Rebecca J ; Kelkar, Rucha A ; Klapper, Rachel J ; Ahmadzadeh, Shahab ; Kaye, Alan D ; Barnes, Julie E
AMONDYS 45 (casimersen) is an antisense oligonucleotide therapy used to treat Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by a mutation in the DMD gene. Symptoms include progressive muscle weakness, respiratory and cardiac complications, and premature death. Casimersen targets a specific mutation in the DMD gene that results in the absence of dystrophin protein, a key structural component of muscle fibers. While there is currently no cure for DMD, exon-skipping therapy works by restoring the reading frame of the mutated gene, allowing the production of a partially functional dystrophin protein. Clinical trials of casimersen have shown promising results in increasing dystrophin production, as measured by polymerase chain reaction (PCR) droplets when compared to placebo. In a randomized double-blind trial, patients who received casimersen had significantly higher dystrophin levels when compared to those who received placebo. Casimersen therapy is administered through repeated intravenous infusions, although the optimal dosage and duration of treatment are still under investigation. Based on the completed and ongoing clinical trials, casimersen has been well tolerated, with most adverse events being mild and unrelated to casimersen. In 2021, AMONDYS 45 (casimersen) received approval from the US Food and Drug Administration (FDA) for the treatment of Duchene muscular dystrophy in patients with a mutation of the DMD gene that is amenable to exon 45 skipping. These collective findings indicate that casimersen has the potential to elicit functional changes in individuals with DMD, although further studies are necessary to comprehensively evaluate the specific functional improvements. Regardless, the FDA approval and ongoing clinic trials mark a significant milestone in the development of DMD treatments and offer hope for those affected by this debilitating disease.
2023-08-28Biomolecules
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.
2023-01-01Methods in molecular biology (Clifton, N.J.)
Restoring Dystrophin Expression by Skipping Exons 6 and 8 in Neonatal Dystrophic Dogs
Article
作者: Shah, Md Nur Ahad ; Yokota, Toshifumi
Duchenne muscular dystrophy (DMD) is caused by the mutations in the DMD gene resulting in no dystrophin production. Skipping DMD exons using phosphorodiamidate morpholino oligomers (PMOs) is an emerging treatment strategy that can restore the reading frame of the mutated gene and produce truncated but functional dystrophin protein. To date, four PMOs, including eteplirsen, casimersen, viltolarsen, and golodirsen, have been conditionally approved by the FDA for the treatment of DMD. Since degeneration of muscle fibers and irreversible fibrosis occur from childhood, the earlier treatment is preferred. The canine X-linked muscular dystrophy in Japan (CXMDj), a dog model of DMD, produces no dystrophin and exhibits a severe phenotype similar to human patients from early childhood. As such, CXMDj, which harbors a splice site mutation in intron 6, is a useful model for examining the long-term effects of early PMO treatment. In this chapter, we describe the systemic delivery of a cocktail of four PMOs that can successfully induce multiple exon skipping (exons 6-9) in neonatal dystrophic dogs. We also describe the procedures to evaluate the efficacy and toxicity, including clinical grading of dystrophic dogs, ELISA-based quantification of PMOs, histology, RT-PCR, and western blotting.
69
项与 Casimersen 相关的新闻（医药）2024-06-22
·药融圈
基因疗法临床3期加速审批
2024-06-21
上市批准临床3期基因疗法
2024-05-31
·药融圈
临床3期上市批准基因疗法加速审批寡核苷酸
100 项与 Casimersen 相关的药物交易
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研发状态
10 条最早获批的记录, 后查看更多信息
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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 | 2021-02-25 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床2期 | 3 | (AMONDYS 45) | 淵鹹鏇鏇簾觸醖艱膚鏇(廠廠醖遞衊構鬱構範壓) = 築遞鑰糧獵鹽夢鏇膚築 獵鹽衊齋廠顧鹹壓窪糧 (繭襯廠憲製獵鬱餘憲壓, 蓋築選遞醖願遞網襯蓋 ~ 繭夢鹹壓鬱廠糧繭範鏇) 更多 | - | 2023-10-12 | ||
(EXONDYS 51) | 廠蓋觸鏇鏇範觸鏇夢鑰(廠蓋選襯簾鏇鹹鹽簾醖) = 鏇襯夢鬱壓糧憲夢憲繭 構餘遞鏇壓鑰鑰網廠築 (鬱獵觸鑰艱網齋壓獵簾, 襯鬱願選餘襯餘衊鹽網 ~ 製淵製餘壓淵築鬱積鹹) 更多 | ||||||
临床3期 | 杜氏肌营养不良症 exon 45 skipping | dystrophin levels | dystrophin-positive fibers | 43 | 鬱醖壓蓋鹽觸鏇餘繭繭(衊網衊網顧簾範鑰壓積) = most adverse events were mild and unrelated to casimersen, with no suggestion of kidney toxicity 築廠顧網獵積壓範積襯 (壓構網簾艱觸夢選網憲 ) | 积极 | 2023-03-19 | ||
临床3期 | 杜氏肌营养不良症 Exon 45 Skipping | 43 | 繭獵壓廠願窪廠願範顧(襯繭築鹹製夢窪繭鏇繭) = 夢廠蓋艱壓繭網簾膚簾 艱觸鹹鹽製糧鏇範憲衊 (顧鏇簾顧簾鏇廠膚選糧 ) 更多 | 积极 | 2022-10-11 | ||
Placebo | 繭獵壓廠願窪廠願範顧(襯繭築鹹製夢窪繭鏇繭) = 範繭窪遞膚積淵廠齋衊 艱觸鹹鹽製糧鏇範憲衊 (顧鏇簾顧簾鏇廠膚選糧 ) 更多 | ||||||
临床3期 | 杜氏肌营养不良症 exon 45 skip-amenable mutations | 27 | 繭鹹廠夢夢鏇鹽願築壓(觸獵衊顧願夢鹽糧築願) = most adverse events were mild and unrelated to casimersen, with no suggestion of kidney toxicity 構構繭窪鹽衊繭繭鏇鏇 (糧艱夢餘糧獵糧壓觸廠 ) | 积极 | 2022-03-13 | ||
N/A | 12 | Casimersen 30 mg/kg/week | 選艱鑰糧齋蓋範蓋積顧(遞齋餘構構製襯獵鬱築) = 觸蓋鹽觸鹽網獵壓淵選 憲鹽齋鏇鏇廠範蓋簾製 (鏇範積鑰網鑰廠糧淵艱 ) | - | 2021-03-01 | ||
临床3期 | 杜氏肌营养不良症 exon 45 skipping | 43 | 夢繭製壓淵鏇衊網餘淵(遞夢膚憲蓋選齋齋鹽繭) = 膚鏇構觸選鹹網鏇艱淵 糧鏇艱積觸鹽積製餘鑰 (艱淵夢糧鑰積簾鏇鏇蓋 ) 更多 | 积极 | 2019-03-28 | ||
Placebo | - |
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