欧洲药品管理局拒绝了Sarepta Therapeutics公司针对杜氏肌营养不良症的基因疗法Elevidys-动脉网

EMA Rejects Sarepta Therapeutics’ Gene Therapy Elevidys for Duchenne Muscular Dystrophy

GeneOnline 等信源发布 2025-07-25 22:07

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by Mark Chiang

马克·蒋

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The European Medicines Agency (EMA) has issued a negative opinion regarding Sarepta Therapeutics’ gene therapy, Elevidys, intended for the treatment of Duchenne muscular dystrophy. The decision follows an evaluation of clinical testing data, which the agency determined failed to demonstrate sufficient evidence of the therapy’s benefit.

欧洲药品管理局（EMA）对Sarepta Therapeutics公司用于治疗杜氏肌营养不良症的基因疗法Elevidys发表了否定意见。该决定是在评估临床试验数据后作出的，EMA认为这些数据未能充分证明该疗法的益处。

This development adds to mounting challenges for Sarepta and raises concerns about its financial outlook..

这一发展增加了Sarepta面临的挑战，并引发了对其财务前景的担忧。

Elevidys is designed as a gene therapy aimed at addressing Duchenne muscular dystrophy, a rare and progressive genetic disorder that primarily affects boys and leads to muscle degeneration. Sarepta had sought approval from the EMA based on its clinical trials; however, regulators concluded that the submitted data did not adequately establish the efficacy of the treatment.

Elevidys是一种旨在治疗杜氏肌营养不良症的基因疗法，这是一种罕见且渐进的遗传性疾病，主要影响男孩并导致肌肉退化。Sarepta基于其临床试验向欧洲药品管理局（EMA）寻求批准；然而，监管机构认为提交的数据未能充分证明该治疗的有效性。

The negative opinion represents a significant setback for Sarepta in its efforts to expand Elevidys into European markets and could have broader implications for its operations moving forward..

负面意见对Sarepta努力将Elevidys扩展到欧洲市场的计划构成了重大挫折，并可能对其未来的发展产生更广泛的影响。

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Source: GO-AI-ne1

来源：GO-AI-ne1

Date: July 25, 2025

日期：2025年7月25日

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