Avidity Biosciences获得FDA加速审批协议，用于FSHD治疗-动脉网

Avidity Biosciences Secures FDA Accelerated Filing Agreement for FSHD Therapy

GeneOnline 等信源发布 2025-06-20 17:13

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by Mark Chiang

由马克·蒋

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Avidity Biosciences has reached an agreement with the U.S. Food and Drug Administration (FDA) regarding the accelerated filing of its experimental therapy for facioscapulohumeral muscular dystrophy (FSHD), a rare genetic disorder. The arrangement addresses concerns within the pharmaceutical industry about regulatory pathways for rare disease treatments, signaling potential progress in expediting drug development for underserved patient populations..

Avidity Biosciences 已与美国食品药品监督管理局 (FDA) 就其针对面肩肱型肌营养不良症 (FSHD) 的实验性疗法的加速提交达成协议，这是一种罕见的遗传性疾病。该协议解决了制药行业对罕见病治疗监管路径的担忧，标志着在加快服务不足患者群体的药物开发方面可能取得进展。

The agreement centers on Avidity’s investigational drug designed to treat FSHD, a condition characterized by progressive muscle weakness and wasting. The FDA’s decision to allow an accelerated filing process reflects ongoing efforts to streamline approvals for therapies targeting rare diseases, which often lack effective treatment options.

该协议围绕Avidity公司用于治疗FSHD的在研药物，这种疾病的特点是进行性肌肉无力和萎缩。FDA决定允许加速提交审批的决定反映了为针对罕见病的疗法简化审批流程的持续努力，这些疾病通常缺乏有效的治疗选择。

Industry observers have noted that such agreements could reduce uncertainty surrounding regulatory requirements and timelines, potentially encouraging further investment in rare disease research and development..

行业观察人士指出，此类协议可减少围绕监管要求和时间表的不确定性，可能会鼓励对罕见病研发的进一步投资。

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Source: GO-AI-ne1

来源：GO-AI-ne1

Date: June 9, 2025

日期：2025年6月9日

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