FDA跳过Capricor的杜氏肌营养不良细胞疗法的咨询委员会-动脉网

FDA Skips Advisory Committee For Capricor's Duchenne Cell Therapy

benzinga 等信源发布 2025-06-24 14:51

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Capricor Therapeutics Inc

卡普里科尔治疗公司

CAPR

山羊

on Tuesday provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the company’s lead cell therapy candidate for the treatment of Duchenne Muscular Dystrophy (DMD)—

周二提供了与其生物制品许可申请（BLA）相关的监管更新，该申请涉及公司用于治疗杜氏肌营养不良症（DMD）的主要细胞疗法候选药物Deramiocel——

associated cardiomyopathy

Also Read:

另请阅读：

Vinay Prasad’s Appointment To FDA’s CBER Triggers Questions Over Future Of Cell And Gene Therapy Regulation

Vinay Prasad任职于FDA的CBER引发了关于细胞和基因治疗监管未来的疑问。

As part of the FDA’s ongoing review, the company has been informed that an Advisory Committee meeting is not indicated at this time.

作为 FDA 持续审查的一部分，公司已被告知目前不需要召开咨询委员会会议。

The BLA remains under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025.

BLA仍在优先审查中，处方药使用者费用法案（PDUFA）的目标行动日期为2025年8月31日。

As per some media reports,

根据一些媒体报道，

Vinay Prasad

文奈·普拉萨德

, director of the FDA’s Center for Biologics Evaluation and Research (CBER), canceled the advisory committee meeting for the company’s cell therapy for

，FDA生物制品评估与研究中心（CBER）主任，取消了该公司细胞疗法的咨询委员会会议，因为

Duchenne muscular dystrophy

杜氏肌营养不良症

。

The FDA informed Capricor of its intent to hold the Advisory Committee meeting on July 30, 2025, although that date is pending confirmation by the FDA. At the time of the mid-cycle review, no significant issues or major

FDA通知Capricor，计划于2025年7月30日召开咨询委员会会议，但该日期尚待FDA确认。在中期审查时，未发现重大问题或主要问题。

deficiencies were noted

发现了缺陷

。

Linda Marbán, CEO of Capricor, says the company has hit all key regulatory milestones as planned, including a successful pre-license inspection and a smooth mid-cycle review. With the application still under Priority Review, she believes Capricor is in a strong position heading into the PDUFA date.

琳达·马尔布兰，Capricor公司首席执行官，表示公司已按计划达成了所有关键的监管里程碑，包括成功的预许可检查和顺利的中期审查。由于申请仍在优先审查中，她认为Capricor在进入PDUFA日期时处于非常有利的地位。

Capricor recently presented four-year data from its HOPE-2 Open-Label Extension (OLE) study at the 2025 Parent Project Muscular Dystrophy (PPMD) Conference, representing one of the longest-running treatment datasets in DMD, including measures of both cardiac and skeletal muscle function.

Capricor最近在2025年父母项目肌肉萎缩症（PPMD）会议上展示了其HOPE-2开放标签扩展（OLE）研究的四年数据，这是DMD中最长的治疗数据集之一，包括心脏和骨骼肌功能的测量。

Price Action:

价格行为：

CAPR stock is up 20% at $9.22 at the last check on Tuesday

CAPR股票在周二最后一次检查时上涨了20%，达到9.22美元。

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Image: Shutterstock

图片：Shutterstock