Fordadistrogene movaparvovec

试验暂停：欧洲药监局紧急行动近期，萨雷普塔疗法公司（Sarepta Therapeutics）与罗氏合作研发的杜氏肌营养不良症（Duchenne muscular dystrophy，DMD）基因疗法 Elevidys 在欧洲遭遇重大挫折。欧洲药品管理局（EMA）要求萨雷普塔和罗氏暂停三项关于 Elevidys 的临床试验，这一决定源于一名美国青少年在接受该疗法后出现急性肝衰竭并死亡。3 月 31 日，罗氏在致世界杜氏肌营养不良症组织的信中透露，公司已暂停 Elevidys 的 I 期、II 期和 III 期临床试验。这些试验均旨在研究该基因疗法对杜氏肌营养不良症的治疗效果。EMA 表示，在确定这名青少年的确切死因之前，试验将一直处于暂停状态。萨雷普塔上个月在患者死亡时就已宣布，会更新 Elevidys 的标签以体现这一安全信号。各方反应：市场与行业观点分化对于此次试验暂停，市场分析师看法不一。蒙特利尔银行（BMO）的分析师相对乐观，他们认为此次临床暂停只是行政举措，在数据监测委员会会议后有望解决。考虑到杜氏肌营养不良症是一种致命疾病，Elevidys 的获益风险比使其有望在约 6 个月内恢复试验。而杰富瑞（Jefferies）的分析师则更为谨慎。他们指出，虽然致命性急性肝衰竭的风险较低（0.125%），但在萨雷普塔管理层未解答一些关键问题之前，不能忽视这起死亡事件。这反映出在面对此类安全事件时，市场对该疗法的未来存在一定担忧。企业态度：承诺透明与患者社区互动在这起事件中，萨雷普塔和罗氏都非常重视与患者社区的沟通。萨雷普塔在事件发生后，直接向杜氏肌营养不良症患者群体致信，承诺在调查死亡原因的过程中保持透明，并及时分享相关数据。罗氏在本周早些时候给患者群体的信中也表达了类似的态度，强调与患者社区合作的重要性。这种紧密的互动体现了生物医药企业对患者权益的重视，也有助于稳定患者群体的信心。行业反思：基因疗法的安全之殇这起死亡事件并非个例，杜氏肌营养不良症基因疗法领域此前就出现过多起类似悲剧。2021 年和 2024 年，分别有患者在接受辉瑞的研究性基因疗法 fordadistrogene movaparvovec 时死亡，该项目随后被终止。2022 年，一种基于 CRISPR 技术的杜氏肌营养不良症疗法也导致了一名患者死亡，可能是由所用的病毒载体引发。这些事件为整个基因疗法行业敲响了警钟。尽管基因疗法为攻克疑难病症带来了新希望，但安全问题始终是悬在行业头顶的达摩克利斯之剑。在追求创新和疗效的同时，如何确保患者安全是生物医药企业和监管机构需要共同面对的重大课题。此次 Elevidys 试验暂停事件，也让行业再次审视基因疗法的安全性评估和监测机制，期待后续能有更严格的标准和措施，保障患者在接受前沿治疗时的生命健康。参考来源：https://www.biospace.com/drug-development/sareptas-duchenne-gene-therapy-placed-on-hold-in-europe-pending-death-investigation识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。

Courtesy of Sarepta Therapeutics The European Medicines Agency asked for three clinical trials to be placed on hold until the exact cause of death of a US teenager, who suffered acute liver failure after receiving Elevidys, can be precisely determined. The European Medicines Agency has requested that Sarepta Therapeutics and partner Roche pause a trio of clinical trials of their Duchenne muscular dystrophy gene therapy Elevidys in response to a patient death that occurred outside the scope of the studies. In a letter addressed to the World Duchenne Organization and dated March 31, Roche shared that it was pausing Phase I , Phase II and Phase III trials all studying the gene therapy for treating Duchenne muscular dystrophy (DMD). The European Medicines Agency (EMA) asked for the trials to be paused until the exact cause of death of a U.S. teenager , who had suffered acute liver failure (ALF) after taking Elevidys, can be precisely determined. Sarepta announced last month at the time of the patient’s death that it would update Elevidys’ label to reflect the safety signal. Analysts at BMO were nonplussed by the trial pause. “We view the requested clinical hold as an administrative step, pending [data monitoring committee] meeting, that can be resolved within ~6mo given Elevidys benefit/risk in the context of DMD being a universally fatal disease ,” the analysts wrote in a note late Wednesday (emphasis theirs). Other analysts were more mixed. “On one hand, the risk of fatal ALF is rare (0.125%) and our experts affirm the news is unsurprising to them. However, we’ve felt the death should not be fully dismissed unless [Sarepta management] can provide color on some outstanding questions,” Jefferies analysts wrote Wednesday. The connection between biopharmas working on DMD and the patient community is especially tight knit . Following the patient’s death, Sarepta sent a letter directly to the DMD patient community, pledging transparency and sharing data as the company investigates the death. The sentiment mirrors those reflected in Roche’s letter earlier this week to the DMD community. The company signed off saying, “We truly appreciate your leadership and the opportunity to work together with you in these efforts.” The patient death after taking Elevidys is one of a series of deaths from DMD gene therapies. Two patients died, one in 2021 and another in 2024 , while taking Pfizer’s investigational gene therapy fordadistrogene movaparvovec. That program was later scrapped. In 2022, a CRISPR-based Duchenne therapy led to another patient death, potentially caused by the viral vector used.