Roche has stopped enrolling and treating patients in three studies of the gene therapy Elevidys following the death of a 16-year-old patient with Duchenne muscular dystrophy.

罗氏公司已停止在 Elevidys 基因疗法的三项研究中招募和治疗患者，此前一名16岁的杜氏肌营养不良症患者死亡。

The European Medicines Agency asked for the temporary clinical hold until researchers and regulators finish an analysis into the young man’s cause of death, Roche said in

欧洲药品管理局要求暂时停止临床试验，直到研究人员和监管机构完成对该年轻男子死因的分析，罗氏表示。

to the patient community this week. Sarepta Therapeutics, which developed the therapy and

本周向患者群体推出了这种疗法的Sarepta Therapeutics公司

licensed it to Roche

将其授权给罗氏

in markets outside the U.S.,

在美国以外的市场，

announced the death last month

上个月宣布了死亡消息

The patient suffered from acute liver failure and also had a recent cytomegalovirus infection that might have contributed to his death. Sarepta

患者患有急性肝衰竭，近期还感染了巨细胞病毒，这可能与其死亡有关。赛雷普塔

noted

已备注

that no other cases of acute liver failure leading to death had been seen in the more than 800 patients treated with Elevidys to date, though liver injury is a known possible side effect for Elevidys and similar gene therapies.

在迄今为止接受Elevidys治疗的800多名患者中，尚未出现其他因急性肝衰竭导致死亡的病例，但肝损伤是Elevidys及类似基因疗法已知的可能副作用。

Dive Insight:

潜水洞察：

The clinical trial holds at European sites represent another setback for Elevidys, which has seen

Elevidys 在欧洲的临床试验受阻，这代表着该药物再次遭遇挫折。

strong sales

强劲的销售

even as questions persist about its benefits. The Food and Drug Administration originally

即使对其益处的质疑仍然存在。食品和药物管理局最初

approved Elevidys

批准了Elevidys

for a limited population of Duchenne patients in 2023 and then

针对2023年有限数量的杜氏患者，然后

expanded its clearance

扩大了其清除范围

the next year despite mixed results in testing of patients with the muscle-wasting condition.

尽管对患有肌肉萎缩症的患者进行的测试结果喜忧参半，但明年仍将继续。

News of the patient’s death in March sent Sarepta shares tumbling, and the slide continued Thursday, outpacing the larger market drop on the heels of

三月份患者死亡的消息使Sarepta的股价暴跌，而周四股价继续下滑，跌幅超过了紧随其后的大市下跌。

President Donald Trump’s tariff announcement

特朗普总统的关税声明

. Sarepta shares fell more than 7% to about $58 apiece in early trading. The stock traded above $100 before Sarepta announced the patient death on March 18.

Sarepta股价在早盘交易中下跌超过7%，至每股约58美元。在Sarepta于3月18日宣布患者死亡之前，该股交易价格高于100美元。

“This is yet another unfortunate development for a story that cannot seem to catch a break,” Leerink Partners analyst Joseph Schwartz wrote in a note to clients Wednesday.

“这一事件又迎来了不幸的发展，这个故事似乎无法得到一丝喘息，”Leerink Partners分析师Joseph Schwartz在周三给客户的一份报告中写道。

Still, he and other analysts said investors are now discounting Sarepta too much. “We acknowledge that Elevidys has taken a reputational hit as of late; however, the pendulum seems to have swung too far,” Schwartz said.

不过，他和其他分析师表示，投资者现在对Sarepta的估值折扣过大。施瓦茨说：“我们承认Elevidys近期声誉受损，但钟摆似乎摆得太过头了。”

The clinical holds could delay study results and affect the

临床控制可能会延迟研究结果并影响

ongoing review

持续审查

in Europe, but sales outside the U.S. would account for a relatively small portion of Sarepta’s revenue, analysts said. The holds only affect the enrollment and dosing of new patients; those who have already received the one-time treatment will still be monitored as researchers continue to collect data, Roche said..

在欧洲，但分析师表示，美国以外的销售额将占Sarepta收入的相对较小部分。罗氏公司称，该禁令仅影响新患者的入组和给药；而已接受过一次性治疗的患者仍将被继续监测，研究人员也会持续收集数据。

The three trials affected include one called Envision, which is a key global Phase 3 study of non-ambulatory patients and those between the ages of eight and 17 who can walk. Analysts had expected data from that trial in 2027.

受影响的三项试验包括一项名为Envision的试验，这是一项针对非卧床患者以及8至17岁能够行走的关键全球三期研究。分析师曾预计该试验的数据将在2027年发布。

In the U.S.,

在美国，

Elevidys

艾勒维迪斯

is currently cleared for patients who are at least four years old and have specific gene mutations. The therapy has full approval for patients in that group who can walk and an accelerated approval for non-ambulatory patients that must be confirmed by the Envision study.

目前已被批准用于至少四岁且具有特定基因突变的患者。该疗法已获得可行走患者群体的完全批准，以及针对非行走患者的加速批准，但需通过Envision研究确认。