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UniQure

UniQure

needs to run another study to prove that its gene therapy 'actually helps people with Huntington's disease,' a senior U.S. Food and Drug Administration official said on a call with reporters Thursday.

需要进行另一项研究，以证明其基因疗法“确实有助于亨廷顿病患者”，美国食品和药物管理局一位高级官员在周四与记者的电话中表示。

The official, who requested anonymity before discussing sensitive information, confirmed the agency has asked the company to run a placebo controlled trial of its treatment, which is administered directly into the brain. UniQure has said that type of study isn't ethical because it would require putting people under general anesthesia for hours, a characterization the official disputed.

这位官员在匿名的情况下讨论了敏感信息，他证实该机构已要求该公司对其直接注入大脑的治疗方法进行安慰剂对照试验。UniQure 表示，这种研究是不道德的，因为它需要让患者全身麻醉数小时，而这位官员则对此说法提出异议。

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'So what is really going on? UniQure is the latest company to make a failed therapy for Huntington's patients,' the official said. 'They likely acknowledge or understand at some deep level that their trial failed years ago, and instead of doing the right thing and running the correct clinical study, UniQure is performing a distorted or manipulated comparison in the mind of FDA.'.

“那么到底发生了什么？UniQure是最新一家为亨廷顿病患者提供失败疗法的公司，”这位官员说。“他们可能在某种程度上承认或理解他们的试验多年前就失败了，但UniQure没有做正确的事情、进行恰当的临床研究，而是在FDA的意识中进行了一种扭曲或操纵的比较。”

The comments mark the latest development in a messy public spat between UniQure and the FDA, and as

这些评论标志着UniQure与FDA之间混乱的公开争端的最新发展，以及

the agency comes under fire

机构受到批评

for a number of recent drug approval application rejections, including some where companies have accused it of going back on previous guidance. FDA Commissioner Marty Makary in an interview with CNBC's

由于最近一些药物批准申请被拒绝，包括一些公司指责其违背了之前的指导。FDA专员马蒂·马卡里在接受CNBC采访时

Becky Quick

贝琪·奎克

last week seemingly

上周看似

criticized UniQure's gene therapy

批评了UniQure的基因疗法

for Huntington's disease. Makary didn't name UniQure but described its treatment.

针对亨廷顿病。马卡里没有点名UniQure，但描述了其治疗方法。

watch now

立即观看

VIDEO

视频

13:49

13:49

13:49

13:49

FDA Commissioner Dr. Makary on rare disease therapy approvals, internal politics at the agency

FDA专员马卡里博士谈罕见病治疗审批及该机构内部政治局势

Squawk Box

喧闹盒子

UniQure then accused the FDA of reversing its stance that the company's clinical trial data would be sufficient to seek approval. UniQure's study used an outside database to measure how patients with Huntington's disease might decline without treatment, known as an external control. UniQure has said it wouldn't be feasible to run a true randomized, double-blind placebo-controlled study, considered the gold standard, because it wouldn't be ethical to make people undergo a sham hours-long brain surgery.

UniQure随后指责FDA改变了其立场，即该公司的临床试验数据足以申请批准。UniQure的研究使用了一个外部数据库来衡量亨廷顿病患者在没有治疗的情况下可能会如何恶化，这被称为外部对照。UniQure表示，进行一个真正的随机双盲安慰剂对照研究（被认为是黄金标准）是不可行的，因为让患者接受数小时的假脑部手术是不道德的。

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The FDA official said the agency 'never agreed to accept this distorted comparison' and the FDA 'never makes such assurances.' Instead, the 'FDA will always say, 'Well, we have to see the data when we get it.''

美国食品和药物管理局官员说，该机构“从未同意接受这种扭曲的比较”，而且美国药管局“从不做这样的保证”。相反，美国药管局“总是说：‘好吧，我们拿到数据时必须看看。’”

UniQure didn't immediately comment.

UniQure没有立即置评。

The company's stock rose more than 10% on Thursday and has fallen 58% this year as of Thursday afternoon.

该公司股票在周四上涨了超过10%，而截至周四下午，今年以来已下跌了58%。