Biogen Alzheimer药物公司未能获得FDA小组的支持
Biogen Alzheimer’s Drug Fails to Gain FDA Panel’s Backing

Biogen Alzheimer’s Drug Fails to Gain FDA Panel’s Backing

Biogen Alzheimer药物公司未能获得FDA小组的支持

(布隆伯格)— —周五,生物制剂公司(Biogen Inc .)的阿尔茨海默氏症实验疗法未能得到美国食品与药品管理局(Food and Drug Administration ,简称FDA)顾问小组的支持,导致该药物在美国食品与药品管理局(Food and Drug Administration ,简称FDA)权衡批准时处于十字路口。

外部专家以8票赞成、 1票反对、 2票反对的结果尚未确定,一项临床试验的结果是肯定的,其数据不足以显示生物剂的药效。投票结果与林业发展局在会前编写的一份报告相矛盾,该报告支持这种名为“ aducanumab ”的药物的功效,尽管林业发展局内部存在异议。

顾问们表示,他们被要求评估的审判尚未结束,尽管数据似乎是正面的,但他们无法确定,特别是考虑到来自不同审判的相互矛盾的证据。

密苏里州圣路易斯华盛顿大学医学院(Washington University School of Medicine)的研究小组成员、神经学教授乔尔·珀尔穆特(Joel Perlmutter)说, “批准一些无效的东西有很大的危险。 ”

批准病原体疗法将是抗击阿尔茨海默氏症(Alzheimer ’ s)的漫长斗争中的一个里程碑。阿尔茨海默氏症是一种折磨人的脑消瘦疾病,影响到大约580万美国人。阿尔茨海默氏症协会(Alzheimer ’ s Association)在去年的一份报告中表示,虽然还有其他一些药物暂时改善了一些症状,但它们都没有减缓或阻止大脑神经元最终的致命破坏。

阿杜卡努马布(Aducanumab)也已成为生物剂的一种特别重要的药物。在过去两年里,由于阿杜卡努马布停止了对该药物的研究,重启了研究,然后于今年早些时候将其提交监管机构审批,该药物的市场价值大幅波动。

周五的整个正常交易日, Biogen的股票停牌。周三,在林业发展局(FDA)工作人员给出乐观评估后,该公司股价飙升44% ,尽管其中一些涨幅是在周四生物剂下滑7.5%时得到的。

Biogen的代表没有立即回应置评请求。

咨询委员会的建议不具约束力。林业局在考虑是否批准一种新药物时,经常召集专家小组,包括研究人员、医疗专业人员和病人代表。华尔街分析师表示,林业发展局似乎已开始积极看待这种药物。

瑞穗(Mizuho)分析师萨利姆•赛义德(Salim Syed)在写给彭博新闻社(Bloomberg News)的一封电子邮件中表示: “这将是林业发展局面临的一个考验,即林业发展局处于极端,而专家小组似乎处于另一个极端,科学和证据或缺乏证据是讨论的核心。 ”

动荡的道路

阿杜卡努马布的目标是在阿尔茨海默氏症患者的大脑中积攒起来的淀粉板,尽管科学家不知道它在该疾病中起着什么作用。脑部扫描显示,该药物去除了牌匾,但这是否有任何好处尚不清楚。

这一次,林业局没有遵循咨询小组的典型程序,首先是在会前与Biogen公司发布一份关于aducanumab临床试验数据的联合报告。林业发展局通常公布自己的报告,概述工作人员对数据的审查情况。

该机构拒绝回答为何将该报告合并在一起,以及生物剂是否能够在报告公布前对其进行审查的问题。该机构还允许小组成员在通常要求回答是赞成还是反对的情况下进行未经决定的表决。

这种疗法在林业发展局看来是一条曲折的道路。Biogen于2019年3月停止了对该药物的研究,原因是有迹象表明该药物不起作用。同年10月,该公司表示,对两项试验中的一项的数据进行的审查显示,该药物是成功的。

总部设在马萨诸塞州坎布里奇的生物剂公司在12月的一次会议上介绍了两项土豆蔻试验的数据。一项试验表明,这种药物可能减缓疾病的发展,而另一项试验则没有发现任何效果。研究人员对这一积极结果提出质疑,因为并非所有参与者都完成了试验。

FDA临床分析师凯尔·克鲁迪斯(Kyle Krudys)在一份有记录的报告中对咨询小组表示,该药物对患者没有任何影响的试验结果不应影响试验结果。Krudys说, “我的结论是,申请人提供了支持批准的有效证据。 ”

同时,林业发展局统计审查员Tristan Massie确定了第三个临床试验,直到完成,这对于确定aducanumab是否有效是必要的。

“在没有充分理由的情况下排除大型试验的数据是不科学的,在统计学上是不恰当的,也是误导性的, ”马西在为顾问准备的陈述中说。

`明显不一致'

新药物办公室林业发展局神经科学办公室主任Billy Dunn在会议开始时从积极的角度总结了林业发展局对该药物的看法,但没有提及Massie的关切。美国疾病控制与预防中心(Centers for Disease Control and Prevention)的数据显示,阿尔茨海默症是美国人第六大死亡原因。

“我们对阿尔茨海默症新药的需求非常敏感, ”邓恩说。

约翰·霍普金斯大学布隆伯格公共卫生学院(Johns Hopkins Bloomberg School of Public Health)的研究小组成员和流行病学家G ·卡莱布·亚历山大(G . Caleb Alexander)称,林业局的材料“极其不一致” ,并称马西的统计审查“做得很好” 。

亚历山大说, “感觉电视上的音频和视频不同步。 ”

彭博(Bloomberg)汇编的数据显示,如果aducanumab被清除, 2025年的销售额预计将达到47亿美元左右。Biogen与日本制药商Eisai Co .开发了这一疗法。

总部位于印第安纳波利斯的艾莉•利(Eli Lilly & amp); Co .目前正在研发几种阿尔茨海默(Alzheimer)实验药物,该公司的股价在委员会投票后下跌了4.4% 。和生物制剂的药物一样,利的donanemab的目标是从大脑中提取出清晰的淀粉胶质斑块。

(从第3段开始提供咨询会议的最新详细情况)

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2020 Bloomberg L . P .

(Bloomberg) -- Biogen Inc.'s experimental Alzheimer's disease therapy failed to gain support from a panel of U.S. Food and Drug Administration advisers on Friday, putting the drug at a crossroads as the agency weighs approval.

The outside experts voted 8 to 1, with 2 undecided, that data from a single clinical trial with positive results was insufficient to show Biogen's drug works. The vote contradicts a report FDA reviewers prepared ahead of the meeting that supported the efficacy of the drug, called aducanumab, though there was dissent in the agency.

Advisers said the trial they were asked to assess wasn't finished and even though the data appeared positive, they couldn't know for sure, particularly in light of conflicting evidence from a different trial.

"There's a huge danger in approving something that is not effective," said Joel Perlmutter, a panel member and neurology professor at Washington University School of Medicine in St. Louis, Missouri.

Approval of Biogen's therapy would be a milestone for the long battle against Alzheimer's, a harrowing brain-wasting disease that affects some 5.8 million Americans. While there are other drugs that temporarily improve some symptoms, none slow or stop the eventually lethal destruction of brain neurons, the Alzheimer's Association said in a report last year.

Aducanumab has also become a singularly important drug for Biogen, which has seen its market value fluctuate substantially over the past two years as it halted study on the drug, restarted its research, and then submitted it to regulators for approval earlier this year.

Shares of Biogen were halted throughout the regular trading day on Friday. On Wednesday, the stock surged 44% after the upbeat assessment from the FDA staff, though some of those gains were given back Thursday, when Biogen slid 7.5%.

Representatives for Biogen didn't immediately respond to a request for comment.

The advisory committee's recommendations aren't binding. The FDA often calls on panels of experts, including researchers, medical professionals and patient representatives, when it is considering whether to approve a new drug. Wall Street analysts said the FDA seemed set on viewing the drug positively.

"This will be a test for the FDA on what happens when the FDA is on one extreme and the panel is seemingly on the other, with science and evidence or the lack thereof being at the core of discussion," Mizuho analyst Salim Syed said in an email to Bloomberg News.

Tumultuous Path

Aducanumab targets amyloid plaque that builds up in the brains of Alzheimer's patients, though scientists don't know what role it plays in the disease. Brain scans showed the drug removed the plaque, but whether that had any benefit is unclear.

The FDA hasn't followed the typical process for advisory panels this time, starting with posting a joint report with Biogen on aducanumab's clinical trial data ahead of the meeting. Usually, the FDA posts its own report outlining staff's review of the data.

The agency declined to answer questions on why it combined the report and whether Biogen was able to review it prior to it being made public. The agency also gave panel members the option to vote undecided when typically it asks for yes or no answers.

The therapy has had a tumultuous path to FDA consideration. Biogen halted its study of the drug in March 2019 on signs it wouldn't work, then revived it in October that year after it said a review of data in one of two trials showed it was successful.

Biogen, based in Cambridge, Massachusetts, presented data from the two aducanumab trials at a conference in December. One trial showed the drug may slow the progression of the disease, while the other found no effect. Researchers questioned the positive results because not all participants completed the trials.

Kyle Krudys, a clinical analyst at the FDA, told the advisory panel in a recorded presentation that the trial results that showed no effect of the drug on patients shouldn't detract from the positive trial. "I have concluded that the applicant has provided substantial evidence of effectiveness to support approval," Krudys said.

Meanwhile, Tristan Massie, a statistical reviewer at the FDA, determined a third clinical trial followed through to completion is necessary to determine whether aducanumab works.

"Excluding data from a large trial without sufficient justification is unscientific, statistically inappropriate and misleading," Massie said in a prepared presentation for the advisers.

'Strikingly Incongruent'

Billy Dunn, the director of FDA's Office of Neuroscience in the Office of New Drugs, summed up the agency's view on the drug in a positive light at the beginning of the meeting without a nod to Massie's concerns. He focused on Alzheimer's ranking as the sixth leading cause of death among Americans, according to the Centers for Disease Control and Prevention.

"We are highly sensitive to the need for new drugs for Alzheimer's disease," Dunn said.

G. Caleb Alexander, a panel member and epidemiologist at Johns Hopkins Bloomberg School of Public Health, called the FDA's materials "strikingly incongruent" and said Massie's statistical review was "well done."

"It just feels like the audio and video on the TV are out of sync," Alexander said.

If aducanumab is cleared, sales are expected to total about $4.7 billion in 2025, according to data compiled by Bloomberg. Biogen developed the therapy with the Japanese drugmaker Eisai Co.

Indianapolis-based Eli Lilly & Co.has several experimental Alzheimer's drugs in development and shares of the company fell as much as 4.4% after the panel vote. Like Biogen's drug, Lilly's donanemab aims clear amyloid plaque from the brain.

(Updates with additional details from advisory meeting beginning in third paragraph)

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2020 Bloomberg L.P.

(Bloomberg) -- Biogen Inc.'s experimental Alzheimer's disease therapy failed to gain support from a panel of U.S. Food and Drug Administration advisers on Friday, putting the drug at a crossroads as the agency weighs approval.

(布隆伯格)— —周五,生物制剂公司(Biogen Inc .)的阿尔茨海默氏症实验疗法未能得到美国食品与药品管理局(Food and Drug Administration ,简称FDA)顾问小组的支持,导致该药物在美国食品与药品管理局(Food and Drug Administration ,简称FDA)权衡批准时处于十字路口。

The outside experts voted 8 to 1, with 2 undecided, that data from a single clinical trial with positive results was insufficient to show Biogen's drug works. The vote contradicts a report FDA reviewers prepared ahead of the meeting that supported the efficacy of the drug, called aducanumab, though there was dissent in the agency.

外部专家以8票赞成、 1票反对、 2票反对的结果尚未确定,一项临床试验的结果是肯定的,其数据不足以显示生物剂的药效。投票结果与林业发展局在会前编写的一份报告相矛盾,该报告支持这种名为“ aducanumab ”的药物的功效,尽管林业发展局内部存在异议。

Advisers said the trial they were asked to assess wasn't finished and even though the data appeared positive, they couldn't know for sure, particularly in light of conflicting evidence from a different trial.

顾问们表示,他们被要求评估的审判尚未结束,尽管数据似乎是正面的,但他们无法确定,特别是考虑到来自不同审判的相互矛盾的证据。

"There's a huge danger in approving something that is not effective," said Joel Perlmutter, a panel member and neurology professor at Washington University School of Medicine in St. Louis, Missouri.

密苏里州圣路易斯华盛顿大学医学院(Washington University School of Medicine)的研究小组成员、神经学教授乔尔·珀尔穆特(Joel Perlmutter)说, “批准一些无效的东西有很大的危险。 ”

Approval of Biogen's therapy would be a milestone for the long battle against Alzheimer's, a harrowing brain-wasting disease that affects some 5.8 million Americans. While there are other drugs that temporarily improve some symptoms, none slow or stop the eventually lethal destruction of brain neurons, the Alzheimer's Association said in a report last year.

批准病原体疗法将是抗击阿尔茨海默氏症(Alzheimer ’ s)的漫长斗争中的一个里程碑。阿尔茨海默氏症是一种折磨人的脑消瘦疾病,影响到大约580万美国人。阿尔茨海默氏症协会(Alzheimer ’ s Association)在去年的一份报告中表示,虽然还有其他一些药物暂时改善了一些症状,但它们都没有减缓或阻止大脑神经元最终的致命破坏。

Aducanumab has also become a singularly important drug for Biogen, which has seen its market value fluctuate substantially over the past two years as it halted study on the drug, restarted its research, and then submitted it to regulators for approval earlier this year.

阿杜卡努马布(Aducanumab)也已成为生物剂的一种特别重要的药物。在过去两年里,由于阿杜卡努马布停止了对该药物的研究,重启了研究,然后于今年早些时候将其提交监管机构审批,该药物的市场价值大幅波动。

Shares of Biogen were halted throughout the regular trading day on Friday. On Wednesday, the stock surged 44% after the upbeat assessment from the FDA staff, though some of those gains were given back Thursday, when Biogen slid 7.5%.

周五的整个正常交易日, Biogen的股票停牌。周三,在林业发展局(FDA)工作人员给出乐观评估后,该公司股价飙升44% ,尽管其中一些涨幅是在周四生物剂下滑7.5%时得到的。

Representatives for Biogen didn't immediately respond to a request for comment.

Biogen的代表没有立即回应置评请求。

The advisory committee's recommendations aren't binding. The FDA often calls on panels of experts, including researchers, medical professionals and patient representatives, when it is considering whether to approve a new drug. Wall Street analysts said the FDA seemed set on viewing the drug positively.

咨询委员会的建议不具约束力。林业局在考虑是否批准一种新药物时,经常召集专家小组,包括研究人员、医疗专业人员和病人代表。华尔街分析师表示,林业发展局似乎已开始积极看待这种药物。

"This will be a test for the FDA on what happens when the FDA is on one extreme and the panel is seemingly on the other, with science and evidence or the lack thereof being at the core of discussion," Mizuho analyst Salim Syed said in an email to Bloomberg News.

瑞穗(Mizuho)分析师萨利姆•赛义德(Salim Syed)在写给彭博新闻社(Bloomberg News)的一封电子邮件中表示: “这将是林业发展局面临的一个考验,即林业发展局处于极端,而专家小组似乎处于另一个极端,科学和证据或缺乏证据是讨论的核心。 ”

Tumultuous Path

动荡的道路

Aducanumab targets amyloid plaque that builds up in the brains of Alzheimer's patients, though scientists don't know what role it plays in the disease. Brain scans showed the drug removed the plaque, but whether that had any benefit is unclear.

阿杜卡努马布的目标是在阿尔茨海默氏症患者的大脑中积攒起来的淀粉板,尽管科学家不知道它在该疾病中起着什么作用。脑部扫描显示,该药物去除了牌匾,但这是否有任何好处尚不清楚。

The FDA hasn't followed the typical process for advisory panels this time, starting with posting a joint report with Biogen on aducanumab's clinical trial data ahead of the meeting. Usually, the FDA posts its own report outlining staff's review of the data.

这一次,林业局没有遵循咨询小组的典型程序,首先是在会前与Biogen公司发布一份关于aducanumab临床试验数据的联合报告。林业发展局通常公布自己的报告,概述工作人员对数据的审查情况。

The agency declined to answer questions on why it combined the report and whether Biogen was able to review it prior to it being made public. The agency also gave panel members the option to vote undecided when typically it asks for yes or no answers.

该机构拒绝回答为何将该报告合并在一起,以及生物剂是否能够在报告公布前对其进行审查的问题。该机构还允许小组成员在通常要求回答是赞成还是反对的情况下进行未经决定的表决。

The therapy has had a tumultuous path to FDA consideration. Biogen halted its study of the drug in March 2019 on signs it wouldn't work, then revived it in October that year after it said a review of data in one of two trials showed it was successful.

这种疗法在林业发展局看来是一条曲折的道路。Biogen于2019年3月停止了对该药物的研究,原因是有迹象表明该药物不起作用。同年10月,该公司表示,对两项试验中的一项的数据进行的审查显示,该药物是成功的。

Biogen, based in Cambridge, Massachusetts, presented data from the two aducanumab trials at a conference in December. One trial showed the drug may slow the progression of the disease, while the other found no effect. Researchers questioned the positive results because not all participants completed the trials.

总部设在马萨诸塞州坎布里奇的生物剂公司在12月的一次会议上介绍了两项土豆蔻试验的数据。一项试验表明,这种药物可能减缓疾病的发展,而另一项试验则没有发现任何效果。研究人员对这一积极结果提出质疑,因为并非所有参与者都完成了试验。

Kyle Krudys, a clinical analyst at the FDA, told the advisory panel in a recorded presentation that the trial results that showed no effect of the drug on patients shouldn't detract from the positive trial. "I have concluded that the applicant has provided substantial evidence of effectiveness to support approval," Krudys said.

FDA临床分析师凯尔·克鲁迪斯(Kyle Krudys)在一份有记录的报告中对咨询小组表示,该药物对患者没有任何影响的试验结果不应影响试验结果。Krudys说, “我的结论是,申请人提供了支持批准的有效证据。 ”

Meanwhile, Tristan Massie, a statistical reviewer at the FDA, determined a third clinical trial followed through to completion is necessary to determine whether aducanumab works.

同时,林业发展局统计审查员Tristan Massie确定了第三个临床试验,直到完成,这对于确定aducanumab是否有效是必要的。

"Excluding data from a large trial without sufficient justification is unscientific, statistically inappropriate and misleading," Massie said in a prepared presentation for the advisers.

“在没有充分理由的情况下排除大型试验的数据是不科学的,在统计学上是不恰当的,也是误导性的, ”马西在为顾问准备的陈述中说。

'Strikingly Incongruent'

`明显不一致'

Billy Dunn, the director of FDA's Office of Neuroscience in the Office of New Drugs, summed up the agency's view on the drug in a positive light at the beginning of the meeting without a nod to Massie's concerns. He focused on Alzheimer's ranking as the sixth leading cause of death among Americans, according to the Centers for Disease Control and Prevention.

新药物办公室林业发展局神经科学办公室主任Billy Dunn在会议开始时从积极的角度总结了林业发展局对该药物的看法,但没有提及Massie的关切。美国疾病控制与预防中心(Centers for Disease Control and Prevention)的数据显示,阿尔茨海默症是美国人第六大死亡原因。

"We are highly sensitive to the need for new drugs for Alzheimer's disease," Dunn said.

“我们对阿尔茨海默症新药的需求非常敏感, ”邓恩说。

G. Caleb Alexander, a panel member and epidemiologist at Johns Hopkins Bloomberg School of Public Health, called the FDA's materials "strikingly incongruent" and said Massie's statistical review was "well done."

约翰·霍普金斯大学布隆伯格公共卫生学院(Johns Hopkins Bloomberg School of Public Health)的研究小组成员和流行病学家G ·卡莱布·亚历山大(G . Caleb Alexander)称,林业局的材料“极其不一致” ,并称马西的统计审查“做得很好” 。

"It just feels like the audio and video on the TV are out of sync," Alexander said.

亚历山大说, “感觉电视上的音频和视频不同步。 ”

If aducanumab is cleared, sales are expected to total about $4.7 billion in 2025, according to data compiled by Bloomberg. Biogen developed the therapy with the Japanese drugmaker Eisai Co.

彭博(Bloomberg)汇编的数据显示,如果aducanumab被清除, 2025年的销售额预计将达到47亿美元左右。Biogen与日本制药商Eisai Co .开发了这一疗法。

Indianapolis-based Eli Lilly & Co.has several experimental Alzheimer's drugs in development and shares of the company fell as much as 4.4% after the panel vote. Like Biogen's drug, Lilly's donanemab aims clear amyloid plaque from the brain.

总部位于印第安纳波利斯的艾莉•利(Eli Lilly & amp); Co .目前正在研发几种阿尔茨海默(Alzheimer)实验药物,该公司的股价在委员会投票后下跌了4.4% 。和生物制剂的药物一样,利的donanemab的目标是从大脑中提取出清晰的淀粉胶质斑块。

(Updates with additional details from advisory meeting beginning in third paragraph)

(从第3段开始提供咨询会议的最新详细情况)

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