史上最贵药物存数据造假，FDA对诺华展开调查
当地时间8月6日，FDA发布声明称，诺华最近获批的基因疗法——Zolgensma存在部分临床前“数据操纵”（>
Zolgensma于今年5月24日被批准用于治疗2岁以下，在两个编码运动神经元生存蛋白（SMN）的SMN1等位基因上携带突变的脊髓性肌肉萎缩症（SMA）患者，定价高达210万美元，被称为“史上最贵药物”，其研发生产公司为诺华子公司AveXis。如下：
Amid a fresh scandal, this time over data manipulation for the world’s most expensive med, top Novartis execs laid out a detailed account Wednesday and defended the company’s decision not to inform the FDA until after its own internal probe wrapped up.In the end, CEO Vas Narasimhan said the company did the “bestwe could."
诺华 Zolgensma，这个全球最昂贵的药物，爆出一个新的丑闻：数据处理过程中存在数据操纵，在公司发现这一问题时，Zolgensma正处于FDA审批阶段，但公司并未向FDA报告这一问题。诺华曾为该公司决定在其内部调查结束后不通知FDA做出辩护。首席执行官Vas Narasimhan说公司做到了“我们能做到的最好”。
According to Novartis" timeline, anAveXisscientist stepped up in mid-March to report the manipulated data, which the FDA said wassubmitted as part of Zolgensma"s application for approval. The company launched an internal probe, and by early May had determined the scientist was correct. It didn"t tell the FDA about the allegations, however, until June 28, more than a month after Zolgensma won its FDA green light.
根据诺华公司的提供的时间表，一名AveXis科学家在3月中旬紧急报告了数据操纵事件，FDA称这些数据是作为Zolgensma申请批准的一部分提交的。该公司发起了内部调查，并在5月初确定该科学家是正确的。然而，直到6月28日，在Zolgensma赢得FDA批准的一个多月之后，才告知FDA这些问题。
The company is in the process of "exiting the small number ofAveXisscientists involved," CEO Vas Narasimhansaid during the call.
该公司正在“清退部分涉及的AveXis科学家”，首席执行官Vas Narasimhan在电话会议中表示。
The data dust-up is the latest in a series of scandals at Novartis and the second major controversy to surface since Narasimhan took the reins. The CEO has said rehabbing the company"s reputation is one of his top goals as chief executive. But now Novartis has to not only spruce up its image, but regain trust at the FDA, which made its own grievances very public on Tuesday.
数据作假是诺华公司一系列丑闻中的最新消息。首席执行官表示，恢复公司的声誉是他作为首席执行官的首要目标之一。但现在诺华公司不仅要改善自己的形象，还要重新获得FDA的信任，因为FDA周二已公布了不满情绪。
Narasimhan said，theinternal investigation that started in Marchaimed to confirmthat the scientist"s claims were true and to determine whether the data problem would affect Zolgensma"s efficacy, safety or quality.
Narasimhan表示：3月份发起的内部调查是确认科学家的说法是否属实，并确定数据问题是否会影响Zolgensma的功效，安全性或质量。
Meanwhile, the FDA was reviewing Zolgensma"s application and approved it on May 24, without knowing about the scientist"s allegations or Novartis" internal probe. The company launched its new drug, which treats babies two years or younger with spinal muscular atrophy, at a sticker price of $2.1 million, making it the most expensive drug in the world.
与此同时，FDA正在审查Zolgensma的申请并于5月24日批准，但不知道科学家的指控和诺华的内部调查。此后，该公司推出该新药，以210万美元的价格用于治疗患有脊髓性肌萎缩症的两岁或两岁以下的婴儿，使其成为世界上最昂贵的药物。
The FDA on Tuesday took an unusually harsh tone over Novartis’ conduct. Acting chief Ned Sharpless tweeted that theregulator is considering civil and criminal punishments, and the agency sent out a more detailed announcement to the media.
FDA本周二对诺华的行为采取了异乎寻常的严厉态度。代理首席执行官内德·夏普莱斯（Ned Sharpless）在推特上表示，监管机构正在考虑民事和刑事处罚，该机构向媒体发出了更详细的通知。
来源 gmp在线
