The data-manipulation scandal with Novartis is widening, undermining investors’ trust in company chief executive officer Vas Narasimhan.
In early August, the U.S. Food and Drug Administration (FDA) announced that the data involved in the preclinical approval process of Novartis’ gene therapy Zolgensma for spinal muscular atrophy (SMA) was manipulated. The therapy, which was developed by AveXis, a Novartis subsidiary, was approved by the agency in May.
In the original announcement, the FDA said AveXis informed the agency “about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application.” The data was used by the company to support the development of its production process, and the data does not seem to change the agency’s positive assessment of the information from the human clinical trials.
But the U.S. Senate has weighed in on the issue. Specifically, U.S. Senate Finance Committee Chairman Chuck Grassley (R-IA) has requested details about the data manipulation. Five senators, including presidential candidates Elizabeth Warren (D-MA) and Bernie Sanders (I-VT) wrote a letter to the FDA urging it to take forceful action.
Reportedly the FDA has referred the matter to its Office of Criminal Investigations to launch a preliminary inquiry. Submitting false data to the FDA as part of a New Drug Application (NDA) could be a crime if the investigation finds the actions were intentional.
In what might be a coincidence, an unnamed Novartis executive sold $946,000 worth of company shares less than three weeks before the agency announcement.
A Novartis spokesman told Reuters, “As is usual in such cases, the transaction was thoroughly checked beforehand and then approved accordingly. The person in question was not in possession of relevant material information.”
However, it’s just one more scandal the company’s Narasimhan has to handle, who took over the reins in February 2018. Part of his first and apparently increasingly difficult duties was to rebuild trust in the company. One of the first issues he had to tackle was the finding that the company paid $1.2 million to President Trump’s former personal lawyer, Michael Cohen, for “insight” into the administration’s healthcare policy. That decision was made before Narasimhan took over, and he has called the decision a mistake. He is also overseeing the company’s negotiation with the U.S. to settle a lawsuit alleging the company basically bribed physicians with expensive dinners and other events in return for them prescribing company drugs. Novartis has set aside $700 million for a settlement.
“He’s certainly in big danger of losing credibility,” Brad Loncar, chief executive officer of Loncar Investments, an independent biotech investor and analyst, told The Wall Street Journal. “They’ve handled this is just about the worst way possible.” Loncar does not hold Novartis shares.
So far that action has included ousting five of the scientists involved in the scandal and appointing a new chief scientific officer for AveXis. AveXis has also parted ways with its scientific founder and chief scientific officer. Brian Kaspar, chief scientific officer, and Allan Kaspar, the company’s head of research and development, are no longer with the company and, according to AveXis, have not been involved in AveXis operations since May.
Page Bouchard, who has been with Novartis for 10 years, is taking over their roles.
Novartis acquired AveXis in 2018 for $8.7 billion. Novartis has stood behind the data submitted to the FDA. The company indicates the questionable assays were used for initial product testing but are not in use for the commercial product release.
One reason this particular gene therapy is receiving so much attention is its price tag, which is $2.1 million. It is a one-time gene therapy that appears to cure the disease. It was only recently treatable. That other treatment is Biogen’s Spinraza, which has a price tag of $750,000 for the first year and $375,000 for every year after that for the life of the patient. It is approved for all forms of SMA, types 0 through 5. At this time, Zolgensma is only approved for SMA type 1, the severest form. It is almost always fatal by two years of age, with a 50% mortality rate by 7 months and a 90% mortality rate by 12 months.
诺华( Novartis )的数据操纵丑闻正在扩大，削弱了投资者对该公司首席执行官瓦什•纳拉希姆( Vas Narasimhan )的信任。
8月初，美国食品药品监督管理局（ FDA ）宣布，诺华（Novartis）基因治疗佐尔甘斯瘤用于脊柱肌肉萎缩症（ SMA ）的临床前审批过程中涉及的数据被篡改。该疗法是由诺华（Novartis）的子公司 AveXis 开发的，于5月份获得了该机构的批准。
FDA 在最初的声明中表示， AveXis 公司向 FDA 通报了“一个数据操纵问题，这会影响生物制剂许可申请中提交的动物产品测试的某些数据的准确性”。该数据被公司用于支持其生产工艺的开发，该数据似乎没有改变该机构对人体临床试验信息的积极评估。
但美国参议院对这个问题进行了权衡。具体而言，美国参议院财政委员会主席查克·格拉斯利（ R-IA ）要求提供有关数据操纵的细节。包括总统候选人伊丽莎白·沃伦（ ElizabethWarren ）和伯尼·桑德斯（ I-VT ）在内的五位参议员致函 FDA ，敦促其采取有力行动。
据报道， FDA 已将此事提交其刑事调查办公室展开初步调查。如果调查发现这些行为是故意的，向 FDA 提交虚假数据作为新药申请（ NDA ）的一部分可能是犯罪。
然而，这只是该公司 Narasimhan 不得不处理的又一桩丑闻，他在2018年2月接管了该公司。他的第一个明显越来越困难的任务是重建对公司的信任。他首先要解决的问题之一是，该公司向特朗普总统的前私人律师迈克尔·科恩( Michael Cohen )支付了120万美元，以“洞察”政府的医疗政策。这个决定是在 Narasimhan 接手之前做出的，他把这个决定称为一个错误。他还负责监督该公司与美国的谈判，以了结一项诉讼，指控该公司基本上以昂贵的晚餐和其他活动贿赂医生，以换取他们开公司的药品。诺华（Novartis）已拨出7亿美元用于和解。
独立生物技术投资者和分析师 LonCar Investments 首席执行官布拉德•伦奇( Brad Loncar )对《华尔街日报》表示：“他肯定面临失去信誉的巨大危险。”“他们处理这件事的方式几乎是最糟糕的。”Loncar 不持有诺华（Novartis）股份。
到目前为止，这一行动包括驱逐5名卷入这一丑闻的科学家，并任命一名新的首席科学官。AveXis 也与其科学创始人和首席科学家分道扬镳。首席科学官布赖恩·卡斯帕( Brian Kaspar )和公司研发主管艾伦·卡斯帕( Allan Kaspar )已不再与该公司合作，根据 AveXis 的说法，自5月份以来，一直未参与 AveXi 的运营。
诺华（Novartis）于2018年以87亿美元收购了 AveXis 。诺华（Novartis）公司一直支持向 FDA 提交的数据。该公司表示，可疑的检测用于初始产品测试，但不用于商业产品发布。
这种特殊的基因疗法之所以受到如此广泛的关注，一个原因是它的价格标签是210万美元。这是一种一次性的基因治疗，似乎可以治愈疾病。这只是最近才可以处理的。另一种治疗方法是百健（Biogen）公司的 Spiraza ，第一年的价格为75万美元，之后每年的价格为37.5万美元，用于患者的生命。它被批准用于所有形式的 SMA ，类型0到5。此时， Zolgensma 只被批准用于 SMA 类型1，最严重的形式。它几乎总是致命的两年年龄，50％死亡率7个月和90％死亡率12个月。