NEW YORK - Novartis AG said on Wednesday it replaced the two top research and development executives at its Avexis unit after some data was manipulated from early testing of a gene therapy for infants that costs more than $2 million.
Avexis' Chief Scientific Officer Brian Kaspar and Senior Vice President of Research and Development Allan Kaspar have not been involved in any operations at Avexis since early May 2019, Novartis said in a statement.
The Kaspars are brothers. Brian Kaspar was a founder and key executive at Avexis and made more than $380 million from the company's 2018 acquisition by Novartis, according to SEC filings.
The Kaspars could not immediately be reached for comment.
Page Bouchard has taken on both those roles at Avexis as of Aug. 5, Novartis said.
The Swiss drugmaker said last week that it was in the process of "exiting" the Avexis scientists responsible for the manipulation of data on gene therapy Zolgensma. A Novartis spokesman said on Wednesday that a few other scientists beyond the Kaspars were no longer at the company but did not give any further details.
Novartis has not said who is responsible for the manipulated data.
The U.S. Food and Drug Administration said last week that Novartis had notified regulators in June - more than a month after Zolgensma had been approved - that some of the early testing data had been manipulated.
The company had been aware of the problems for as many as two months before the drug's U.S. approval, the FDA said, and it could face criminal or civil penalties.
Novartis said last week that it learned of allegations of data manipulation in mid-March and finished a preliminary investigation into the allegations in early May, confirming data discrepancies and raising data integrity concerns.
U.S. Senate Finance Committee Chairman Chuck Grassley sent a letter to Novartis last week asking the company to provide details about the manipulation by Aug. 23.
Novartis acquired Avexis last year, paying $8.7 billion for the maker of the rare-disease treatment, which is expected to bring in billions of dollars in sales.
Zolgensma - the world's most expensive drug - was approved as a one-time treatment for spinal muscular atrophy (SMA) in late May.
The disease often leads to paralysis, breathing difficulty and death within months for babies born with the most serious Type I form. SMA affects about one in 10,000 live births, with 50% to 70% having Type I disease, and is the leading genetic cause of death in infants.
纽约——诺华公司( Novartis AG )周三表示，该公司更换了旗下艾维斯公司( Avexis )的两名研发高管。此前，该公司的一些数据受到了操纵，因为该公司对婴儿进行基因治疗的早期测试花费了200多万美元。
诺华（Novartis）公司在一份声明中表示，自2019年5月初以来，艾维斯公司的首席科学官 Brian Kaspar 和研发高级副总裁 Allan Kaspar 从未参与过该公司的任何业务。
卡斯帕是兄弟。据 SEC 备案文件显示， Brian Kaspar 是 Avexis 的创始人和关键执行官，从诺华（Novartis）2018年收购该公司时赚了3.8亿多美元。
诺华（Novartis）表示，截至8月5日， Page Bouchard 已经在 Avexis 担任过这两个职位。
瑞士制药商上周表示，他们正在“退出” Avexis 的科学家，负责操纵基因治疗佐尔根瘤的数据。诺华（Novartis）公司的一位发言人周三表示，除了 Kaspas 之外，其他几位科学家已不再在该公司工作，但没有透露更多细节。
美国食品和药物管理局( FDA )上周表示，诺华（Novartis）已于6月通知监管机构，部分早期检测数据已被篡改。这是在佐尔甘斯曼获得批准一个多月之后。
诺华( Novartis )上周表示，该公司在3月中旬获悉了有关数据操纵的指控，并于5月初完成了对这些指控的初步调查，证实了数据差异，并引发了数据完整性方面的担忧。
诺华( Novartis )去年收购了艾维斯( Avexis )，斥资87亿美元收购了这家罕见疾病治疗制造商，预计将带来数十亿美元的销售额。
Zolgensma 是世界上最昂贵的药物，在五月底被批准作为一种一次性治疗脊柱肌肉萎缩症（ SMA ）。
这种疾病往往导致瘫痪，呼吸困难和死亡数月内出生的婴儿与最严重的 I 型。SMA 影响了大约十分之一的活产，50%到70%的人患有 I 型疾病，是婴儿死亡的主要遗传原因。