Drugmaker Novartis’ gene therapy subsidiary has fingered two former executives as the culprits in a recent scandal over manipulation of data for its approved gene therapy.
The Basel, Switzerland-based pharmaceutical company on Tuesday announced the posting on the Food and Drug Administration website of a 59-page response, dated Aug. 23, to the FDA’s Form 483, from Novartis’ AveXis division, about the data manipulation scandal concerning the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), which the FDA approved in May for spinal muscular atrophy in infants.
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The scandal erupted last month when the FDA revealed that certain mouse data from an in vivo relative potency assay, or IVRPA, had been manipulated, and that Novartis had been aware of the issue two months before the therapy’s approval, but did not inform the FDA until one month after the approval. The issue predated AveXis’ acquisition by Novartis in April 2018, for $8.7 billion. The company had ceased using the IVRPA about eight months before Novartis learned of the issue, which the FDA has said does not affect Zolgensma’s approval.
In the wake of the news, AveXis said that two former executives, brothers Brian and Allan Kaspar, who had respectively served as head of research and development and chief scientific officer, were “no longer with the company.” According to subsequent media reports, the Kaspars were ousted from the company in connection with the scandal.
In the document posted Tuesday, Novartis stated, “[It] was alleged that two AveXis senior executives altered or instructed others to alter a small amount of raw data used to run the IVRPA. Such conduct is unacceptable, and the two AveXis senior executives have been terminated.”
The document is redacted in several places and does not appear to refer to the Kaspars by name. Allan Kaspar did not immediately respond to a request for comment. In an emailed statement, attorney John Hueston, who is representing Brian Kaspar, defended his client.
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“AveXis shamelessly attempts to blame others for its own disclosure decisions to the FDA,” Hueston wrote. “Contrary to AveXis’s allegations, Dr. Kaspar cooperated with the company investigation. Then and now, Dr. Kaspar has appropriately and categorically denied all wrongdoing.”
The scandal became a headache for Novartis, which saw its share price fall as much as 4 percent following the revelation and its executives, including CEO Vas Narasimhan, face tough questioning from investment bank analysts. The company sought to explain the three-month delay in informing the FDA about the issue by stating that it wanted to complete an internal probe first. Meanwhile, it was reported last month that the FDA was looking into a two-month gap between the company discovering the problem, in March, and its launch of a formal probe in May.
In the Form 483 response, AveXis said it looked into the matter when an employee reported it on March 14 and on March 28 informed Novartis, which then launched the internal investigation with the help of an outside law firm, on April 3. “Immediately upon learning of what was then an allegation of data manipulation, AveXis launched an internal investigation on March 14, 2019, led by senior officials from AveXis HR, Quality, and Legal, to fully understand the allegations and determine their merit, and to assess the veracity of the claims and potential scope of the data manipulation,” the document read.
Photo: Novartis
