Novartis has given the FDA a list of actions it intends to take to avoid a repeat of the data manipulation that has overshadowed its Zolgensma gene therapy, but puts the blame squarely on two already-sacked senior employees.
AveXis co-founders Brian and Allan Kaspar either manipulated the data themselves or pressured employees to do so, says Novartis in its submission to the US regulator. It also alleges they held up the internal investigation through a “lack of cooperation and categorical denial of the allegations”. It fired the pair on 13 August.
While pharma companies often allow biotechs they acquire to retain independence in order to preserve the agility and ingenuity that made them attractive targets, Novartis has decided it will shift AveXis’ quality control functions in-house, with a new senior executive appointed to oversee the process and future compliance.
The Swiss pharma’s remediation plan also includes rolling out a Novartis whistleblower scheme across AveXis to encourage reporting of misconduct and fraud, will retrain AveXis’ quality unit employees and has said it will let the FDA know within five business days if it receives credible allegations related to data integrity that could affect any pending drug applications.
Shifting the blame to the Kaspars gives Novartis a means of sidestepping the main criticism levelled by the FDA, namely that it was aware of the manipulation of animal testing data included in its submission before Zolgensma was approved but only told the agency after receiving marketing authorisation in the US.
That element of the scandal has brought Novartis into the sights of US lawmakers who have accused it of stalling the disclosure of the manipulation in order to rush Zolgensma to market.
Legal counsel representing Brian Kaspar told Reuters that the former executive denies any wrongdoing and says he cooperated fully with the investingation, claiming that AveXis “shamelessly attempts to blame others for its own disclosure decisions to the FDA”.
Brian Kaspar was chief scientific officer of AveXis, while his brother Allan was senior vice president of R&D.
Zolgensma was approved as a one-shot treatment for the rare genetic disorder spinal muscular atrophy in May, becoming the world’s most expensive medicine with a price tag of $2.1m.