The “voluntary and proactive” pledge was provided to the FDA in response to the Form 483 issued to Novartis following the Zolgensma data falsification scandal.
Novartis has been struggling with a serious data integrity issue related to its new gene therapy Zolgensma (onasemnogene abeparvovec-xioi). Novartis' AveXis unit notified the U.S. Food and Drug Administration (FDA) in late June about a problem with animal testing data included in its Biological License Application for the drug. This notification came about one month after Zolgensma was approved despite Novartis having determined in early May that the data were falsified, drawing criticism that the company was delaying to ensure a timely approval.
Novartis’ CEO Vas Narasimhan said the company did not purposely delay but was conducting “a full technical quality investigation” to determine whether the regulatory filings needed to be updated. However, this formal internal probe wasn’t initiated until May 15th –– two months after an AveXis scientist had raised the alarm to management.
Novartis says during those two months it was conducting the first phase of the investigation, and did not make it formal until mid-May. And Narasimhan insists the company did everything “fully in line with regulations, [...] past industry precedents and with our own standard operating procedures.” He does agree, however, that the company should have taken into account that Zolgensma was soon to be a new FDA-approved gene therapy when considering timing of the FDA notification of the data integrity investigation that was underway.
Regardless, Novartis and AveXis were issued a Form 483 by the FDA for the data falsification and mishandling of the data integrity issue. The company says it has included a detailed explanation of the dual-phase nature of its investigation in response to the Form 483. It has also “voluntarily and proactively" pledged to the FDA that it will inform the agency within five business days about "any credible allegation of a data integrity issue of any filing in the Novartis Group that reaches our quality organization," according to Narasimhan.
While the FDA has indicated that it believes Zolgensma should stay on the market, the agency is evaluating possible civil and criminal penalties.