The AveXis controversy began shortly after the FDA approved Zolgensma in May 2018. Two months after that green light, Novartis reported that some of the data in its FDA application were inaccurate. But it wasn't the data problem itself that spun the incident into scandal.
Rather, it was Novartis' timing on revealing the problem. An AveXis scientist had told Novartis about the faked data before the drug’s approval, but the company didn't notify the FDA until weeks later. The company acknowledged it had the information earlier, but said it was completing its own internal investigation before reporting to the FDA.
When the news first broke, the FDA quickly concluded Zolgensma was still safe and effective, but launched an investigation into what had happened. The agency ultimately decided not to punish AveXis for the manipulated Zolgensma mice testing data in its drug application package.
In a news release, Novartis said the name change is a “natural evolution” as the company scales up Zolgensma and expands it AAV-based gene therapy pipeline.
The new Novartis Gene Therapies will lead research, development, manufacturing and commercialization of that pipeline as well as manufacture for other Novartis groups. Dave Lennon, who moved from Novartis oncology in June 2018 to become president of AveXis, stays on as the president of Novartis Gene Therapies and continues to report to Narasimhan.
Zolgensma is the world’s most expensive drug priced at more than $2 million. Since its approval, Novartis reports more than 600 patients have been treated by Zolgensma, a number that includes clinical trials, commercial use and managed access programs. Novartis says it is the most widely used gene therapy in the world.