Helping you make wiser investment decisions

With new AveXis name, Novartis spotlights marquee role for gene therapy business

September 4, 2020 Beth Snyder


Call it a two for one: By changing the name of its AveXis unit to Novartis Gene Therapies, the Swiss drugmaker highlights the importance of gene therapies to its future hopes while nixing a moniker linked to last year’s Zolgensma data scandal.

Eventually cleared of wrongdoing by the FDA, AveXis’ data missteps drew an unwelcome spotlight to Novartis’ next-gen therapies business and its newly approved Zolgensma, triggered a management shakeup—and put CEO Vas Narasimhan on the spot. But the dust-up didn’t dampen the company’s ambitions in the field, and its renaming move underscores that.

“With the creation of Novartis Gene Therapies, we will continue to advance our gene therapy pipeline for rare genetic diseases, to accelerate the delivery of transformative innovation in areas of high unmet need, and to reimagine medicine for patients all around the world,” Narasimhan said in a Friday news release.

Featured Webinar

Increasing Patient Adherence for Isolated Populations During the Pandemic

Formulation strategies focused on patient centric solutions featuring several panel speakers representing different perspectives including regulatory, excipients quality related to dose forms, and challenges regarding patience adherence and formulation strategies.

Novartis acquired AveXis for $8.7 billion in April 2018, picking up the phase 3 gene therapy for spinal muscular atrophy that would become Zolgensma and establishing the company as a frontrunner in the emerging gene therapy sector.

RELATED: FDA lets Novartis off the hook in Zolgensma data manipulation

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.

RELATED: Novartis’ AveXis opens Zolgensma plant in Colorado with 350 employees

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.



Source link