“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,” said  Novartis group CEO Vas Narasimhan.

In addition to further developing and marketing Zolgensma to treat spinal muscular atrophy (SMA), the new genetics unit will also provide manufacturing support for gene therapy work conducted by other Novartis units. The spinal disease is the leading genetic cause of infant death.

Zolgensma is approved in the US, Japan, Europe and Brazil. The new Novartis Gene Therapies is currently pursuing registration for the drug in numerous other countries. Regulatory decisions are expected in Switzerland, Canada, Australia, Argentina and South Korea by late 2020 or early 2021.

As the Swiss group scales up to deliver Zolgensma globally, it is also seeking to expand its reach via a “robust” pipeline of AAV-based gene therapies for rare genetic diseases including investigational treatments for Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia

Novartis acquired AveXis for $8.7 billion in April 2018, and the drug was approved by the US Food and Drug Administration (FDA) a month later. Shortly afterward, the drugmaker acknowledged belatedly it had failed to report that some of the data in the FDA filing was inaccurate as it wanted to investigate internally first. The agency did not levy a penalty.

Author: Dede Williams, Freelance Journalist