Novartis is buying US gene therapy group AveXis in a deal valued at $8.7 billion. The Swiss drug giant is expecting the move to expand its position in the gene therapy space, supporting both its ambition to be a leader in neurodegenerative diseases.

AveXis’ lead product candidate, AVXS-101, has the potential to become the first-ever one-time gene replacement therapy for spinal muscular atrophy (SMA), and the underlying gene therapy platform also has potential beyond the condition. US regulators have already designated AVXS-101 an orphan drug for the treatment of SMA as well as a breakthrough therapy for SMA Type 1, the number one genetic cause of death in infants.

AveXis is expecting to file a Biologics Licence Application with the FDA for AVXS-101 in the second half of 2018, and approval and launch in the US is expected in 2019. PRIME and Sakigake designations have also already been secured in Europe and Japan, respectively.

“We believe AVXS-101 could create a lifetime of possibilities for the children and families impacted by this devastating condition,” said Novartis’ new chief executive Vas Narasimhan. “The acquisition would also accelerate our strategy to pursue high-efficacy, first-in-class therapies and broaden our leadership in neuroscience. We would gain with the team at AveXis another gene therapy platform, in addition to our CAR-T platform for cancer, to advance a growing pipeline of gene therapies across therapeutic areas.”