AveXis, Inc. the US-based Nasdaq-listed clinical-stage gene therapy company has been acquired by Novartis for USD 218 per share or a total of USD 8.7 billion in cash.

AveXis is a gene-therapy company conducting several clinical studies for the treatment of spinal muscular atrophy or SMA, an inherited neurodegenerative disease caused by a defect in a single gene, Novartis said. Some form of SMA affects an estimated one out of every 6,000 to 10,000 children born, it said.

This would add another feather to the cap of gene therapy, a treatment type Novartis is already leading in the field of cancer.

“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,” said Dr. Narasimhan, Chief Executive, Novartis.

After it paid US$105 million upfront and up to another US$65 million in milestone payments for rights to Spark’s blindness treatment, Luxturna, outside the United States, this is Novartis’ second notable gene therapy deal this year.
“We believe the medicine would have a multibillion-dollar peak sales potential,” Mr. Narasimhan said on a conference call with reporters, of AVXS-101, which has so-called breakthrough therapy designation in the US and is expected to be submitted to regulators this year.

“It also provides us capabilities in gene therapy,” he added. “We have a robust internal portfolio of gene therapies in ophthalmology and neuroscience in Novartis Institutes for Biomedical Research. And we look forward to using AveXis’ capabilities and technical development capabilities to be able to advance that portfolio.”