Stephan Emmerth

Stephan Emmerth

PhD | Director Therapeutic Innovation and BaseLaunch

Tel. +41 61 295 50 17

report Life Sciences
Copyright: Michael Heinrich

Copyright: Michael Heinrich


Novartis to acquire gene therapy specialist

Novartis announced that it will acquire AveXis for $8.7 billion. The US-based specialist for gene therapies developed a therapy for spinal muscular atrophy, which is expected to be launched on the market in 2019. AveXis also operates a gene therapy platform that is scalable for other diseases.

Listed on the New York Nasdaq and headquartered in Bannockburn, Illinois, AveXis develops gene therapies. Its lead product candidate, AVSX-101, was granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of spinal muscular atrophy, making it the only potential treatment for the disease. Similar designations have been secured in Europe and Japan, namely PRIME and Sakigake respectively. 

AVXS-101 has the potential to be the first gene therapy that addresses the root cause of spinal muscular atrophy, according to a Novartisstatement announcing the acquisition. The therapy is expected to be launched on the market in 2019.

According to Novartis CEO Vas Narasimhan, the acquisition’s relevance extends beyond spinal muscular atrophy.

“The acquisition would also accelerate our strategy to pursue high-efficiency, 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.” 

AveXis shares soared after the announcement, rising from just under $116 on Friday to nearly $210 on Monday, an increase of 80 per cent. Novartis shares on the Swiss stock exchange, in contrast, dropped after a brief gain.


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