AveXis Logotype Preview
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AveXis is a biotechnology company that develops treatments for rare neurological genetic disorders. It was founded in 2015 by Brian Kaspar around a discovery of a novel method of treating spinal muscular atrophy using gene therapy. AveXis was acquired by Novartis in 2018 for USD 8.7 billion. In 2019, Avexis’s first gene therapy drug onasemnogene abeparvovec received regulatory approval in the United States and has a list price of USD 2.125 million per injection, becoming the most expensive drug in the world. In May 2020, Zolgensma® is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy and a clinical diagnosis of SMA Type 1; or SMA patients with up to three copies of the SMN2 gene. Shortly after the approval, the US Food and Drug Administration accused the company of data manipulation in their regulatory submission. As a result, Brian Kaspar lost his position within the company.