Novartis Gene Therapies
| File:AveXis.svg | |
| Industry | Biotechnology, Pharmaceutical |
|---|---|
| Headquarters | , United States |
| Parent | Novartis |
| Website | www |
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.[1]
AveXis was acquired by Novartis in 2018 for USD 8.7 billion.[2][3][4][5][6][7][8]
In 2019, Avexis's first gene therapy drug onasemnogene abeparvovec (Zolgensma®) received regulatory approval in the United States[9][10] and has a list price of USD 2.125 million per injection, becoming the most expensive drug in the world.[11] In May 2020, Zolgensma® is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1; or SMA patients with up to three copies of the SMN2 gene.[12]
Shortly after the approval, the US Food and Drug Administration accused the company of data manipulation in their regulatory submission.[13] As a result, Brian Kaspar lost his position within the company.[14]
References
- ^ "Zolgensma's Journey from Lab Idea to Gene Therapy for SMA". SMA News Today. 2019-05-27. Retrieved 2019-08-14.
- ^ Herper, Mathew (19 December 2018). "A Year In, Novartis' Boss Faces The World". Forbes. Retrieved 10 January 2018.
- ^ Delclaux, Alberto (9 April 2018). "Novartis Bets $8.7 Billion on Gene-Therapy Company". The Wall Street Journal. Retrieved 10 January 2018.
- ^ Rose, Marla (9 April 2018). "Gene-therapy company with research ties to Columbus sold to Novartis for $8.7 billion". The Columbus Dispatch. Retrieved 10 January 2018.
- ^ Miller, John (9 April 2018). "Novartis bets big on gene therapy with $8.7 billion AveXis deal". Reuters. Retrieved 10 January 2018.
- ^ Kresge, Naomi (9 April 2018). "Novartis Wager on AveXis Shows Rare Diseases Command Mega Prices". Bloomberg News. Retrieved 10 January 2018.
- ^ Kresge, Naomi (9 April 2018). "Novartis CEO Spurs Rare-Disease Shift With $8.7 Billion Deal". Bloomberg News. Retrieved 10 January 2018.
- ^ Woodyard, Chris (9 April 2018). "Novartis buying AveXis for $8.7B in big gene therapy bet". USA Today. Retrieved 10 January 2018.
- ^ Kaiser, Kaiser (1 November 2017). "Gene therapy's new hope: A neuron-targeting virus is saving infant lives". Science. Retrieved 10 January 2018.
- ^ "$2.1m Novartis gene therapy to become world's most expensive drug". The Guardian. 24 May 2019. Retrieved 3 July 2019.
- ^ Reuters (2019-05-25). "$2.1m Novartis gene therapy to become world's most expensive drug". The Guardian. ISSN 0261-3077. Retrieved 2019-08-14.
{{cite news}}:|last=has generic name (help) - ^ AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA), PM Novartis May 19, 2020; retrieved May 20, 2020
This article incorporates text from this source, which is in the public domain.
- ^ Commissioner, Office of the (2019-08-06). "Statement on data accuracy issues with recently approved gene therapy". FDA. Retrieved 2019-08-14.
- ^ "Novartis replaces top scientists at Avexis after drug data manipulated". Reuters. 2019-08-14. Retrieved 2019-08-14.
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