Novartis Gene Therapies
Parent Novartis | | |
Website | www |
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Novartis Gene Therapies, until 2020 known as AveXis, is a
AveXis was acquired by Novartis in 2018 for USD 8.7 billion.[6][7][8][9][10][11][12]
In 2019, AveXis's first gene therapy drug onasemnogene abeparvovec (Zolgensma) received regulatory approval in the United States[13][14] and, with a list price of USD 2.125 million per injection, became the most expensive drug in the world.[14] In May 2020, the drug was conditionally approved in the European Union 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.[15]
Shortly after the approval, the US Food and Drug Administration accused AveXis of data manipulation in their regulatory submission.[16] As a result, Brian Kaspar lost his position within the company.[17]
In September 2020, Novartis changed the company's name to Novartis Gene Therapies.[18][19]
References
- ^ Moore, Charles. "John Carbona Steps Down as AveXis CEO; Dallas Based AveXis Inc. Announces Management Succession Plan - SMA News Today". Retrieved 2022-06-15.
- ^ Speights, Keith (2019-05-30). "Bothered by the Price Tag of a $2.1 Million Drug? Read This Before Complaining Too Much". The Motley Fool. Retrieved 2022-06-15.
- ^ Ghose, Carrie (April 9, 2018). "Nationwide Children's sold stock, won't get $100M windfall in $8.7B acquisition of gene therapy spinoff". Columbus Business First. Retrieved 2022-06-15.
- ^ "AveXis- BioLife Licenses Spinal Muscular Atrophy (SMA) Patent Portfolio from Nationwide Childrens Hospital and The Ohio State University". Nationwide Childrens Hospital. Retrieved 2022-06-15.
- ^ "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.
- ^ a b "$2.1m Novartis gene therapy to become world's most expensive drug". The Guardian. 24 May 2019. Retrieved 3 July 2019.
- ^ 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.
- ^ "With new AveXis name, Novartis spotlights marquee role for gene therapy business". FiercePharma. 4 September 2020. Retrieved 2020-10-16.
- ^ Melamed, David. "AveXis, Developer of Zolgensma, Now Known as Novartis Gene Therapies". Retrieved 2020-10-16.