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SMA is a neuromuscular disorder caused by a mutation in the SMN1 gene, which in turn reduces the amount of SMN protein, a protein necessary for survival of motor neurons. Onasemnogene abeparvovec is a biologic drug consisting of AAV9 virus capsids that have been deprived of the original viral DNA and instead contain a SMN1 transgene along with promoters. The drug is administered intravenously or intrathecally. Upon administration, the AAV9 viral vector delivers the SMN1 transgene to cell nuclei where the transgene begins encoding SMN protein, thus addressing the root cause of the disease. Since motor neurons do not divide, it is thought that a single dose of the drug will have a lifelong effect.
The medication was developed by a US biotechnology company AveXis, a subsidiary of Novartis, based on an earlier discovery by French researchers. The purification process, representing up to 90% of the production costs of a drug, was developed by the Slovenian company BIA Separations. The intravenous formulation was approved in May 2019 in the United States for use in children under two years old. It carries a list price of US$2.125 million per dose (one-time treatment), making it the most expensive medication in the world as of 2019.
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- "Novartis successfully completes acquisition of AveXis, Inc". Novartis. Retrieved 2018-10-06.
- "AveXis receives FDA approval for Zolgensma®, the first gene therapy for paediatric patients with SMA". SMA Europe. 2015-05-25. Retrieved 2019-05-25.
- "Slovenian Firm Involved in First Gene Therapy for Spinal Muscular Atrophy". Total Slovenia News. 3 June 2019.
- "FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality". FDA. 2019-05-24. Retrieved 2019-05-24.
- "$2.1m Novartis gene therapy to become world's most expensive drug". The Guardian. London. Reuters. 2019-05-25. ISSN 0261-3077. Retrieved 2019-05-25.
- "Onasemnogene abeparvovec - AveXis - AdisInsight". adisinsight.springer.com. Retrieved 2018-10-06.
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