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DNA (synthetic adeno-associated virus 9 vector scAAV9.CB.hSMN human survivor motor neuron protein-specifying)
FDA 2019/5/24 APPROVED
Spinal muscular atrophy treatment
Treatment of Spinal Muscular Atrophy (SMA) Type 1
Gene therapy product
SMA is a neuromuscular disorder caused by a mutation in the SMN1 gene, which in turn reduces the amount of SMN 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 intravenous formulation was approved in May 2019 in the United States for use in children under 2 years.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.
FDA approves a gene therapy that is the most expensive drug in the world
FDA on Friday approved onasemnogene abeparvovec-xioi (Zolgensma—AveXis), a one-time gene therapy for the treatment of spinal muscular atrophy (SMA).
FDA on Friday approved onasemnogene abeparvovec-xioi (Zolgensma—AveXis), a one-time gene therapy for the treatment of spinal muscular atrophy (SMA). The ultrarare disease affects infants. In announcing the approval, Novartis—which acquired AveXis last year—also disclosed the price of the drug, $2.1 million. The company noted that it would provide rebates to insurance companies if the drug is not successful, though it did not offer details about what would be considered failure. Novartis also said it will set up 5-year payment plans for states, small insurance firms, and self-insured employers. Another drug, nusinersen (Spinraza—Biogen) is already available for the treatment of SMA; however, that drug must continue to be injected into patients’ spines throughout their lives, at a cost of $750,000 in the first year and $375,000 a year after that. “Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research.
- “Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1 | Novartis”. Novartis. Retrieved 2018-12-04.
- “Novartis successfully completes acquisition of AveXis, Inc. | Novartis”. 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.
- “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.
- Reuters (2019-05-25). “$2.1m Novartis gene therapy to become world’s most expensive drug”. The Guardian. ISSN 0261-3077. Retrieved 2019-05-25.
- “Onasemnogene abeparvovec – AveXis – AdisInsight”. adisinsight.springer.com. Retrieved 2018-10-06.
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/////////Onasemnogene abeparvovec, Zolgensma, FDA 2019, オナセムノジーンアベパルボベック ,Spinal muscular atrophy, Gene therapy product, AVXS-101