AveXis Receives USA Approval for their Gene Therapy, Zolgensma®, for the Treatment of Children with SMA Aged Less than 2 Years
28 May 2019
On 24th May, the Food and Drug Administration (FDA) in the USA has announced their approval of this ground-breaking treatment. This one-time injection into the blood is designed to address the genetic root cause of SMA by replacing the faulty or missing SMN1 gene to limit the progression of the condition. Children who are pre-symptomatic (meaning that, based on their genetics they are very likely to develop SMA) may also be treated.
We will now have to wait to see if it will be approved by the European Medicines Agency (EMA). It will then need to be reviewed by the National Institute of Health and Care Excellence (NICE) for England and the Scottish Medicines Consortium (SMC) for Scotland to see if it will be recommended for provision by the NHS in the UK. It is a suggested Highly Specialised Technology (HST) where it is listed under its full name: onasemnogene abeparvovec.
We are also closely watching the further development by Avexis of the intrathecal administration route, which is directly into the fluid bathing the spinal cord and brain, to address the needs of those who have SMA and are older than 2 years.
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