ZolgensmaTM / AVXS-101

Last updated 8th August 2019

Pharmaceutical Company AveXis has been leading the development of the gene therapy treatment onasemnogene abeparvovec, trademarked as ZolgensmaTM (previously known as AVXS-101). It uses harmless, genetically-engineered viruses to increase SMN protein levels. A single injection of ZolgensmaTM 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.

  • On 24th May 2019, ZolgensmaTM was approved in the USA for the treatment of children with SMA under the age of 2 years and for children who are pre-symptomatic (meaning that, based on their genetics they are very likely to develop SMA).
  • It is currently being considered by the European Medicines Agency (EMA) for licencing in Europe.
  • The National Institute of Health and Care Excellence (NICE) has started to progress its review of whether it will recommend if the treatment should be provided by NHS England for infants with SMA Type 1. We were invited to make a submission to NICE by 7th August, and asked people in the SMA Community to complete a survey to let us know their views. Thank you to those of you who replied, you can see the results here. We passed them on to MDUK and TreatSMA and used them in the submission we made jointly with MDUK - you can read this here. The first committee meeting will be on 28th November 2019.
  • AveXis is also conducting clinical trials to see if an intrathecal administration route, which is directly into the fluid bathing the spinal cord and brain, will address the needs of those who have SMA and are older than 2 years. 

How Does It Work?

What are the Clinical Trial Results for this Intravenous Treatment?

Access to this Intravenous Treatment in the UK

What We Are Doing To Progress Access in the UK