ZolgensmaTM / AVXS-101

Last updated 6th January 2020

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. A screening blood test establishes if any potential patient has natural immunity to the virus which would mean they would not benefit from the treatment. Read more about it works, here.

  • 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 licensing in Europe.
  • Clinicians and SMA patient groups in SMA Europe have welcomed the news that AveXis will be launching a global compassionate use programme in January 2020 for eligible children under 2 years of age, but have concerns about how it will operate. The statement issued by SMA Europe (of which SMA UK are members) outlines these. AveXis have agreed to further discussions in the new year but are committed to the launch.

    Currently it is not known if UK regulations will allow UK patients to be part of this programme. The UK medical community should receive further information of the ongoing discussion between AveXis and the UK regulatory authorities early in the new year. Eligible children in this age group are able to access nusinersen treatment. Click here for more about the programme as announced so far.

  • 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, scheduled inititally for November 2019 and then March 2020, has been further delayed following requests from AveXis. This is due to changes in AveXis' regulatory timings.
  • 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

Read more about access routes in the UK