ZolgensmaTM / AVXS-101

Pages in this website section last updated 14th July 2020

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  • What is it?
  • How it works
  • Clinical Trials
  • Where is it licensed for use?
  • Global Access Programme
  • Progress towards licensing and access in the USA, Europe and the UK
  • Access to this Intravenous Treatment in the UK
  • Our Work to Progress Access in the UK

What Is It?

Pharmaceutical Company AveXis has been leading the development of the gene therapy treatment onasemnogene abeparvovec, trademarked as ZolgensmaTM (previously known as AVXS-101).

How it Works

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.

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Clinical Trials

ZolgensmaTM has been, and is still being, tested in several different clinical trials (e.g. START, STR1VE EU, STR1VE, STRONG and SPR1NT), which have enrolled a variety of patients with SMA Types (1-2). The gene therapy has been consistently shown to be safe and well tolerated by those treated with the viruses.

AveXis was also conducting clinical trials to see if an intrathecal administration route (directly into the fluid bathing the spinal cord and brain) would address the needs of those who are older than 2 years. These clinical trials are currently on hold.

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Where is it licensed for use?

  • On 24th May 2019, IV-delivered 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 (based on their genetics they are very likely to develop SMA).

  • On 19th May 2020The European Commission approved IV-delivered Zolgensma® for the treatment of babies and young children who have 5q SMA with a bi-allelic mutation in the SMN1 gene and either a clinical diagnosis of SMA Type 1 or up to three copies of the SMN2 gene.

    According to the approved dosing guidance this covers babies and young children who have SMA up to 21 kg.

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To facilitate the clinical introduction and use of this gene therapy, a group of prominent European neuromuscular experts have provided eleven consensus statements covering many aspects of treatment with onasemnogene abeparvovec. 


Access in the UK

In January 2020, AveXis started to roll-out a Global Managed Access Programme (GMAP). This was not available in the UK for regulatory reasons - read more. Following the European Commission (EC) marketing authorisation, enrolment in all member states closed for new patients on 19th May 2020 - read more

The UK Early Access to Medicines Scheme (EAMS) is only available prior to marketing authorisation so is not an option for UK patient access.

ZolgensmaTM will be assessed in a Health Technology Appraisal by both NICE (National Institute for Health and Care Excellence) and the SMC (Scottish Medicines Consortium) which will make recommendations to the NHS for the most appropriate use of the treatment. The NICE committee will meet on 8th October 2020 and is scheduled to report on 25th March 2021. 

Wales and Northern Ireland generally follow the advice of NICE.

For further information about the steps so far towards UK access, please click here.

Read more about the NICE process and what happens in the rest of the UK, here.

Our Work to Progress Access in the UK

We made a joint submission to NICE on 7th August 2019 after asking people in the SMA Community to complete a survey to let us know their views. Our two Patient Experts were nominated jointly with MDUK and will join the patient expert nominated by TreatSMA and clinical experts to give evidence when the NICE committee meets to appraise the treatment on 8th October 2020.

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Read more about access routes in the UK

Find out what stage other drugs have reached in our drug pipeline