ZolgensmaTM / AVXS-101
Pages in this website section last updated 30th July 2021
Scroll down further for:
- What is it?
- How it works
- Access in the UK
- The European Clinical Consensus Statement
- When and what marketing authorisation has it had in the USA and Europe?
- Global Access Programme
- Clinical Trials
- Our work that has helped to progress access in the UK
What Is It?
Pharmaceutical Company, Novartis Gene Therapies (previously AveXis until September 2020), 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 Zolgensma 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.
Access in the UK
On 8th March 2021, NHS England announced a landmark agreement giving access to some children who have SMA Type 1. In parallel, the regulatory authority NICE (the National Institute of Health and Care Excellence) completed its appraisal of the treatment and issued its final guidance on 7th July 2021.
There are now four infusion Centres in England providing this treatment. The overall programme is overseen by the National Multidisciplinary Team (NMDT) of expert clinicians. The latest information from the team, including which children are eligible, referral and prioritisation is on this NMDT updates page.
Also on 8th March 2021, the Scottish Medicines Consortium (SMC) concluded the first Health Technology Appraisal of Zolgensma in Europe and announced its landmark decision giving access to some children who have SMA Type 1. There is more information about eligibilty here. Treatment takes place at the Royal Hospital for Children, NHS Greater Glasgow and Clyde. Clinical teams will have all the detail families need.
Wales and Northern Ireland
Please see Q.1 on our England Q&As page for latest information on access in these devolved countries.
Our England Q&A page covers a broader range of Q and As about: access in England, Scotland, Wales and Northern Ireland; how the treatment is delivered, preparation and follow-up; if your child is currently receiving another treatment and other topics.
Though our webinar 'Zolgensma Treatment for SMA Type 1 in the UK' recorded on 21st April 2021 with leading health professionals, focused on access in England, the discussions about the treatment itself and making treatment decisions may still be helpful.
The European Clinical Consensus Statement
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.
When and what marketing authorisation has it had in the USA and Europe?
- On 24th May 2019, IV-delivered Zolgensma 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 2020, The 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
According to the approved dosing guidance this covers babies and young children who have SMA up to 21 kg. Read more about this approval, here.
Global Managed Access Programme
In January 2020, Novartis Gene Therapies rolled out a Global Managed Access Programme (GMAP).
Early access programmes are available up until a treatment receives marketing authorisation from the regulatory authority that has jurisdiction over a country. Access stops at this point even if the country has not made an agreement to provide the treatment for its potentially eligible citizens.
This programme was not available in the UK for regulatory reasons - read more.
Zolgensma has been, and is still being, tested in several different clinical trials (e.g. START, STR1VE EU, STR1VE, STRONG, SPR1NT and SMART - which is a new global clinical trial that will be investigating the safety, tolerability and efficacy of intravenous Zolgensma in children who have SMA and weigh between 8.5 kgs and 21 kgs). The gene therapy has been consistently shown to be safe and well tolerated by those treated with the viruses.
Novartis Gene Therapies (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.
Our Work that has Helped to Progress Access in the UK
After asking people in the SMA Community to complete a survey to let us know their views, we made a joint submission with MDUK to NICE on 7th August 2019 in which we strongly supported access.
You can read our submission and the survey results, here.
We nominated two Patient Experts jointly with MDUK. They were selected along with two others (nominated by Treat SMA and the Ali Cadence Trust) and joined two clinical experts to give evidence when the NICE committee met to appraise the treatment on 8th October 2020. Following this, on 8th March 2021, NICE released its draft recommendations for consultation. You can read our
response to these. The final committee meeting is on 13th May 2021.
We are also working closely with NHS England and Novartis Gene Therapies to support the set up of this new service.
Working from our survey results and other information from the Community, we made we made a joint submission with MDUK to the Scottish Medicines Consortium on 30th November 2020 in which we strongly supported access. Our Patient Representatives attended the Patient and Clinician Engagement (PACE) meeting. The SMC announcement acknowledges their advice takes account of the views expressed at this meeting.
Our involvement and advocacy in the earlier steps of this process started in October 2018. You can find a summary here.