ZolgensmaTM / AVXS-101

Pages in this website section last updated 12th April 2022

Recent Headlines

18th January 2022
Study Results Published: Zolgensma Can Benefit SMA Children Previously Treated With Nusinersen

19th November 2021
Update from the National Multidisciplinary Team (NMDT) and NHS England on the progress of the Zolgensma Treatment Programme in England

15th November 2021
Webinar: Zolgensma - 6 Months On

Scroll down further for:

  • What is it, how does it work, how is it given, safety considerations FAQs
  • ‚ÄčThe assessment, treatment process and aftercare FAQs
  • UK access now
  • The European Clinical Consensus Statement
  • Clinical trials
  • Our work and steps to access in the UK

What is it, How does it work, How is it given, Safety considerations - FAQs

Onasemnogene abeparvovec (Zolgensma™) 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.

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‚ÄčThe Assessment, Treatment Process and Aftercare FAQs

With the help of clinical experts, we take you through these questions.

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UK Access Now

England

Infants who have SMA Type 1 and meet eligibility criteria may be assessed for NHS funded treatment with Zolgensma.

Read more about access in England

Scotland

Infants who have SMA Type 1 and meet eligibility criteria may be assessed for NHS funded treatment with Zolgensma.

READ MORE ABOUT ACCESS IN SCOTLAND

Wales and Northern Ireland

Infants who have SMA Type 1 and meet eligibility criteria may be assessed for NHS funded treatment with Zolgensma.

Read more about access in Wales and Northern Ireland

The European Clinical Consensus Statement

In July 2020, a group of prominent European neuromuscular experts provided eleven consensus statements covering many aspects of treatment with onasemnogene abeparvovec. 

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Clinical Trials

Intravenous Zolgensma has been tested in human clinical trials, with children up to the age of 2 years, since 2014. It has been consistently shown to have a well-understood safety profile and to be well-tolerated in these trials.

In many children who have SMA, when compared to no treatment, zolgensma has been shown to increase the availability of SMN protein, leading to clinically meaningful improvements in muscle function. Several trials of IV – delivered Zolgensma are currently ongoing with a variety of children, including those who are older and heavier than in the initial trials

trial investigating intrathecal administration of the treatment with older heavier children resumed in August 2021.

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Our Work and Steps to Access in the UK

We have worked hard with the SMA Community, MDUK, TreatSMA and clinicians, to advocate for access. There have been many steps on the way.

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