Elevidys

Pharmaceutical company: Roche

People with Duchenne muscular dystrophy lack the dystrophin protein. Elevidys delivers a gene into the body that leads to the production of micro-dystrophin, a smaller version of the dystrophin protein that works in a similar way.

Elevidys has been designed as a single-dose treatment, meaning that it is only administered once. It comes as a liquid and is administered into the bloodstream (known as intravenous infusion).

Elevidys can only be accessed through a clinical trial until it has gone through the regulatory process. For more information on these clinical trials please use the DMD hub.

Roche were planning on using the decision from the European Medicines Agency (EMA) to support the approval for use in the UK. However, in July the EMA were against approval (known as a negative opinion). This method of getting approval in the UK is now not possible.

Roche are engaging with the Medicines and Healthcare products Regulatory Agency (MHRA) to find the best possible route for Elevidys in the UK.

There currently isn’t a timeline for the approval processes in the UK.

Once the approval process is underway in the UK, we will work in partnership with other Duchenne charities to make sure the experiences and views of the community are heard.

The phase 3 EMBARK clinical trial involved 125 boys with DMD aged 4-7 years, who could walk. They were randomly assigned to receive Elevidys (63 participants) or placebo (dummy drug, 59 participants).

Early analysis (top-line data) after 52 weeks showed the trial didn’t meet its primary aim (also known as endpoint). While treatment with Elevidys did improve the ability to move, measured by the NorthStar Ambulatory Assessment (NSAA) score, this was not statistically significant. This means that the researchers could not confirm that the results weren’t due to chance.

However, statistically significant improvements were seen for other measures of movement, such as time to rise from floor and 10 meter walk test. Elevidys was also found to be safe.

Long term data

Two years after treatment with Elevidys, participants showed improvements in movement measures (NSAA score) compared to what would have been expected if they hadn’t received the treatment. To do this comparison, the researchers used data from people with DMD who never received the treatment but had similar characteristics (such as age, steroid use and NSAA scores) to those in the EMBARK study. This data was collected from other clinical trials and a natural history study (where people with DMD are followed throughout their life).

It is important to note that using data collected outside of the EMBARK trial can come with some limitations. Comparing to a placebo would make the evidence collected more reliable but researchers also consider how ethical it is to use a placebo for a long period of time. Further results from this trial will be announced in the future.

Roche has sadly reported the deaths of two people with DMD after receiving the treatment Elevidys. Both individuals were not able to walk (non-ambulatory), and the cause of death in each case was acute liver failure.

As a result, Roche has advised that Elevidys should no longer be given to people with DMD who are non-ambulatory, no matter their age. Several clinical trials testing Elevidys have also been paused while more information is gathered. However, Roche has said that this does not affect people with DMD who are able to walk (ambulatory), as the benefits-risk balance remains unchanged in this group. This means Roche believes the potential benefits of treatment continue to outweigh the risks.

Elevidys is delivered using a type of virus called an AAV (adeno-associated virus), which helps carry the treatment into the body’s cells. This same type of virus is also being used in other experimental treatments, including one for limb girdle muscular dystrophy. Sadly, someone taking part in an early-stage trial for that treatment has also died.

We are in contact with Roche to understand what this means for the approval of Elevidys in the UK and will share more updates as soon as we know more.

Last updated: 05/08/2025