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 have applied to the European Medicines Agency (EMA), which decides whether a treatment can be prescribed in the EU. Roche are engaging with the Medicines and Healthcare products Regulatory Agency (MHRA) to find the best possible route for Elevidys in the UK.

The EMA approval decision is expected in 2025. There currently isn’t a timeline for the approval processes in the UK.

Once the approval process is resumed 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.

In March 2025, Roche sadly announced that a 16 year old boy with Duchenne muscular dystrophy passed away following treatment with Elevidys in the USA, having suffered acute liver failure. Roche highlighted that acute liver injury is a known side effect of Elevidys and other AAV-mediated gene therapies. Roche have said this is not a new safety signal and the risk-benefit of Elevidys remains unchanged. However, acute liver failure leading to death has not previously been reported for Elevidys. Roche said that testing revealed the young man had a recent cytomegalovirus (CMV) infection which was identified by the treating clinician as a possible contributing factor. CMV can infect and damage the liver, a condition known as CMV hepatitis.

Roche are continuing to investigate this sad case and have reported it to relevant heath authorities. We are in contact with Roche to understand if this will impact the approval processes in the UK.

Last updated: 19/03/2025