ENVISION study news

The pharmaceutical company Roche has shared an update about the development programme for gene therapy delandistrogene moxeparvovec (also called Elevidys),including changes to the Phase 3 ENVISION study for older individuals with Duchenne muscular dystrophy, who are still able to walk (ambulatory patients)and those unable to walk (non-ambulatory patients).

Roche and its partner Sarepta Therapeutics have decided to close recruitment to the Phase 3 ENVISION study, which enrolled 90 older ambulant and non-ambulant people with Duchenne across 14 countries, including the UK.

The companies will now proceed straight to analysing the data from part one of the study.

In the letter, Roche have also confirmed that plans for a new Phase 3 study in younger children, who are still able to walk (ambulatory), are progressing, with the study set up continuing.

ENVISION study update

The decision to close recruitment for the ENVISION study follows ongoing evaluation after a global clinical hold was introduced in 2025, which paused the clinical trial in all countries, including in the UK.

This pause followed the tragic, deaths of two non-ambulatory participants, who suffered acute liver failure after being treated with delandistrogene moxeparvovec in other countries.

Since 2025, the two companies had been exploring ways to continuethe study safely, including potential preventative measures which could be introduced, such as introducing a preventative immunosuppression regimen, as is being used in another study.

However, introducing new measures would mean different participants would have received different treatment. This would create a mixed population and materially alter the study design, which would have practical implications for the study. The decision has therefore now been taken to close recruitment.

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What does this mean for existing ENVISION participants?

Those who have already received the gene therapy through the study will be invited to transition into the EXPEDITION long-term follow-up study for ongoing monitoring.

The letter also states that “it is still being evaluated whether participants who received a placebo will have the opportunity to enter an open-label crossover”, which could potentially give participants the opportunity to still receive delandistrogene moxeparvovec.

This is not currently an option, as the letter notes that this requires further assessment and evaluation of the risk benefit profile and would also require regulators to lift the global clinical hold.

No time frame for a decision on this or further information is set out, and regulators have not indicated plans to lift the hold at present.

Parents of children and individuals participating in the ENVISION study should speak to their clinical teams about what this update means for them.

New global Phase 3 Study for younger ambulatory boys

Meanwhile, Roche has confirmed that preparations are continuing for a new global Phase 3 study in approximately 100 young ambulatory boys with Duchenne. Further details, including eligibility criteria and participating sites, will be announced when recruitment is due to open.

We will share any further updates about the Elevidys research programme as soon as they are published. You can read Roche's full letter to the community here.