Roche has announced the launch of a new global Phase III trial for Elevidys (delandistrogene moxeparvovec), as it seeks to secure regulatory approval in Europe and broaden patient access to the Duchenne muscular dystrophy (DMD) gene therapy.
The study follows a previous setback from the European Medicines Agency (EMA), which issued a negative opinion on Elevidys after earlier clinical data failed to demonstrate a statistically significant improvement in motor function.
Roche says that it has since worked with the regulator and the Duchenne community to design a new trial capable of addressing these concerns.
Elevidys, originally developed by Sarepta Therapeutics, is the first approved gene therapy targeting the underlying genetic cause of DMD, a rare and fatal muscle-wasting disease that primarily affects boys.
The therapy is currently approved for ambulatory patients in nine countries, but access remains limited, especially in Europe.
The newly announced trial will enrol around 100 boys with DMD and compare Elevidys with a placebo during a 72-week period.
The primary endpoint will assess changes in "time to rise" (TTR) from the floor, which is a clinically relevant measure of disease progression and functional decline.
Roche said in a statement that the study is intended to generate the additional placebo-controlled data required for a regulatory resubmission to the EMA, with the broader goal of enabling reimbursement and access in new markets.
Patients initially assigned to the placebo arm will be eligible to receive the gene therapy after the primary study period, a design intended to support recruitment and address ethical considerations.
"The initiation of this new study reflects Roche's commitment to the Duchenne community and our resolve to make this disease-modifying therapy available to ambulatory boys in Europe and around the world," said Dr Levi Garraway, Chief Medical Officer and Head of Global Product Development.
Our confidence is rooted in robust long-term data showing the durable efficacy and safety of Elevidys, alongside the experience of treating more than a thousand ambulatory boys worldwide.
Roche's decision comes amid ongoing debate around the drug's clinical profile.
While long-term data on Elevidys suggest sustained benefits, earlier Phase III results, including those from the EMBARK study, did not meet their primary endpoint.
Safety concerns have also emerged, including reports of serious adverse events in non-ambulatory patients, prompting regulatory restrictions in some regions.
Despite these challenges, Roche maintains confidence in the therapy's potential. According to the company, more than 1000 patients have been treated globally, with data indicating durable efficacy and a manageable safety profile.
With limited treatment options available for DMD and significant unmet need among ambulatory patients, the success of this new Phase III trial could play a key role in determining Elevidys' future in Europe and beyond.