Roche is initiating a new global phase 3 clinical trial of its Duchenne muscular dystrophy gene therapy, Elevidys, following a previous rejection from European regulators, as the company seeks to strengthen evidence for approval in ambulatory patients.
The Swiss pharmaceutical group is aiming to address concerns raised by the European Medicines Agency, which last year declined to support approval of Elevidys for patients who are still able to walk. Regulators cited insufficient statistical significance in earlier trial results, particularly in measures of motor function improvement.
In response, Roche plans to generate additional placebo-controlled data to support a fresh submission. The upcoming study will enrol around 100 ambulatory boys and will compare Elevidys against placebo over a 72-week period. The primary endpoint will focus on changes in time taken to rise from the floor, a key indicator of disease progression in Duchenne muscular dystrophy.
Elevidys, originally developed by Sarepta Therapeutics, is already approved in the United States for ambulatory patients. However, its approval was based on a phase 3 trial that failed to meet its primary endpoint, raising questions among regulators despite supportive long-term data. Roche maintains that extended follow-up studies demonstrate sustained efficacy and safety benefits.
The therapy’s regulatory journey has also been complicated by safety concerns. Reports of severe liver injury, including two deaths in non-ambulatory patients, led to restrictions on its use. In the United States, regulators ultimately limited treatment to ambulatory patients, while Roche halted or adjusted distribution in some international markets.
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The new study design includes a provision allowing patients initially assigned to placebo to receive Elevidys after the main trial period, which Roche believes will support recruitment and ensure broader access.
While Roche continues to focus on ambulatory patients, further data from ongoing studies may shape future strategies, particularly regarding non-ambulatory populations where the therapy’s benefit-risk profile remains uncertain.
This news item has been summarised using AI and checked by humans before publication.
Sources
fiercepharma.com
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