Sarepta's Duchenne Therapy Maintains Motor Function Over Three Years

• Elevidys helped patients perform physical activities like jumping and standing on one leg better than untreated control patients, with 73% slowing in time to rise from floor
• Two non-ambulatory teenage boys died in 2024 from acute liver failure linked to the treatment, increasing regulatory and safety concerns
• The three-year efficacy data may help restore confidence in the therapy and support broader adoption amid ongoing safety challenges

Summary: Sarepta's Duchenne Therapy Maintains Motor Function Over Three Years

Sarepta Therapeutics released three-year data from a late-stage study showing its gene therapy Elevidys maintains sustained benefits for patients with Duchenne muscular dystrophy. The therapy demonstrated improved motor function in physical activities including jumping, standing on one leg, and hopping, compared to an untreated external control group.

Key Data Points:

• Study participants were aged 4-7 at treatment and ambulatory (able to walk)
• Elevidys showed 73% slowing of disease progression measured by time to rise from the floor versus the control group
• Data spans three years of treatment observation

Market Context:

Sarepta has faced heightened scrutiny over Elevidys' safety following the deaths of two non-ambulatory teenage patients in the previous year due to acute liver failure linked to the treatment. These three-year results demonstrating long-term functional improvement could help restore confidence in the therapy and support broader adoption.

Market Implications:

The positive long-term efficacy data arrives at a critical time for Sarepta as the company works to address safety concerns that have overshadowed Elevidys. Evidence of sustained motor function benefits over three years may reassure physicians, regulators, and investors about the therapy's risk-benefit profile, potentially supporting continued market uptake for this treatment of the progressive muscle-wasting disorder.

The announcement comes as Sarepta seeks to balance demonstrating clinical value against ongoing safety monitoring requirements for this gene therapy in the rare disease space.