Roche Shares Long-Term Efficacy Data for Evrysdi in SMA

At the Muscular Dystrophy Association (MDA) 2025 conference, Roche reported five-year results from the pivotal SUNFISH study of Evrysdi (risdiplam) for spinal muscular atrophy (SMA) types 2 and 3. Patients demonstrated sustained stabilization of motor function—measured by the Motor Function Measure 32 scale—over five years, a stark contrast to expected declines in untreated cohorts. The final readout reinforces Evrysdi’s disease-modifying benefit and durable safety profile for both children and adults with SMA.

Stabilization Attested in Gene Therapy for Duchenne

Roche also presented data from the EMBARK trial of Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD). MRI and clinical outcome measures over two years showed stabilization or slowing of muscle disease progression in treated patients compared with placebo. Notably, Elevidys-treated patients maintained muscle mass and function—outcomes that mark a turning point for managing DMD, where deterioration is historically relentless.

Scientific Community and Patient Reactions

Dr. Levi Garraway, Roche’s Chief Medical Officer, described the results as “encouraging for people living with SMA and Duchenne,” noting the prospect of lasting improvements and independence. Patient advocacy organizations, including Cure SMA and Parent Project Muscular Dystrophy, hailed these advances as critical for changing the disease trajectory and supporting families.

Outlook: Expanding Access and Next-Generation Research

With these positive data, Roche aims to expand regulatory submissions and post-approval studies for both molecules, while advancing next-generation candidates in neuromuscular research. The focus on long-term data, real-world outcomes, and innovative trial designs is expected to shape clinical guidelines and improve access to transformative therapies.