Sarepta Therapeutics Inc. and Roche Holding AG are facing setbacks after their gene therapy for Duchenne muscular dystrophy (DMD) failed to meet the primary endpoint in a pivotal trial. The results cast doubt on the treatment's potential for broader use in older children with the disease.
The Phase III trial involved 125 young patients with DMD, a progressive muscle-wasting disorder. The study aimed to assess whether the gene therapy could slow disease progression over a one-year period. However, the treatment did not demonstrate a statistically significant benefit compared to the placebo group.
The failure sent Sarepta's shares plummeting, with the stock experiencing its biggest drop since January 2021. Roche also saw a decline in its stock value following the announcement.
Despite the disappointing trial results, Sarepta plans to seek broader approval for the gene therapy, which carries a price tag of $3.2 million. This decision has sparked debate among investors and the medical community, given the lack of clear efficacy demonstrated in the Phase III trial.
Normally, pharmaceutical companies will go back to the drawing board when a drug fails to show a clear effect in a final-stage trial. Sarepta's decision to move forward with the approval process despite the trial's failure is unusual.
