Servier, the French drugmaker, is acquiring an experimental oral therapy for muscular dystrophy from Edgewise Therapeutics. The deal includes $1.55 billion upfront, with additional milestone payments that could push the total value to $2.7 billion, according to BioPharma Dive. The drug, sevasemten, is a small molecule designed to slow disease progression in Duchenne muscular dystrophy (DMD), a rare and fatal genetic disorder.
The candidate has completed Phase 2 testing, showing a statistically significant reduction in muscle damage biomarkers compared to placebo over 12 months. Adverse events were generally mild, including headache and nausea, and no treatment-related serious adverse events were reported. Edgewise had enrolled approximately 100 boys with DMD in its late-stage program.
Servier plans to initiate a Phase 3 trial in the first half of 2027. The therapy has not yet received FDA breakthrough therapy designation, but the company may seek accelerated approval if Phase 2 data hold up. No PDUFA date has been set.
Analysts from BioPharma Dive cite potential peak sales exceeding $2 billion, given the high unmet need in DMD. Edgewise shares rose 23% on the news. Servier gains a promising asset in the mid-stage neuromuscular space, competing with Sarepta’s Elevidys gene therapy and Pfizer’s gene therapy candidate.
Patient advocacy groups cautiously welcomed the deal, noting that an oral therapy could be more convenient than existing treatments. However, they stress that long-term efficacy data are still needed. The Phase 3 trial’s primary endpoint remains under discussion with regulators.