Edgewise Therapeutics Inc. (NASDAQ:EWTX) saw its shares slide by 7.2% after the U.S. Food and Drug Administration (FDA) concluded that current data from the company’s CANYON trial does not meet the threshold for accelerated approval of sevasemten, its experimental treatment for Becker muscular dystrophy.
Despite encouraging signs from the MESA open-label extension trial, which showed long-term disease stabilization among patients, the FDA advised that a full traditional approval process would be required. While the agency acknowledged the positive findings, it recommended that Edgewise continue submitting data from the MESA study and its natural history models as supporting evidence while it awaits more definitive results from its ongoing GRAND CANYON trial.
The GRAND CANYON study, a placebo-controlled Phase 3 trial, has the potential to serve as the key registration-enabling study. It remains on track to deliver topline results by the fourth quarter of 2026. The FDA expressed support for this study’s design and emphasized its importance in the drug’s regulatory pathway.
In parallel, Edgewise also reported promising results from its Phase 2 LYNX and FOX trials in Duchenne muscular dystrophy, identifying 10 mg of sevasemten as the optimal dose for future studies. The company intends to meet with the FDA in Q4 2025 to finalize the design of its Phase 3 program, with plans to begin the pivotal trial in 2026.
Chief Medical Officer Dr. Joanne Donovan struck an optimistic tone despite the regulatory setback. “We’ve been overwhelmed by the enthusiasm from clinicians and families about sevasemten’s potential, and we remain focused on completing the necessary steps to bring this therapy to patients,” she said.
Edgewise remains committed to advancing sevasemten as a potential first-in-class treatment for Becker muscular dystrophy, contingent on the successful outcome of its pivotal trial program.