FDA Declines Accelerated Approval for Edgewise's Muscular Dystrophy Drug

The U.S. Food and Drug Administration (FDA) has declined to consider Edgewise Therapeutics' investigational skeletal myosin blocker sevasemten for accelerated approval in the treatment of Becker muscular dystrophy (BMD). The decision, announced on June 27, 2025, has sent ripples through the pharmaceutical industry and raised questions about the future of muscular dystrophy treatments.

FDA's Decision and Rationale

According to Edgewise, the FDA found that data from the single Phase II CANYON study were "insufficient" to justify an accelerated approval review for sevasemten. The regulatory body emphasized that scores on the North Star Ambulatory Assessment (NSAA), a rating scale used to evaluate motor abilities, can be considered a "clinically meaningful endpoint for traditional approval."

Despite this setback, Edgewise maintains that there is a "clear path to registration" for sevasemten. The company has already launched a global pivotal trial, dubbed GRAND CANYON, which it claims is "highly powered" to demonstrate significant improvements in NSAA scores compared to placebo.

Market Response and Analyst Perspectives

The news of the FDA's decision led to a 10% drop in Edgewise's stock price on the day of the announcement. However, analysts from various firms have offered mixed reactions to the development.

Stifel analysts expressed little surprise at the FDA's decision, stating they had been "skeptical on an accelerated approval path" and that the refusal is "not thesis changing" for Edgewise. They noted that while there is "interesting basic science/mechanistic rationale behind sevasemten," and the Phase III study seems well-powered, it remains "hard to build a lot of conviction."

Truist Securities analysts, while acknowledging the setback, maintained a more optimistic outlook. They continue to "see an opportunity in BMD" and predict that sevasemten's approval will "likely" come after the GRAND CANYON trial results are released in the fourth quarter of 2026.

Implications for Duchenne Muscular Dystrophy Program

In addition to the BMD update, Edgewise provided information on its Duchenne muscular dystrophy (DMD) program for sevasemten. The company reported a favorable tolerability profile in the Phase III LYNX and FOX trials, describing the topline data as "encouraging" with promising signals of efficacy in functional outcomes.

However, analysts remain cautious about these results as well. Stifel analysts noted that they "find it hard to get a ton of conviction" regarding the DMD program and expect investors to "remain generally skeptical" given the "considerable clinical risk" ahead of potential Phase III studies.

As the pharmaceutical industry continues to navigate the complex landscape of muscular dystrophy treatments, all eyes will be on Edgewise Therapeutics and the outcomes of its ongoing clinical trials. The results of the GRAND CANYON study, expected in late 2026, may prove pivotal in determining the future of sevasemten and its potential impact on patients with Becker muscular dystrophy.