Sarepta Recovers: Gene Therapy Battles Setbacks

Sarepta Therapeutics is working to rebound after a challenging period that saw its gene therapy Elevidys, used for Duchenne muscular dystrophy, briefly withdrawn from the market. The company cited a severe flu season at the end of 2025, which necessitated rescheduling infusions for six patients and potentially impacted its current year's financial guidance, suggesting possible uncertainty in drug uptake. This follows a tumultuous year where two teenagers died, leading to the temporary market pull.

Further complicating matters, a recent trial failed to confirm the benefits of two of Sarepta's established drugs, Amondys 45 and Vyondys 53. This development has raised the possibility of their removal from the market by the US Food and Drug Administration. Sarepta has announced plans to meet with US regulators by the end of the current quarter to discuss the future of these exon-skipping treatments.

Despite these headwinds, Sarepta has improved its financial standing by restructuring debt. Elevidys is available for eligible children, with Sarepta exploring a potentially safer administration method for older boys in wheelchairs that could significantly expand market reach and revenue. However, the company faces increasing competition from next-generation treatments being developed by Solid Biosciences and RegenxBio.