What's Happening?
Avidity Biosciences has announced positive data from its EXPLORE44 and EXPLORE44-OLE trials, demonstrating the reversal of disease progression in patients with Duchenne muscular dystrophy (DMD44). The trials showed significant improvements in functional measures such as Time to Rise from Floor and 10-Meter Walk/Run Test. Del-zota, the investigational drug, increased dystrophin production and reduced creatine kinase levels, indicating sustained muscle fiber protection. Avidity plans to submit a Biologics License Application to the FDA by the end of 2025 for accelerated approval.
Why It's Important?
DMD is a severe genetic disorder with limited treatment options. The promising results from del-zota trials offer hope for improved quality of life for patients with DMD44. By enabling the production of functional dystrophin, del-zota addresses the underlying cause of muscle degeneration, potentially transforming the treatment landscape for DMD.
What's Next?
Avidity is preparing for a confirmatory study to support full global approval of del-zota. The company is also planning additional BLA submissions for other rare muscle diseases. The ongoing trials and regulatory submissions will determine the drug's availability and impact on DMD treatment.
