Avidity Biosciences announced that the Company completed a positive pre-BLA meeting with the U.S. Food and Drug Administration (FDA) regarding its upcoming BLA submission of delpacibart zotadirsen (del-zota). Del-zota, which received Breakthrough Therapy designation, is an investigational drug being evaluated as a potential treatment for people living with Duchenne muscular dystrophy who have gene mutations amenable to exon 44 skipping (DMD44).
Learn More: Mutations and Deletions Amenable to Exon 44 Skipping Therapies
The timing for the BLA submission has been updated to Q1 2026 from previous guidance of year end 2025 to ensure the FDA receives additional data to support the chemistry, manufacturing, and controls (CMC) package at time of submission.
“Our recent meeting with the FDA was highly collaborative and provided a clear path forward for our BLA submission,” said Sarah Boyce, President and CEO, Avidity Biosciences. “For DMD, the accelerated approval pathway in the US is the best and fastest way to bring del-zota to people who need it. We are grateful the agency gave us clear guidance on the CMC data it needs at submission, and we believe this alignment can facilitate a successful BLA process. We greatly appreciate the dedicated, diligent reviewers from multiple functions within FDA who are enabling us to move this program forward with the urgency it deserves. We know that every minute matters for boys and young men living with DMD.”
Avidity remains highly confident in the potential of del-zota and looks forward to filing a BLA for del-zota in Q1 2026, which will be the Company’s first of three planned BLA submissions over a 12-month period. Additionally, Avidity continues to prepare a confirmatory study to support full global approval of del-zota.
Read More: Clinical Trials for Duchenne (List of All Researches)
