Avidity Biosciences, Inc. has announced the completion of a positive pre-Biologics License Application (BLA) meeting with the U.S. Food and Drug Administration (FDA) regarding its upcoming BLA submission of delpacibart zotadirsen (del-zota) for the treatment of Duchenne in those amenable to exon 44 skipping. Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) conjugated with a muscle targeting antibody to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene and enable production of near-full length dystrophin, and has been granted Breakthrough Therapy designation by the FDA.
According to Avidity, 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. The company has indicated that the FDA provided clear guidance on a path forward for the BLA submission and Avidity believes this alignment can facilitate a successful BLA process. Additionally, Avidity continues to prepare a confirmatory study to support full global approval of del-zota.
PPMD is encouraged by this update and is glad to hear that Avidity and the FDA are working closely together to ensure a strong and comprehensive submission for del-zota. We appreciate that both the agency and the company are taking careful, deliberate steps to support the safety, efficacy, and long-term success of this potential therapy for the Duchenne community, and we look forward to future updates from Avidity.
Read Avidity’s press release here.
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