Avidity Biosciences, Inc. has announced its plans for filing a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for delpacibart zotadirsen (del-zota) for treatment of individuals living with Duchenne muscular dystrophy mutations amenable to exon 44 skipping. Del-zota (AOC 1044) is an exon skipping therapy conjugated to a monoclonal antibody targeting the transferrin receptor to improve delivery of the exon skipping therapy to muscle.

According to Avidity, the company plans to submit a BLA by the end of 2025. Avidity also reports that the FDA has confirmed the accelerated approval path is available for del-zota and that the clinical data package from the EXPLORE44 program could support the filing. 

Additionally, the company indicated that it expects to present topline data from the EXPLORE44 trial during Q1 of 2025. Topline data from the ongoing EXPLORE44 open label extension trial is expected in Q4.

PPMD is pleased to learn this news and we look forward to further updates from Avidity as research continues.

Read Avidity’s press release here.