FDA Grants Priority Review to Potential Duchenne Treatment Del-Zota

As an early funder of Avidity Biosciences, we are pleased to share that Novartis has announced that the FDA has accepted the Biologics License Application (BLA) for delpacibart zotadirsen (del-zota) for Priority Review under the Accelerated Approval pathway. Del-zota, which was developed by Avidity, is being evaluated as a potential treatment for people with Duchenne who have a genetic variant that may be treated through exon 44 skipping.  

The application includes data from the Phase 1/2 EXPLORE44® and EXPLORE44-OLE™ clinical trials, and the global Phase 3 SAFARI44™ confirmatory trial has already been initiated. Read the community letter here: https://cureduchenne.org/wp-content/uploads/2026/09/Avidity-BLA-Acceptance-PAG-Community-Letter_2026.pdf