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
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