Avidity submits Biological License Application (BLA) to the FDA, seeking approval for exon 44-skipping “del zota”

Exon 44 Skipping

CureDuchenne was an early funder of Avidity Biosciences, which is now a Novartis company.  We are pleased to share that they have submitted a BLA to the FDA for delpacibart zotadirsen (“del-zota”) as a treatment option for individuals with Duchenne amenable to skipping exon 44.  This application was submitted through the FDA’s Accelerated Approval pathway, and will include data from the Phase 1/2 EXPLORE44 and EXPLORE 44-OLE clinical trials. 

In addition, a global Phase 3 trial (SAFARI44) has also been initiated as a confirmatory trial.  Avidity is also aiming to start a trial for individuals amenable to skipping exon 45 in the first half of 2027. 

Community letter attached.