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.
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