Avidity plans to submit for FDA Accelerated Approval for Duchenne exon 44 skipping at the end of 2025; Open-label extension trial is still recruiting
Amenable to skipping exon 44
Avidity Biosciences, which received early funding from CureDuchenne, plans to submit for US FDA Accelerated Approval at the end of 2025 for Delpacibart zotadirsen (del-zota), an exon-skipping experimental therapeutic for individuals with Duchenne amenable to skipping exon 44. Topline data from their EXPLORE44 trial will be released in Q1 of this year. Topline data from the ongoing EXPLORE44 Open Label Extension, which is still recruiting, is expected in Q4 2025.
Link to clinical trial: https://clinicaltrials.gov/study/NCT06244082
Link to press release: https://aviditybiosciences.investorroom.com/2025-01-08-Avidity-Biosciences-Plans-First-BLA-Submission-and-Accelerates-Commercialization-Preparations-for-Three-Rare-Muscle-Disease-Programs-in-2025