Biopharmaceutical company Avidity Biosciences Inc (Nasdaq:RNA) announced on Wednesday its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the United States.
Under an FDA-authorised treatment protocol, Avidity will provide del-zota to eligible boys and men with DMD44 through participating healthcare providers. Enrolment is anticipated to begin by year end, and participants in EXPLORE44-OLE will have the option to transition to the MAP as they complete 2 years of treatment.
Avidity says that it aligned with the FDA on a path forward for a BLA submission for del-zota following an October 2025 pre-BLA meeting, with the submission planned for 2026 for accelerated approval.
Participants will transition to commercial drug supply upon future potential FDA approval and product availability.
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