Avidity Biosciences, Inc. has announced its Managed Access Program (MAP) for delpacibart zotadirsen (del-zota) for eligible individuals living with Duchenne who are amenable to exon 44 skipping in the United States. MAPs allow a limited number of patients to receive an investigational therapy that is still under U.S. Food and Drug Administration (FDA) review outside of a clinical trial, based on specific eligibility and safety criteria.
Avidity indicated that under an FDA-authorized treatment protocol, the company will provide del-zota to eligible individuals through participating healthcare providers. Avidity anticipates MAP enrollment to begin by year end 2025, and reports that participants in EXPLORE44-OLE will have the option to transition to the MAP as they complete two years of treatment.
In October 2025, Avidity shared the completion of a positive pre-Biologics License Application (BLA) meeting with the FDA regarding its upcoming BLA submission of del-zota, and indicated that the submission is planned for 2026 for accelerated approval. According to Avidity, participants will transition to commercial drug supply upon future potential FDA approval and product availability.
PPMD welcomes the news of Avidity’s MAP for del-zota. Expanding access to investigational therapies for individuals for whom there is no comparable alternative treatment can be a lifeline for families, and we appreciate efforts that help bridge the gap while clinical development, learning, and progress continue. We look forward to future updates from Avidity and will share information with the community as it becomes available.
Additional information about the MAP, including eligibility criteria, will be available on www.clinicaltrials.gov. Read Avidity’s press release here.
Upcoming Webinar
Avidity will join PPMD for a community webinar on Wednesday, November 19, 2025 at 1:00 PM ET to discuss topline and functional data from the EXPLORE44-OLE study of del-zota. Please register here and submit questions in advance here.
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