Summary written by DDC Research Volunteer Omar Sheikh
On October 13, Avidity Biosciences shared news on the del-zota treatment for Duchenne. Recently, Avidity met with the U.S. FDA for guidance on their Biologics License Application (BLA), the next stage in approval.
Due to the meeting, Avidity is delaying BLA submission to strengthen their case. The company will now submit the BLA in the first quarter of 2026, not 2025. They aim to include more complete data on the treatment’s consistency of quality and safety.
As a reminder, this treatment attaches a synthetic molecule called ‘oligonucleotide’ to an antibody, a molecule that follows signals from the muscle and heart. When given to a patient with Duchenne, this combination skips exon 44 of the dystrophin gene. This produces shorter yet potentially active dystrophin protein.
Del-zota has received a host of special statuses from the FDA, which aids faster approval. Avidity continues to prepare an international trial on del-zota, possibly with Canadian sites. Defeat Duchenne Canada is working with Avidity Bioscience to learn more about impacts for Canadian families.
Read the full press release from Avidity Biosciences below.
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