Delpacibart Zotadirsen (del-zota), a therapy developed by Avidity Biosciences to treat DMD for a specific group of patients by skipping exon 44 of the dystrophin transcript, is showing clinical promise without many reactions requiring hospitalization. In this press release, Avidity announces that del-zota has just been given the Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA), allowing the treatment to be reviewed through a special pathway that is faster while still rigorous. Over the past two years, del-zota has been granted other similar FDA designations including Orphan Drug and Rare Pediatric Disease.
To create this treatment, a synthetic molecule called an oligonucleotide is attached to an antibody, a biological molecule that can recognize specific signals. This combination molecule, once administered, migrates to skeletal and heart muscle tissue, skips exon 44 of the dystrophin transcript, and restores dystrophin protein.
In support of del-zota’s new status, the ongoing EXPLORE44 Open-Label Extension (OLE) trial [NW3] suggests that del-zota produces targeted delivery to skeletal muscle, increases in exon 44 skipping, and increases in dystrophin protein. Simultaneously, del-zota appears to be safe with the most common complaints after treatment being procedural pain and headaches. Avidity also emphasizes their plans to submit the treatment’s Biologics License Application to the FDA — granting them permission to transport the therapy across U.S. state lines — end of 2025. In the fourth quarter, Avidity also plans to present their initial findings from the EXPLORE44-OLE trial on how well treated patients perform functional tasks like walking and holding objects.
At this time there are no active trials open in Canada for Avidity’s Delpacibart Zotadirsen (del-zota). Defeat Duchenne Canada remains committed to providing our community with the most up to date information on drug trials and new drug developments.
Read the full press release from Avidity Biosciences below.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: