Shares of Avidity Biosciences, Inc. (NASDAQ:RNA) rose 4% Wednesday following the announcement that its drug candidate, delpacibart zotadirsen (del-zota), has been awarded Breakthrough Therapy status by the U.S. Food and Drug Administration. The designation applies to del-zota for treating Duchenne muscular dystrophy (DMD) in patients with mutations eligible for exon 44 skipping.
This FDA status is intended to accelerate the development and review of treatments for serious diseases that demonstrate early signs of significant improvement compared to existing options. Del-zota is currently being evaluated in the Phase 2 EXPLORE44 Open-Label Extension trial targeting patients with DMD44.
In the completed Phase 1/2 EXPLORE44 trial, del-zota showed statistically significant increases in exon skipping, robust boosts in dystrophin production, and a meaningful drop in creatine kinase levels approaching normal ranges, all while maintaining a favorable safety and tolerability profile.
“Breakthrough Therapy designation further underscores the FDA’s appreciation for the significant potential of del-zota to address the underlying cause of DMD44 and the urgent need to bring innovative treatment options to the DMD community,” said Steve Hughes, M.D., chief medical officer at Avidity.
Avidity expects to share topline and functional results from the ongoing Phase 2 trial in the fourth quarter of 2025 and aims to submit a Biologics License Application by year-end 2025.
Duchenne muscular dystrophy is a rare genetic disorder marked by progressive muscle deterioration and weakness due to the loss of dystrophin protein. Del-zota works by delivering phosphorodiamidate morpholino oligomers to skeletal and cardiac muscle tissues, promoting the production of near-full-length dystrophin.
The drug has also earned Orphan Drug designation from both the FDA and European Medicines Agency, along with Rare Pediatric Disease and Fast Track designations from the FDA.
Avidity Biosciences stock price
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