Avidity Touts Functional Improvements for DMD Therapy, Clearing Way to FDA
Avidity Biosciences reported recent positive one-year data showing that its investigational therapy del-zota reversed disease progression and improved functional outcomes in patients with Duchenne muscular dystrophy (DMD) during mid-stage trials25.
Del-zota is an exon-44 skipping therapy using antibody oligonucleotide conjugates (AOCs) designed to enable production of functional dystrophin in DMD patients with mutations amenable to exon 44 skipping235.
The therapy has shown robust improvements in biomarkers (including increased dystrophin production and reduced creatine kinase levels) as well as functional measures in treated participants345.
Del-zota has received Breakthrough Therapy designation from the FDA, emphasizing the urgent need and significant promise for this new treatment34.
Avidity is preparing to submit a Biologics License Application (BLA) to the FDA by the end of 2025, aiming for accelerated approval, with a confirmatory trial planned to support global approvals2456.
Analysts expect the lack of approved exon-44 skipping therapies for DMD to facilitate a smooth approval process, potentially enabling Avidity to become a commercial-stage company as early as 20265.
Sources:
2. https://www.drugdiscoverynews.com/avidity-biosciences-reports-positive-one-year-data-for-del-zota-in-dmd44-trials-16625
3. https://www.prnewswire.com/news-releases/avidity-biosciences-receives-fda-breakthrough-therapy-designation-for-delpacibart-zotadirsen-del-zota-for-the-treatment-of-dmd-in-people-with-mutations-amenable-to-exon-44-skipping-302511334.html
4. https://www.parentprojectmd.org/avidity-biosciences-announces-positive-new-data-from-explore44-and-explore44-ole-phase-1-2-studies/
5. https://www.biospace.com/drug-development/avidity-touts-functional-improvements-for-dmd-therapy-clearing-way-to-fda
6. https://www.ainvest.com/news/avidity-biosciences-duchenne-muscular-dystrophy-treatment-shows-promise-phase-1-2-trial-2509/