Avidity Biosciences today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota) for the treatment of Duchenne muscular dystrophy (DMD) in people living with mutations amenable to exon 44 skipping (DMD44).
Del-zota is currently being assessed in the Phase 2 EXPLORE44 Open-Label Extension (EXPLORE44-OLE™) trial for people living with DMD44 and is the first of multiple AOCs the company is developing for DMD.
DMD is a rare genetic condition that is characterized by progressive muscle damage and weakness due to the loss of dystrophin protein that typically starts at a very young age. Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene and enable production of near-full length dystrophin. – Read More: Avidity Biosciences Announces Positive Topline Del-zota Data Showing Consistent, Statistically Significant Improvements in Dystrophin –
“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. “With the remarkable, consistent improvements we’ve seen in multiple biomarkers including dystrophin in the Phase 1/2 EXPLORE44 trial, we are focused on bringing del-zota to people living with DMD44 as quickly as possible and remain on track for our planned BLA submission at year end 2025.”
In the completed Phase 1/2 EXPLORE44® trial for people living with DMD44, del-zota demonstrated statistically significant increases in exon skipping, a substantial increase in dystrophin production, a significant and sustained reduction in creatine kinase levels to near normal and consistent favorable safety and tolerability. Avidity plans to present topline and functional data from the ongoing, fully enrolled Phase 2 EXPLORE44-OLE trial in the fourth quarter of 2025.
Learn More: Mutations and Deletions Amenable to Exon 44 Skipping Therapies for Duchenne
The company remains on track for a planned BLA submission at year end 2025. Avidity’s commercial preparations for a potential U.S. launch of del-zota in DMD44 following FDA approval are underway. Del-zota’s anticipated launch sets the foundation for potential sequential launches of Avidity’s additional neuromuscular programs for del-desiran in myotonic dystrophy type 1 (DM1) and del-brax in facioscapulohumeral muscular dystrophy (FSHD).
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
In addition to receiving Breakthrough Therapy designation, del-zota has previously been granted Orphan designation by the FDA and the European Medicines Agency (EMA) and Rare Pediatric Disease and Fast Track designations by the FDA for the treatment of DMD44.
Learn More: Upcoming Exon 44 Skipping Therapies for the Treatment of Duchenne Muscular Dystrophy
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