Avidity Biosciences Announces U.S. Managed Access Program (MAP) for Investigational Therapy del-zota in DMD44

Core Insights - Avidity Biosciences has launched a Managed Access Program (MAP) for its investigational therapy delpacibart zotadirsen (del-zota) aimed at individuals with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the U.S. [1][2] - The MAP is designed to provide del-zota to eligible patients quickly, with enrollment expected to begin by the end of 2025 [2][3] - Avidity plans to submit a Biologics License Application (BLA) for del-zota in 2026 following a pre-BLA meeting with the FDA [3] Company Overview - Avidity Biosciences focuses on developing a new class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs) [6] - The company aims to address previously unreachable targets and diseases using its proprietary AOC platform [6] - Avidity is advancing clinical programs for rare muscle diseases, including Duchenne muscular dystrophy, and has received multiple designations from the FDA for del-zota [5][6] Product Details - Del-zota is designed to skip exon 44 of the dystrophin gene, enabling dystrophin production in patients with DMD44 [5] - The Phase 1/2 EXPLORE44 trial demonstrated significant increases in dystrophin production and improvements in muscle function, with a favorable safety profile [5] - Del-zota has received designations such as Rare Pediatric Disease, Orphan Drug, Fast Track, and Breakthrough Therapy from the FDA [5]