Avidity Biosciences announced the launch of a Managed Access Program (MAP) for its investigational exon‑44‑skipping therapy del‑zota (delpacibart zotadirsen) on November 19 2025. The program will provide eligible patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 44 skipping with del‑zota through participating health‑care providers under an FDA‑authorized treatment protocol, with enrollment slated to begin by the end of 2025.
The MAP is designed to give patients who have completed two years of treatment in the ongoing EXPLORE44‑OLE trial an option to transition to the program. Eligibility requires a confirmed DMD diagnosis, the presence of a pathogenic mutation that allows exon 44 skipping, and a baseline functional status that meets the protocol’s inclusion criteria. Patients who qualify will receive del‑zota under the same dosing schedule used in the clinical trial, and the program will collect safety and efficacy data in a real‑world setting. Upon FDA approval, participants will transition to commercial supply, ensuring continuity of care without interruption.
The launch of the MAP supports Avidity’s 2026 accelerated‑approval BLA submission by expanding the data set beyond the controlled trial environment. The company estimates that roughly 1,000 U.S. patients are eligible for exon 44 skipping, providing a sizable addressable market for del‑zota. By accelerating data collection and demonstrating real‑world safety and efficacy, the program strengthens the regulatory dossier and positions the therapy for a broader commercial launch under the Novartis partnership, which was announced in October 2025.
Del‑zota is an antibody‑oligonucleotide conjugate (AOC) that delivers a splice‑modifying oligonucleotide directly to skeletal muscle via a muscle‑specific antibody. Phase 1/2 data from EXPLORE44 showed a 30‑plus percent increase in dystrophin production, a 20‑percent reduction in creatine kinase, and clinically meaningful improvements in motor function. The therapy has received Rare Pediatric Disease, Orphan Drug, Fast Track, and Breakthrough Therapy designations from the FDA, underscoring its potential to address an unmet need in the DMD44 population.
Avidity’s CEO Sarah Boyce emphasized that the MAP “enables a compliant approach to providing del‑zota to eligible patients as quickly as possible” and that the program will “accelerate data collection and support the 2026 BLA submission.” The MAP launch is a key step in Avidity’s strategy to bring a first‑in‑class exon‑skipping therapy to patients while leveraging the global reach of its Novartis partner to scale production and distribution once regulatory approval is achieved.
Avidity’s move to expand access through the MAP reflects a broader industry trend of early‑access programs that balance patient need with data generation. By aligning the program with FDA protocols and a clear commercial transition plan, the company demonstrates regulatory readiness and a commitment to patient safety, positioning del‑zota for a potentially rapid market entry once the BLA is approved.
Avidity’s strategic partnership with Novartis, announced in October 2025, provides the manufacturing and commercial infrastructure needed to support a global launch. The MAP’s real‑world evidence will feed into the BLA, while the partnership ensures that, upon approval, del‑zota can be delivered at scale to the estimated 1,000 U.S. patients and a larger global cohort.
The program’s launch underscores Avidity’s focus on delivering innovative therapies to underserved patient populations and highlights the company’s ability to navigate complex regulatory pathways while maintaining a patient‑centric approach.
The MAP launch is expected to strengthen investor confidence in Avidity’s pipeline and its ability to meet regulatory milestones, reinforcing the company’s position as a leader in the emerging AOC therapeutic space.
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