Avidity Biosciences Launches Managed Access Program for Groundbreaking DMD Treatment

Avidity Biosciences Launches Managed Access Program for del-zota

Avidity Biosciences, Inc., a prominent player in RNA therapeutics, is making strides in the fight against Duchenne Muscular Dystrophy (DMD) with the introduction of its Managed Access Program (MAP) for delpacibart zotadirsen, more commonly referred to as del-zota. This investigational therapy is aimed specifically at patients with DMD mutations that are suitable for exon 44 skipping (DMD44). The announcement, made on November 19, 2025, is a significant development for the DMD community, as current options for exon skipping therapies are sorely lacking.

Sarah Boyce, the President and CEO of Avidity, expressed the company's commitment to addressing the urgent needs of the DMD44 patient community, stating that the MAP will provide a compliant means to facilitate rapid access to del-zota for eligible patients. Under an approved treatment protocol from the FDA, healthcare providers will administer del-zota to qualifying boys and men with DMD44, with enrollment expected to commence by the end of the year.

Navigating Pathways for Approval

Avidity's engagement with the FDA has been proactive; following an operational meeting in October 2025, the company has laid down a roadmap towards the submission of a Biologics License Application (BLA) for del-zota, projected for 2026 under accelerated approval guidelines. This submission is seen as a critical step forward, especially for those awaiting innovative treatments in the DMD space. Participants in the clinical trial, EXPLORE44-OLE, will have the opportunity to transition into the MAP once they complete two years of treatment, enhancing continuity of care for these patients.

Understanding Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy is a serious genetic disorder characterized by progressive muscle degeneration due to a deficiency in the dystrophin protein, which plays a vital role in maintaining muscle cell integrity. The absence of dystrophin leads to the deterioration of muscle fibers and often results in severe physical disabilities, breathing difficulties, and reduced life expectancy. Despite the approval of several treatments, the availability of effective therapies targeting the underlying genetic causes remains critically low.

DMD affects primarily male individuals, with an alarming prevalence of approximately one in every 3,500 to 5,000 boys born globally. As the search for effective treatments continues, del-zota offers hope for those with mutations conducive to exon 44 skipping, aiming to enable dystrophin production and reduce muscle damage.

Mechanism of Action and Clinical Trial Insights

Del-zota is engineered to transport phosphorodiamidate morpholino oligomers (PMOs) directly to skeletal muscle and heart tissues, specifically bypassing exon 44 of the dystrophin gene, thereby promoting the production of dystrophin. This targeted approach is a pivotal advancement over traditional therapies, which often fail to deliver genetic material effectively.

Preliminary findings from the Phase 1/2 EXPLORE44 trial indicate that del-zota significantly enhances PMO delivery to affected tissues and markedly increases dystrophin production. Moreover, participants exhibited substantial improvements in muscle function, as evidenced by improvements in the Time to Rise, Four-Stair Climb, and other functional endpoints. Safety monitoring throughout the trial revealed only minor adverse effects, with a positive long-term safety profile observed in most participants.

A Vision for the Future

As Avidity Biosciences continues its mission to deliver revolutionary RNA therapeutics, the launch of the MAP for del-zota represents a critical step towards addressing the unmet needs of the DMD community. With FDA recognition of del-zota's potential through designations such as Rare Pediatric Disease and Breakthrough Therapy, the company is well-positioned to make a significant impact in the field of genetic therapies.

For more details on the eligibility and enrollment criteria for the Managed Access Program, interested individuals can visit clinicaltrials.gov, where further information will be posted as it becomes available. Avidity stands at the forefront of a new era in therapy for DMD, and their continued efforts may soon offer life-changing options for those affected by this devastating condition.