PepGen Receives U.S. FDA Fast Track Designation for PGN-EDODM1 for the Treatment of Myotonic Dystrophy Type 1

PepGen Inc., a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PGN-EDODM1, an investigational candidate for the treatment of myotonic dystrophy type 1 (DM1). “Receiving Fast Track designation from the FDA for PGN-EDODM1 is a significant milestone in our efforts to deliver a potentially transformative therapy to the DM1 community,” said James McArthur, Ph.D., President and CEO of PepGen. “Patients with DM1 currently have no available treatment options that target the root cause of the disease, which leads to progressive neuromuscular symptoms and reduction in life expectancy. Following robust preclinical data, we are now evaluating PGN-EDODM1 in the ongoing FREEDOM-DM1 Phase 1 trial and expect to report preliminary data later this year. We believe that PGN-EDODM1 has the potential to be disease-modifying and improve outcomes for patients living with DM1.”

The FDA’s Fast Track designation is a process designed to facilitate the development and expedite the review of drug candidates that treat serious conditions and fill an unmet medical need, with the goal of getting important new drugs to the patient earlier. Once a drug candidate receives Fast Track designation, early and frequent communication between the FDA and the drug company is encouraged throughout the entire drug development and regulatory review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

PGN-EDODM1 is currently being evaluated in the ongoing FREEDOM-DM1 Phase 1 clinical trial for the treatment of people living with DM1. The Company expects to report initial data from the trial in 2024. (ClinicalTrials.gov identifier: NCT06204809)

The Company previously announced that the FDA granted Orphan Drug Designation to PGN-EDODM1 in September 2023.