ENCell’s EN001 Receives Orphan Drug Designation from the U.S. FDA for Charcot-Marie-Tooth Disease

• EN001, a novel therapy under development by ENCell, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Charcot-Marie-Tooth (CMT) disease.
• Phase 1 clinical trial confirmed the safety and exploratory therapeutic effects of repeated low-dose administration of EN001; currently conducting a Phase 1b trial, with plans to complete within the year.

SEOUL, South Korea--(BUSINESS WIRE)--ENCell, a leading biopharmaceutical company for cell and gene therapy CDMO and novel drug development, announced today that its investigational drug EN001 has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of Charcot-Marie-Tooth disease (CMT).

CMT is a hereditary neuromuscular disorder that causes progressive muscle weakness and deformities in the hands and feet and, in severe cases, can lead to vision and hearing impairment. Despite being one of the most prevalent rare diseases, there are currently no approved treatments available, making it a significant challenge for both patients and healthcare providers.

EN001 is a mesenchymal stem cell therapy developed using ENCell’s proprietary ENCT (ENCell Technology) platform. This technology enhances cell longevity and optimizes the secretion of therapeutic molecules required for treatment. Once administered, EN001 targets damaged nerves, promotes the secretion of regenerative factors, and facilitates remyelination.

In October 2024, ENCell reported the results of a Phase 1 clinical trial investigating the safety and exploratory efficacy of repeated low-dose administration of EN001 in CMT type 1A patients. In the study, three patients received two doses of EN001, and dose-limiting toxicity (DLT) was assessed at eight weeks post-administration. The results showed no occurrence of DLT, no serious adverse events, and no injection-related reactions. Following this successful outcome, ENCell initiated a Phase 1b trial with a high-dose cohort in December 2024 and aims to complete the study within 2025.

An ENCell representative commented: "The U.S. FDA's Orphan Drug Designation for EN001 is a significant milestone that will accelerate the clinical development of this therapy. We are committed to successfully completing the ongoing Phase 1b trial and ensuring that CMT patients gain timely access to this innovative treatment.”

Additionally, EN001 is actively expanding its indications beyond CMT to include Duchenne Muscular Dystrophy (DMD) and Sarcopenia, further strengthening its potential as a next-generation stem cell therapy for muscular diseases.