ENCell’s EN001 Granted FDA Orphan Drug Designation for Charcot-Marie-Tooth Disease
ENCell, a pioneering biopharmaceutical company specializing in cell and gene therapy CDMO and novel drug development, announced that its investigational drug EN001 has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of Charcot-Marie-Tooth disease (CMT). This designation marks a major milestone in the development of EN001 and reinforces ENCell’s commitment to bringing innovative therapeutic options to patients suffering from rare and currently untreatable neuromuscular disorders.
Understanding Charcot-Marie-Tooth Disease (CMT)
Charcot-Marie-Tooth disease is one of the most common inherited neuromuscular disorders, affecting approximately 1 in 2,500 people worldwide. The disease is caused by genetic mutations that lead to progressive damage of the peripheral nerves, which are responsible for transmitting signals between the brain, spinal cord, and muscles. This results in muscle weakness, loss of sensation, foot deformities, and difficulties with walking and balance. In more severe cases, patients may experience vision and hearing impairment, significantly impacting their quality of life.
Despite its prevalence among rare diseases, CMT currently has no approved treatment options, leaving patients and healthcare providers with limited means to manage its symptoms. Given the progressive nature of the disorder, there is a substantial unmet medical need for disease-modifying therapies that can halt or reverse the damage caused by CMT.
EN001: A Promising Investigational Therapy
EN001 is an advanced mesenchymal stem cell therapy developed using ENCell’s proprietary ENCT (ENCell Technology) platform. The ENCT platform is designed to enhance cell longevity and optimize the secretion of therapeutic molecules essential for nerve regeneration. EN001 is engineered to target damaged peripheral nerves, promote the secretion of regenerative factors, and facilitate remyelination, which is the process of restoring the protective myelin sheath around nerves.
Preclinical studies of EN001 demonstrated its ability to improve nerve function, reduce inflammation, and promote nerve repair in CMT models. These promising findings paved the way for clinical development, and ENCell has been actively conducting clinical trials to evaluate the safety and efficacy of EN001 in CMT patients.
Phase 1 Clinical Trial Results
In October 2024, ENCell announced the results of its Phase 1 clinical trial evaluating the safety and exploratory efficacy of EN001 in patients with CMT type 1A, the most common subtype of the disease. The trial involved three patients who received two doses of EN001, with dose-limiting toxicity (DLT) assessed eight weeks after administration.
The results of the study were highly encouraging:
- No occurrence of dose-limiting toxicity was observed.
- No serious adverse events were reported.
- No injection-related reactions were noted.
Following the successful completion of the Phase 1 trial, ENCell initiated a Phase 1b trial in December 2024, incorporating a high-dose cohort to further assess the safety and potential therapeutic benefits of EN001. The company aims to complete the study within 2025, bringing EN001 one step closer to regulatory approval.
The Significance of FDA Orphan Drug Designation
The Orphan Drug Designation (ODD) granted by the FDA is an important regulatory milestone that provides several benefits to ENCell as it advances the development of EN001. The designation is awarded to promising therapies intended to treat rare diseases affecting fewer than 200,000 people in the United States.
With ODD status, ENCell will benefit from:
- Seven years of market exclusivity upon FDA approval of EN001.
- Tax credits for qualified clinical trial costs.
- Waiver of certain regulatory fees, including the New Drug Application (NDA) filing fee.
- Assistance from the FDA in the design of clinical trials and expedited regulatory pathways.
The FDA’s decision to grant ODD to EN001 reflects the agency’s recognition of the urgent need for new treatment options for CMT and the potential of EN001 to address this unmet medical need.
ENCell’s Commitment to Advancing EN001 and Future Therapeutic Applications
ENCell remains dedicated to accelerating the clinical development of EN001 and ensuring that CMT patients gain timely access to this innovative treatment. An ENCell representative emphasized the importance of this milestone, stating:
“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.”
Beyond Charcot-Marie-Tooth disease, ENCell is actively exploring additional therapeutic applications for EN001. Given its mechanism of action, EN001 holds promise for treating other neuromuscular and degenerative conditions. The company is currently investigating the potential of EN001 in Duchenne Muscular Dystrophy (DMD) and Sarcopenia, two conditions characterized by progressive muscle degeneration and weakness.
- Duchenne Muscular Dystrophy (DMD): DMD is a severe, progressive genetic disorder that primarily affects boys, leading to muscle wasting and early loss of ambulation. There is a critical need for regenerative therapies that can restore muscle function and slow disease progression.
- Sarcopenia: Sarcopenia is an age-related condition characterized by loss of muscle mass, strength, and function. It affects millions of elderly individuals worldwide, contributing to frailty and increased risk of falls and fractures. Stem cell-based therapies like EN001 have the potential to counteract muscle degeneration and improve overall physical function.
By expanding the indications of EN001, ENCell aims to establish its investigational therapy as a next-generation stem cell treatment for a wide range of muscular and neuromuscular diseases.
