Momentum is building for the development of a treatment for Charcot–Marie–Tooth disease, a hereditary peripheral neuropathy for which there is still no therapy.
ENCell, a corporations specializing in contract development and manufacturing of advanced biopharmaceuticals (CDMO) and new drug development, said on the 17th that EN001, under development as a treatment for Charcot–Marie–Tooth disease 1A, obtained approval from the Ministry of Food and Drug Safety for a phase 2a clinical trial.
The company filed in Aug. with the regulator to change and integrate phases 1b and 2a, passed the review process, and received final approval.
Normally, new drugs can be commercialized after completing phases 1 through 3, but for rare and intractable diseases with no appropriate treatments on the market, they can be commercialized after phase 2 through an accelerated review system.
Charcot–Marie–Tooth disease, also known as the Samsung family's hereditary disease, is a rare disorder caused by genetic abnormalities. Muscle atrophy and deformities of the hands and feet, and loss of motor and sensory function make walking and daily life difficult. There is currently no approved treatment worldwide.
ENCell's EN001 is an umbilical cord–derived mesenchymal stem cell therapy, developed and manufactured using mesenchymal stem cells obtained at an early culture stage. The company has a cell culture platform technology. The technology suppresses cellular aging and enhances the secretion of therapeutic factors involved in nerve regeneration and inflammation control, helping restore the function of damaged peripheral nerves.
ENCell previously confirmed the safety of repeated dosing and therapeutic effects through a phase 1 (1a and 1b) clinical trial in patients. The remaining hurdle before marketing is phase 2 (2a and 2b). If development succeeds, it will become the world's first mesenchymal stem cell therapy for patients with Charcot–Marie–Tooth disease.
ENCell is aiming for early commercialization. The company said it plans to apply for designation under the accelerated review system (Accelerated Review) after completing phase 2a of EN001 and pursue conditional approval (Conditional Approval).
The company is also preparing to apply for a treatment plan to implement patient treatment through the advanced regenerative medicine regulatory sandbox project.
Before the amendment to the Act on the Safety and Support of Advanced Regenerative Medicine and Advanced Biopharmaceuticals (the Advanced Regenerative Medicine Act), it could only be used for research purposes for patients with severe, rare, and intractable diseases, but an amendment expanding the disease groups and legally allowing treatment took effect in Feb. this year. In addition, through the advanced regenerative medicine regulatory sandbox, corporations can apply for regenerative medicine treatment immediately after completing clinical trials, which previously only medical institutions could do.
The company said, "In this phase 2a trial, based on the phase 1b results, we will derive the optimal dose of EN001 and prove a definitive therapeutic effect," and added, "Through accelerated review, we will do our best to ensure that patients can receive treatment benefits as quickly as possible."