슬롯사이트 볼트카지노메이저 'EN001' designated as orphan drug for Charcot-Marie-Tooth disease by U.S. FDA

[by Lee, Young Sung] 슬롯사이트 볼트카지노메이저 announced on February 28 that its new drug pipeline, 'EN001,' currently under development, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of Charcot–Marie–Tooth disease (CMT).

슬롯사이트 볼트카지노메이저–Marie–Tooth disease (CMT) is a genetic disorder that causes deformities in the hands and feet, muscle atrophy, and, in severe cases, can result in vision and hearing loss. Although it is a rare disease with a relatively high incidence, there has been no approved treatment to date, presenting a significant challenge for both patients and medical professionals.

EN001 is a mesenchymal stem cell treatment material incubated using 슬롯사이트 볼트카지노메이저's proprietary ENCT (슬롯사이트 볼트카지노메이저 Technology). The company explains that EN001 has the ability to suppress cell aging and secrete

substances essential for treatment. Notably, it targets damaged nerves, secreting therapeutic agents that aid in the regeneration of nerve myelin.

In October 2024, 슬롯사이트 볼트카지노메이저 reported the safety and exploratory therapeutic effects observed in the low-dose group during the repeated-administration clinical trial of EN001 treatment, targeting patients with CMT type 1A.

After administering EN001 twice to three patients in the low-dose group, DLT (Dose-Limiting Toxicity) was assessed at the 8-week point. As a result, no DLT occurred in any of the patients. Additionally, there were no major adverse events or infusion-related reactions. 슬롯사이트 볼트카지노메이저 plans to successfully complete the phase 1b clinical trial within the year, beginning with the administration of EN001 to patients in the high-dose phase 1b clinical trial starting in December.

“The EN001 treatment, which 슬롯사이트 볼트카지노메이저 has been developing intensively, has been granted orphan drug designation by the U.S. FDA, providing more flexibility in the clinical trial process,” an 슬롯사이트 볼트카지노메이저 official said. “We are committed to successfully completing the ongoing phase 1b clinical trial so that EN001 can be made available to Korean CMT patients in a timely manner.”

On the other hand, following the implementation of the Ministry of Health and Welfare's revised bill on February 21, which introduces 'Advanced Regenerative Medicine Treatment' as a new framework for advanced regenerative medicine, 슬롯사이트 볼트카지노메이저 is broadening its interactions with the medical community to ensure the timely delivery of desired treatment options to medical institutions and patients with rare diseases.