ENCell said on the 27th that it applied to the European Medicines Agency (EMA) for orphan drug designation (ODD) for EN001, a candidate to treat Charcot-Marie-Tooth disease (CMT).
The company said this is a strategic move to expand the global development potential of EN001 and to secure institutional support and a business foothold in the European market in advance.
CMT is a rare neurological disease in which muscle weakness, sensory impairment, and gait abnormalities progressively worsen due to peripheral nerve damage. ENCell received orphan drug designation from the U.S. Food and Drug Administration (FDA) in Feb. last year for EN001's CMT indication.
EN001 is an allogeneic umbilical cord-derived mesenchymal stem cell (MSC)-based therapy. ENCell is currently conducting a domestic clinical trial in patients with CMT1A.