NMD Pharma Receives US FDA Orphan Drug Designation for NMD670 in CMT

NMD Pharma A/S, a biotech firm in the clinical stage focused on creating innovative and enhanced therapies for individuals with neuromuscular disorders, revealed that the U.S. Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NMD670, a new, oral, small molecule blocker of the skeletal muscle-specific chloride ion channel ClC-1, aimed at addressing Charcot-Marie-Tooth disease (CMT).

CMT is an inherited nerve disorder marked by muscle weakness and fatigue, for which there are no approved treatments. Approximately 136,000 people in the US are affected by CMT, and it is believed that more than 3 million people globally suffer from CMT. Clinical characteristics of this uncommon neuromuscular disorder involve motor signs and symptoms like muscle weakness, muscle wasting, and tiredness, along with sensory impairments that, together, can significantly diminish quality of life.

"NMD Pharma is committed to addressing the need of patients living with neuromuscular diseases such as CMT, and we are thrilled that the FDA has granted orphan drug designation to NMD670,” commented Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma. "Based on positive results from preclinical studies and the recently published ESTABLISH1 CMT observational study, this designation not only highlights the urgent need for novel, effective treatments for this rare disease, but also underscores the therapeutic potential of our skeletal muscle-specific ClC-1 inhibitor approach to address the associated muscle weakness and fatigue."