The U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for NMD Pharma’s NMD670, a novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of Charcot-Marie-Tooth disease (CMT).
CMT is a hereditary neuropathy characterized by muscle weakness and fatigue with no approved therapies. An estimated 136,000 individuals in the US have CMT and it is estimated that over 3 million individuals worldwide live with CMT. Clinical features of this rare neuromuscular disease include motor signs and symptoms such as muscle weakness, muscle atrophy, and fatigue, as well as sensory deficits that, in combination, may cause substantial reduction in 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 ESTABLISH 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.”
In November 2024, NMD Pharma announced the initiation of a Phase 2 clinical trial, named SYNAPSE-CMT, of a twice daily oral dose of NMD670 over 21 days in 80 adult patients with any genetically confirmed CMT1 or CMT2 subtype, taking place across clinical sites in both the U.S. and Europe.
In addition, NMD670 was previously granted ODD by the FDA for the treatment of generalized myasthenia gravis (gMG) in September 2022.
FDA ODD provides orphan status to therapies intended for the prevention, diagnosis or treatment of diseases affecting fewer than 200,000 people in the US.
