“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.”

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.