FDA Grants Orphan Drug Designation To NMD Pharma’s NMD670 For Charcot-Marie-Tooth Disease Treatment

NMD Pharma is a biotech company focused on developing new treatments for neuromuscular diseases. The firm has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to their experimental drug, NMD670. This oral medication, designed to block the skeletal muscle chloride ion channel ClC-1, is being developed to treat Charcot-Marie-Tooth disease (CMT). 

This rare hereditary condition leads to muscle weakness and fatigue. Currently, no approved treatments exist for CMT. CMT affects approximately 136,000 people in the U.S. and over 3 million globally. The disease presents with muscle weakness, atrophy, fatigue, and sensory issues, which can significantly impact daily life.

“NMD Pharma is committed to addressing the needs of patients living with neuromuscular diseases such as CMT, and we are thrilled that the FDA has granted orphan drug designation to NMD670. 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,” said Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma 

In November 2024, NMD Pharma began a Phase 2 clinical trial called SYNAPSE-CMT to test NMD670 in adults with confirmed CMT1 or CMT2. The trial, involving 80 participants, will take place over 21 days across sites in the U.S. and Europe, with eligible patients encouraged to join. 

This isn’t the first time NMD670 has caught the FDA’s attention back in September 2022, it received orphan drug status for treating generalised myasthenia gravis (gMG). The FDA’s orphan drug designation is given to therapies targeting rare diseases that affect fewer than 200,000 people in the U.S., providing essential incentives to encourage drug development for these conditions.