NMD Pharma’s Charcot-Marie-Tooth disease treatment granted ODD by FDA
Natalia Elliot | January 6, 2025 | News story | | CMT, FDA, NMD Pharma, Orphan Drugs, Pain, nervous system, research and development
Biotech company NMD Pharma has announced that the US Food and Drug Association (FDA) have granted its Charcot-Marie-Tooth (CMT) disease treatment NMD670 orphan drug designation (ODD). NMD670 was previously granted ODD by the FDA for generalised myasthenia gravis in September 2022.
NMD670 is a small molecule inhibitor of the skeletal muscle-specific chloride ion channel CIC-1. In November 2024, NMD Pharma announced the initiation of the phase 2 clinical trial SYNAPSE-CMT, which will test a twice-daily oral dose of NMD670 over 21 days in 80 adult patients with any genetically confirmed CMT1 or CMT2 subtype. An estimated 136,000 US (three million globally) individuals have CMT – a hereditary neuropathy which leads to muscle weakness, fatigue and sensory deficits.
Thomas Holm Pedersen, chief executive officer of NMD Pharma, stated: “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 ODD 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.”
James Spargo
6/1/25
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