The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to NMD Pharma’s oral lead development programme, NMD670, designed to treat Charcot-Marie-Tooth disease (CMT).
A small molecule inhibitor of the skeletal muscle-specific chloride ion channel 1 (CIC-1), NMD670 entered the Phase II SYNAPSE-CMT trial last November, involving 80 adult subjects with genetically confirmed CMT1 or CMT2 subtypes.
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The study involves a twice-a-day oral dose over 21 days and is conducted across clinical sites in Europe and the US.
The FDA’s ODD for the therapy follows a previous grant for the treatment of generalised myasthenia gravis (gMG) in September 2022.
According to the company, the inhibition of CIC-1 can enhance muscle responsiveness to weak signals, potentially ‘improving’ neuromuscular transmission and restoring skeletal muscle function.
This approach claims to have yielded clinical evidence of CIC-1 inhibition in myasthenia gravis and preclinical evidence in other conditions such as sarcopenia, CMT, and spinal muscular atrophy.
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By GlobalDataNMD Pharma CEO Thomas Holm Pedersen said: “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 ODD to NMD670.
“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.”
Marked by weakness in muscles, atrophy, fatigue, and sensory deficits, CMT significantly impacts the quality of life of those affected. The company noted that an estimated 136,000 people in the US and more than three million globally are living with this hereditary neuropathy.
