Clinical-stage biotech company NMD Pharma's NMD670 has been granted orphan drug designation (ODD) by the FDA.
The novel small molecule inhibitor aims to target the muscle-specific chloride ion channel CIC-1, and will be used to treat Charcot-Marie-Tooth disease (CMT) if approved.
There are currently no drugs that have been approved for this rare disease, although more than three million individuals suffer from the disease globally.
The hereditary neuropathy causes muscle weakness, atrophy and fatigue in affected individuals, while also impacting a patient's quality of life significantly.
NMD670
The small molecule, which was discovered and developed by NMD Pharma, is currently being tested in a Phase II clinical trial, SYNAPSE-CMT.
If approved, the therapeutic will be given orally twice daily to patients with CMT1 and CMT2 subtypes.
Patients with these disease indications in the US and Europe are being encouraged to participate in the study.
NMD Pharma is also exploring the efficacy of NMD670 in generalised myasthenia gravis (gMG).
CEO of NMD Pharma, Thomas Holm Pedersen, commented: “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,”
“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.”