NMD Pharma has announced that they have been granted orphan drug designation by the Food and Drug Administration for NMD670, their novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of Charcot-Marie-Tooth disease.

Orphan drug designations are granted by the FDA and provides many advantages that can facilitate development and approval of drugs for rare disorders, which affect fewer than 200,000 people in the U.S.

“We are thrilled to see progress being made towards addressing the critical need for effective treatments for CMT,” said Laura M. MacNeill, CMTRF CEO. “For individuals and families affected by this disease, this FDA designation represents a beacon of hope and bring us closer to a future where no one has to live without options to manage the debilitating effects of this disease.”

In December 2024, NMD Pharma published findings from ESTABLISH, an observational clinical study in CMT Type 1 and 2 patients and healthy patients, which revealed that CMT patients have neuromuscular junction transmission dysfunction that correlates with disease severity. Additionally, preclinical studies demonstrated that treatment with NMD Pharma’s drug candidate, NMD670, corrected neuromuscular junction deficits and led to improved muscle contractile function in two different mouse models of CMT.

Additionally, In November 2024, NMD Pharma announced the initiation of Phase 2 clinical trial (SYNAPSE-CMT) and dosed their first patient at the Austin Neuromuscular Center in Austin, TX.

CMTRF did not fund this project but is very pleased by its success and thanks NMD Pharma for their work in getting us one step closer to treatments and cures for CMT.