NMD Pharma A/S, a CMTA-STAR Alliance Partner, recently announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to NMD670, the investigational drug the company is studying in its ongoing Phase II SYNAPSE-CMT clinical trial. According to the company, NMD670 targets the skeletal muscle-specific chloride ion channel ClC-1, that could address muscle weakness and fatigue, two common symptoms of CMT.
“FDA orphan drug designation not only highlights the urgent need for novel treatments for CMT but also underscores the therapeutic potential of our ClC-1 inhibitor approach,” said Thomas Holm Pedersen, CEO of NMD Pharma. “We are committed to advancing NMD670 to bring effective solutions to the CMT community.”
NMD670 is currently being evaluated in the SYNAPSE-CMT Phase 2 clinical trial, which began in November 2024. This study is investigating the effects of NMD670 for 80 adult patients with genetically confirmed CMT1 or CMT2 subtypes across clinical sites in the U.S. and Europe.
CMTA celebrates this encouraging progress from our CMTA-STAR Alliance Partner, NMD Pharma. This milestone reflects the dedication and innovation driving advancements in CMT research and brings renewed hope to individuals and families living with CMT1 and CMT2.
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Published on: January 22, 2025