NMD Pharma’s NMD670 Secures the US FDA’s Orphan Drug Designation to Treat Charcot-Marie-Tooth Disease
Shots:
- The US FDA has granted ODD to NMD670 for treating Charcot-Marie-Tooth disease (CMT). It is small molecule that inhibits CIC-1 to enhances muscle responsiveness, improving neuromuscular transmission & restoring function
- NMD670 (PO, BID) is under P-IIa (SYNAPSE-CMT) evaluation for its efficacy, safety & tolerability in adults (n=80) with CMT1 or CMT2 subtype for over 21 days across the US & EU. It will assess changes in 6-minute walk test (6MWT), time to complete the 10-meter walk/run test (10MW/R) & time to complete timed-up-and-go test (TUG)
- NMD Pharma is also conducting three P-II global trials of NMD670 for spinal muscular atrophy (SMA) type 3, gMG & CMT, with data expected between H2’25 & H1’26
Ref: NMD Pharma| Image: NMD Pharma
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
