NMD Pharma’s NMD670 Gets Orphan Drug Designation

NMD Pharma has received FDA’s Orphan Drug designation for its NMD670 candidate for treatment of myasthenia gravis.

The investigational drug is a small molecule inhibitor of the skeletal muscle specific chloride ion channel (CIC-1) which plays an important role on skeletal muscle excitability.

The FDA granted the designation based on preclinical efficacy data in animal models, which showed that NMD670 enhances neuromuscular transmission and restores skeletal muscle function.

The investigational drug also went through a combined phase 1/2a clinical trial. Results from that study are due out soon.