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.
About NMD Pharma
NMD Pharma is a clinical-stage biotech company founded with a focus to deliver life-transforming therapies for people living with neuromuscular diseases. NMD Pharma have a unique translational muscle electrophysiology platform leveraging in-depth know-how of muscle physiology and muscular disorders for discovering and developing first-in-class therapeutics in neuromuscular diseases with a significant unmet medical need. NMD Pharma is the leading company utilizing ClC-1 Cl- ion channel inhibitors for neuromuscular diseases and currently has one clinical-stage product candidate for the treatment of myasthenia gravis. Company headquartered in Aarhus, Denmark, and collaborate with internationally renowned companies and key opinion leaders in the field of neuromuscular diseases.
September 28, 2022
