FDA Grants Orphan Drug Status to CF Therapy Now in Phase 2 Study
The U.S. Food and Drug Administration (FDA) has designated Concert Pharmaceuticals‘ next-generation therapy, CTP-656, an Orphan Drug to treat cystic fibrosis (CF). CTP-656 is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that, according to the company’s website, was developed by applying deuterium chemistry to modify Kalydeco (ivacaftor). Concert has said…