The Food and Drug Administration (FDA) has granted Orphan Drug designation for Concert Pharmaceuticals’ CTP-656 (deuterated ivacaftor) for the treatment of cystic fibrosis.

CTP-656 is currently being investigated in a Phase 2 trial evaluating the drug in cystic fibrosis patients with gating (class III) mutations. Results from the study are expected by year-end 2017. 

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CTP-656, a novel, next generation CFTR potentiator, is a once-daily modified form of ivacaftor (Kalydeco; Vertex) developed through the application of deuterium chemistry. CTP-656 has shown to demonstrate superior pharmacokinetic profile compared to Kalydeco in a Phase 1 cross-over study, including a reduced rate of clearance, longer half-life, substantially increased exposure and greater plasma levels at 24 hours.

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