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# Orphan Status for Concert Cystic Fibrosis Therapy
- URL: https://www.fdaweb.com/orphan-status-for-concert-cystic-fibrosis-therapy/
- Published: 2017-01-20T12:00:00.000Z
- Updated: 2026-09-14T22:02:01.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5137787

FDA has granted Concert Pharmaceuticals an orphan drug designation for CTP-656, a next-generation CFTR (cystic fibrosis transmembrane conductance regulator) potentiator being developed for treating cystic fibrosis. Last month, Concert initiated a Phase 2 trial in the U.S. evaluating the drug in cystic fibrosis patients with gating mutations. Topline results from this trial are expected by year-end 2017.

  
CTP-656 is a novel CFTR potentiator that may offer next-generation, once-daily dosing, according to the company. Concert is initially developing CTP-656 as a potential monotherapy treatment for cystic fibrosis due to gating mutations of the gene that encodes for CFTR, a protein that regulates components of sweat, mucus clearance and digestion.