Topline results from the Phase II trial are expected by year-end 2017.

FDA has granted orphan drug designation for Convert Pharmaceuticals' CTP-656, Concert’s next generation CFTR potentiator being developed for the treatment of cystic fibrosis (CF). In December 2016, Concert initiated a Phase II trial in the US evaluating CTP-656 in CF patients with gating mutations. Topline results from the Phase II trial are expected by year-end 2017.

"Receiving orphan drug designation is an important regulatory milestone, and we are pleased that CTP-656 for cystic fibrosis has been granted this status," said Roger Tung, President and Chief Executive Officer of Concert Pharmaceuticals. "We are developing CTP-656 to potentially offer advantages over standard of care, and our team is committed to advancing the clinical development program to address the unmet needs of individuals with CF."