FDA grants orphan drug designation to Concert’s cystic fibrosis treatment

Last month, the company had begun a phase 2 trial in the US for studying CTP-656 in patients with cystic fibrosis and having gating mutations. The phase 2 trial’s topline results are likely to be released by the year end.

CTP-656 is a next generation cystic fibrosis transmembrane conductance regulator (CFTR) potentiator which Concert developed through the application of deuterium chemistry to modify ivacaftor which is marketed by Vertex Pharmaceuticals under the brand name Kalydeco.

Concert Pharmaceuticals president and CEO Roger Tung, Ph.D. said: "Receiving orphan drug designation is an important regulatory milestone, and we are pleased that CTP-656 for cystic fibrosis has been granted this status.

"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 cystic fibrosis."

Initially, CTP-656 will be developed by Concert as a potential monotherapy treatment for cystic fibrosis caused by gating mutations of the CFTR encoding gene.

CFTR, in turn is a protein responsible for regulating components of mucus clearance, sweat and digestion. Later on, Concert plans to enable more effective combinations with CTP-656 for treating other mutations via partnership with complementary CFTR modulators.

The orphan drug designation allows the company to get incentives to develop the treatment for the rare hereditary genetic disease that affects lesser than 200,000 people in the US.

Incentives for Concert could be in the form of tax credits associated with expenditure on clinical trials, exemption from the user fee to FDA, assistance from the state regulator in designing of clinical trial and possible market exclusivity for seven years if CTP-656 gets approved for cystic fibrosis.