Concert Pharmaceuticals has announced that the FDA has granted Orphan Drug designation for CTP-656, Concert’s next generation CFTR potentiator being developed for the treatment of cystic fibrosis. In December 2016, Concert initiated a phase II trial in the U.S. evaluating CTP-656 in cystic fibrosis 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, Ph.D., 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 cystic fibrosis.”
The Orphan Drug Act provides incentives for companies to develop products for rare diseases affecting fewer than 200,000 people in the U.S. Incentives may include tax credits related to clinical trial expenses, an exemption from the FDA user fee, FDA assistance in clinical trial design and potential market exclusivity for seven years following approval.
CTP-656 is a novel CFTR potentiator that may offer next generation, once-daily dosing and was developed by Concert’s novel application of deuterium chemistry to modify ivacaftor. Ivacaftor is marketed by Vertex Pharmaceuticals under the brand name Kalydeco. Concert is initially developing CTP-656 as a potential monotherapy treatment for cystic fibrosis due to gating mutations of the gene that encodes for cystic fibrosis transmembrane conductance regulator (CFTR), a protein, which regulates components of sweat, mucus clearance and digestion. The company also intends to enable potentially more effective combinations to treat other mutations, including homozygous F508del, by partnering with other potentially complementary CFTR modulators.
Cystic fibrosis is a life-threatening, hereditary genetic disease that has systemic effects and can cause significantly reduced lung and digestive system function. According to the Cystic Fibrosis Foundation, an estimated 70,000 people worldwide have cystic fibrosis.