Nivalis Therapeutics, Inc., a clinical stage pharmaceutical company focused on developing innovative solutions for people with cystic fibrosis (“CF”), announced the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation to the Company's lead investigational drug, N91115, a novel stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
“The Orphan Drug Designation represents an important milestone in the development and regulatory strategy for N91115 and underscores the unmet need that remains in treating CF,” said Jon Congleton, president and chief executive officer of Nivalis. "We look forward to the continued clinical advancement of this first-in-class CFTR stabilizer, a new approach to modulating the defective CFTR protein.
The Company recently initiated a Phase 2, 12-week, double-blind, randomized, placebo-controlled, parallel group study to investigate the efficacy and safety of N91115 in 135 adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with Orkambi™. Results of this study are planned to be reported in the second half of 2016. N91115 works through a novel mechanism of action called S-nitrosoglutathione reductase (GSNOR) inhibition that is presumed to modulate the unstable and defective CFTR protein responsible for CF.
The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.
The Company will also seek Fast Track status for its development program with N91115. A Fast Track designation enables more frequent interactions with the FDA to expedite the development and review process for drugs intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
