Regulus Therapeutics Inc. (NASDAQ:RGLS) received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its lead drug RGLS8429 in the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD).
RGLS8429 is being investigated in a Phase 1 single-ascending dose study in healthy volunteers.
Jay Hagan, CEO, commented : ”Patients living with ADPKD currently have limited treatment options and approximately half of patients develop end-stage renal disease by age 60 requiring dialysis or transplantation. We look forward to advancing RGLS8429 through the clinic with the goal of improving future treatment options for patients in need.”
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs.
FDA grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. It also provides benefits of seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.
Regulus Therapeutics shares are trading at $0.29.
