Regulus Therapeutics Inc. (Nasdaq: RGLS), a US-based biopharmaceutical company, announced on Tuesday that it has received orphan drug designation (ODD) from the United States Food and Drug Administration (FDA) for its RGLS8429 intended to treat Autosomal Dominant Polycystic Kidney Disease (ADPKD).

RGLS8429 is a novel, next generation oligonucleotide intended to treat ADPKD aimed at inhibiting miR-17 and to preferentially target the kidney. Presently, the company is conducting a Phase one single-ascending dose study in healthy volunteers to evaluate safety, tolerability, and pharmacokinetics of the product.

The FDA's Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended to treat, diagnose, or prevent rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

Jay Hagan, Regulus Therapeutics CEO, said, '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. ADPKD is a disease of high unmet need affecting nearly 160,000 Americans. We look forward to advancing RGLS8429 through the clinic with the goal of improving future treatment options for patients in need.'