Regulus Therapeutics Gets FDA Orphan Designation for RGLS8429

By Colin Kellaher

The U.S. Food and Drug Administration granted orphan-drug designation to Regulus Therapeutics Inc.'s RGLS8429 for the treatment of autosomal dominant polycystic kidney disease, a leading cause of end-stage renal disease, the company said Tuesday.

There are currently limited treatment options for autosomal dominant polycystic kidney disease, which affects nearly 160,000 people in the U.S., with about half of patients developing end-stage renal disease by age 60 requiring dialysis or transplantation, the San Diego clinical-stage biopharmaceutical company said.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.

Regulus is currently conducting a Phase 1 single-ascending dose study in healthy volunteers to assess safety, tolerability and pharmacokinetics of RGLS8429.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

06-21-22 0838ET