China-based Alebund Pharmaceuticals announced that the U.S. FDA granted Orphan Drug Designation to its investigational drug for the treatment of autosomal dominant polycystic kidney disease (ADPKD).
AP303, a pan-inhibitor of sodium-dependent phosphate transporters, completed a first-in-human study in healthy subjects in Australia and is set to begin a phase 2 trial. ADPKD, which has a prevalence of between 1 in 400 and 1 in 1,000 live births, leads to end-stage kidney disease (ESKD) and requires renal replacement therapy.
This designation is intended to facilitate drug development for rare diseases affecting fewer than 200,000 people in the U.S., offering benefits such as marketing exclusivity, tax credits for clinical research, and exemption from prescription drug user fees.
Alebund, established in Shanghai in 2018, specializes in the development of treatments for kidney diseases and their complications. The company has a diverse pipeline of drug candidates targeting various renal diseases, including ADPKD, with several programs in clinical stages.
