Policy & Regulation
Alebund Pharmaceuticals' AP303 receives US FDA Orphan Drug Designation
1 April 2024 -

Alebund Pharmaceuticals, a China-based integrated biopharmaceutical company, announced on Friday that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for AP303 for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

AP303 is a novel drug candidate developed in-house by the company. According to Alebund, it has demonstrated a meaningful improvement of renal survival in an ADPKD mice model and has completed its first-in-human study in healthy subjects in Australia. The company is ready for a Phase II trial in ADPKD.

Alebund said that this Orphan Drug Designation -- a special status granted to support the development and evaluation of potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US -- reflects the need for more new treatment options and the potential for AP303 to address the pressing needs of people living with ADPKD.

Dr Gavin Xia, Alebund co-founder, chairman and CEO, said: "We are very excited that AP303 has been granted Orphan Drug Designation by the FDA. It is an important milestone for Alebund as well as our efforts to address the significant unmet medical needs with ADPKD. We look forward to advancing AP303 to improve future treatment options for patients in need."