Alebund Pharmaceuticals Receives FDA Orphan Drug Designation for AP303 in ADPKD Treatment

01 April 2024 | Monday | News

The designation marks a significant step forward in developing new therapies for autosomal dominant polycystic kidney disease, with Alebund Pharmaceuticals advancing AP303 to Phase II trials
Image Source : Public Domain

Image Source : Public Domain

 Alebund Pharmaceuticals ("Alebund" or the "Company"), an integrated biopharmaceutical company focusing on developing innovative therapies for the treatment of renal diseases and related chronic conditions, announced today that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AP303 for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

"We are very excited that AP303 has been granted Orphan Drug Designation by the FDA", Dr. Gavin Xia, Co-founder, Chairman, and CEO of Alebund commented. "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."

AP303, a novel drug candidate developed in-house by Alebund, has demonstrated a meaningful improvement of renal survival in an ADPKD mice model. AP303 has completed its first-in-human study in healthy subjects in Australia and is ready for a Phase II trial in ADPKD. The prevalence of ADPKD is reported to be between 1 in 400 and 1 in 1,000 live births; it is an important cause of end-stage kidney disease (ESKD) requiring renal replacement therapy [1]. This Orphan Drug Designation reflects the need for more new treatment options and the potential for AP303 to address the pressing needs of people living with ADPKD.

The US FDA's ODD is 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. The designation provides incentives to advance the development of treatments for rare diseases, including marketing exclusivity, tax credits for clinical research costs, and exemption from prescription drug user fees.

Stay Connected

Sign up to our free newsletter and get the latest news sent direct to your inbox


Forgot your password?



Lost your password? Please enter your email address. You will receive a link to create a new password.

Back to log-in