Alebund AP303 Granted FDA Orphan Drug Designation

Alebund AP303 Granted FDA Orphan Drug Designation

By, Admin 1 April 2024

Alebund AP303 Granted FDA Orphan Drug Designation for Autosomal Dominant Polycystic Kidney Disease

Overview

Alebund Pharmaceuticals, a biopharmaceutical company dedicated to pioneering therapies for renal diseases and related chronic ailments, announced today that the US Food and Drug Administration (FDA) has bestowed Orphan Drug Designation (ODD) upon AP303 for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

Words from CEO: Alebund

Dr. Gavin Xia, Co-founder, Chairman, and CEO of Alebund, expressed enthusiasm, stating, ""The FDA's granting of Orphan Drug Designation to AP303 marks a significant milestone for Alebund and our commitment to addressing the substantial unmet medical needs associated with ADPKD. We eagerly anticipate advancing AP303 to enhance treatment options for patients in need.""

About AP303 and Epidemiology

  • AP303, an innovative drug candidate developed internally by Alebund, has shown promising results in improving renal survival in an ADPKD mouse model. 
  • Following the completion of its first-in-human study in healthy subjects in Australia, AP303 is poised for a Phase II trial in ADPKD patients. 
  • ADPKD affects between 1 in 400 and 1 in 1,000 live births and stands as a significant contributor to end-stage kidney disease (ESKD) necessitating renal replacement therapy. 
  • The Orphan Drug Designation underscores the demand for novel treatment options and the potential of AP303 to address the urgent needs of individuals living with ADPKD.

ODD: Specialized Status for Development of Treatments

  • The US FDA's ODD is a specialized status granted to facilitate the development and assessment of potential new medications aimed at treating, diagnosing, or preventing rare diseases or disorders affecting fewer than 200,000 people in the US. 
  • This designation offers incentives to advance the development of treatments for rare diseases, including marketing exclusivity, tax credits for clinical research expenses, and exemption from prescription drug user fees.

About ADPKD

  • ADPKD, the most prevalent monogenic kidney disease globally, is a leading cause of end-stage kidney disease in adults. 
  • It is characterized by the formation and progressive enlargement of multiple cysts throughout the kidney tissue. 
  • ADPKD primarily stems from mutations in the PKD1 gene (in 78% of cases) or the PKD2 gene (in 15% of cases). 
  • Current treatment recommendations for ADPKD patients include antihypertensive therapy, dietary modifications, and pharmacotherapy for a limited subset of eligible patients. 
  • However, there is a pressing need for more universally applicable treatment options with fewer side effects to effectively slow kidney function decline and enhance kidney outcomes in ADPKD patients.

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