Article ; Online: Drug Development in Autosomal Dominant Polycystic Kidney Disease: Opportunities and Challenges.
Advances in kidney disease and health
2023 Volume 30, Issue 3, Page(s) 261–284
Abstract: Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary disorder characterized by relentless growth of innumerable renal cysts bilaterally, associated with decline in glomerular filtration rate over the course of decades. The burden of ADPKD ...
| Abstract | Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary disorder characterized by relentless growth of innumerable renal cysts bilaterally, associated with decline in glomerular filtration rate over the course of decades. The burden of ADPKD and its treatment is associated with a significant economic and societal cost. Despite several clinical studies conducted over the past decade, only one treatment has been approved by regulatory agencies to slow disease progression in ADPKD. Elucidating feasible endpoints and clear regulatory pathway may stimulate interest in developing and translating novel therapeutics. This review summarizes the recent progress, challenges, and opportunities in drug development for ADPKD. We discuss the traditional and accelerated regulatory approval pathways, the various clinical trials endpoints, and biomarkers in ADPKD. Furthermore, we propose strategies that could optimize the clinical trial design in ADPKD. Finally, we owe it to our ADPKD patient community to strive for international collaborative studies geared toward discovery and validation of surrogate endpoints and to rally for funded infrastructure that would allow phase 3 master protocols in ADPKD. These advances will serve to derisk and potentially accelerate the development of therapies and eventually bring hope to patients and families who endure through this devastating disease. |
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| MeSH term(s) | Humans ; Polycystic Kidney, Autosomal Dominant/drug therapy ; Disease Progression ; Glomerular Filtration Rate ; Biomarkers ; Drug Development |
| Chemical Substances | Biomarkers |
| Language | English |
| Publishing date | 2023-04-24 |
| Publishing country | United States |
| Document type | Journal Article ; Review |
| ZDB-ID | 3156601-7 |
| ISSN | 2949-8139 |
| ISSN (online) | 2949-8139 |
| DOI | 10.1053/j.akdh.2023.01.004 |
| Database | MEDical Literature Analysis and Retrieval System OnLINE |
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