Polycystic Kidney Disease Drug Development: A Conference Report.
Journal
Kidney medicine
ISSN: 2590-0595
Titre abrégé: Kidney Med
Pays: United States
ID NLM: 101756300
Informations de publication
Date de publication:
Mar 2023
Mar 2023
Historique:
entrez:
26
1
2023
pubmed:
27
1
2023
medline:
27
1
2023
Statut:
epublish
Résumé
Autosomal dominant polycystic kidney disease (ADPKD) is part of a spectrum of inherited diseases that also includes autosomal recessive polycystic kidney disease, autosomal dominant polycystic liver disease, and an expanding group of recessively inherited disorders collectively termed hepatorenal fibrocystic disorders. ADPKD is the most common monogenic disorder frequently leading to chronic kidney failure with an estimated prevalence of 12 million people worldwide. Currently, only one drug (tolvaptan) has been approved by regulatory agencies as disease-modifying therapy for ADPKD, but, given its mechanism of action and side effect profile, the need for an improved therapy for ADPKD remains a priority. Although significant regulatory progress has been made, with qualification of total kidney volume as a prognostic enrichment biomarker and its later designation as a reasonably likely surrogate endpoint for progression of ADPKD within clinical trials, further work is needed to accelerate drug development efforts for all forms of PKD. In May 2021, the PKD Outcomes Consortium at the Critical Path Institute and the PKD Foundation organized a PKD Regulatory Summit to spur conversations among patients, industry, academic, and regulatory stakeholders regarding future development of tools and drugs for ADPKD and autosomal recessive polycystic kidney disease. This Special Report reviews the key points discussed during the summit and provides future direction related to PKD drug development tools.
Identifiants
pubmed: 36698747
doi: 10.1016/j.xkme.2022.100596
pii: S2590-0595(22)00229-1
pmc: PMC9867973
doi:
Types de publication
Journal Article
Langues
eng
Pagination
100596Informations de copyright
© 2022 The Authors.
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