Polycystic Kidney Disease Drug Development: A Conference Report
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 fi...
| Main Authors: | , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2023-03-01
|
| Series: | Kidney Medicine |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2590059522002291 |
| _version_ | 1811154729442476032 |
|---|---|
| author | Max C. Liebau Djalila Mekahli Ronald Perrone Belle Soyfer Sorin Fedeles |
| author_facet | Max C. Liebau Djalila Mekahli Ronald Perrone Belle Soyfer Sorin Fedeles |
| author_sort | Max C. Liebau |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-10T04:20:53Z |
| format | Article |
| id | doaj.art-a51b69e5577e4313966d6d84ac980f92 |
| institution | Directory Open Access Journal |
| issn | 2590-0595 |
| language | English |
| last_indexed | 2024-04-10T04:20:53Z |
| publishDate | 2023-03-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Kidney Medicine |
| spelling | doaj.art-a51b69e5577e4313966d6d84ac980f922023-03-11T04:20:23ZengElsevierKidney Medicine2590-05952023-03-0153100596Polycystic Kidney Disease Drug Development: A Conference ReportMax C. Liebau0Djalila Mekahli1Ronald Perrone2Belle Soyfer3Sorin Fedeles4Department of Pediatrics, Center for Family Health, Center for Rare Diseases, and Center for Molecular Medicine, University Hospital Cologne and Faculty of Medicine, University of Cologne, Cologne, Germany; Address for Correspondence: Max Liebau, MD, PhD, Department of Pediatrics, Center for Family Health, Center for Rare Diseases, and Center for Molecular Medicine, University Hospital Cologne and Faculty of Medicine, University of Cologne, Cologne, Germany.Department of Pediatric Nephrology, University Hospitals Leuven, Leuven, Belgium; PKD Research Group, Department of Development and Regeneration, KU Leuven, Leuven, Leuven, BelgiumDivision of Nephrology, Department of Medicine, Tufts Medical Center and Tufts University School of Medicine, Boston, MassachusettsR. Ken Coit College of Pharmacy, University of Arizona, Tucson, ArizonaCritical Path Institute, Polycystic Kidney Disease Outcomes Consortium, Tucson, Arizona; Sorin Fedeles, PhD, MBA, Critical Path Institute, Polycystic Kidney Disease Outcomes Consortium, Tucson, AZ 85718.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.http://www.sciencedirect.com/science/article/pii/S2590059522002291 |
| spellingShingle | Max C. Liebau Djalila Mekahli Ronald Perrone Belle Soyfer Sorin Fedeles Polycystic Kidney Disease Drug Development: A Conference Report Kidney Medicine |
| title | Polycystic Kidney Disease Drug Development: A Conference Report |
| title_full | Polycystic Kidney Disease Drug Development: A Conference Report |
| title_fullStr | Polycystic Kidney Disease Drug Development: A Conference Report |
| title_full_unstemmed | Polycystic Kidney Disease Drug Development: A Conference Report |
| title_short | Polycystic Kidney Disease Drug Development: A Conference Report |
| title_sort | polycystic kidney disease drug development a conference report |
| url | http://www.sciencedirect.com/science/article/pii/S2590059522002291 |
| work_keys_str_mv | AT maxcliebau polycystickidneydiseasedrugdevelopmentaconferencereport AT djalilamekahli polycystickidneydiseasedrugdevelopmentaconferencereport AT ronaldperrone polycystickidneydiseasedrugdevelopmentaconferencereport AT bellesoyfer polycystickidneydiseasedrugdevelopmentaconferencereport AT sorinfedeles polycystickidneydiseasedrugdevelopmentaconferencereport |