Abstract: INFO04-TH
US Food and Drug Administration (FDA)-Qualified Kidney Safety Biomarker Context of Use: An Anchor for Future Biomarker Qualification
Session Information
- Informational Posters - 1
October 22, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Acute Kidney Injury
- No subcategory defined
Authors
- Bonventre, Joseph V., Brigham and Women's Hospital, Boston, Massachusetts, United States
- Murray, Patrick T., University College Dublin, Dublin, Leinster, Ireland
- Radhakrishnan, Jai, Columbia University, New York, New York, United States
- Roberts, Glenda V., Icahn School of Medicine at Mount Sinai, New York, New York, United States
- King, Nicholas M., Critical Path Institute, Tucson, Arizona, United States
- Peron, Katrina Jolene, Critical Path Institute, Tucson, Arizona, United States
- Poalillo, Andrew, Critical Path Institute, Tucson, Arizona, United States
- Sultana, Stefan, Critical Path Institute, Tucson, Arizona, United States
- Hoffmann, Steven C., Foundation for the National Institutes of Health, North Bethesda, Maryland, United States
- Friedman, Gary Steven, Critical Path Institute, Tucson, Arizona, United States
Description
In 2018, FDA qualified a kidney safety biomarker context of use (COU) for combined use of standard biomarkers (SBM; serum creatinine, eGFR) and emerging novel urine biomarkers (NBM) for Phase 1 study cohorts. Subsequently, pharmaceutical early clinical development programs have used the resulting composite measure (PFC Index) to support drug development go/no-go decisions through more sensitive detection of nephrotoxicity (Zabka et al., 2025).
In May 2026, C-Path submitted to FDA a Full Qualification Package (FQP) seeking qualification of a COU for quantitative assessment of nephrotoxicant exposure in individual study participants. To support current and future biomarker qualifications, C-Path has established a Biomarker Data Repository (BmDR) resource of de-identified, patient-level datasets (baseline and post-baseline values) identified from pharmaceutical companies, ClinicalTrials.gov, the EU Clinical Trials Registry, and published literature. BmDR de-identified patient-level datasets with contemporaneous SBM-NBM values (baseline and post-baseline) can be accessed by qualified researchers through the Data Analytics Platform (DAP) using CDISC-compliant metadata structures that facilitate pooled analyses.
With over 2,000 study participant data values, BmDR provides a foundation to develop future biomarker COUs. Potential analyses include: (1) hierarchical evaluation of biomarker panel performance using subsets of qualified biomarkers; (2) pediatric versus adult analyses across the age spectrum; (3) demographic subgroup analyses; (4) CKD stage- and baseline eGFR-stratified analyses; (5) evaluation across kidney injury and kidney disease populations; and (6) population pharmacokinetics and quantitative biomarker modeling to support in vitro diagnostic development and precision medicine applications.
BmDR can accelerate the pathway from biomarker discovery to qualification by leveraging regulatory-submission grade datasets, standardized metadata, and prior FDA qualification experience. In addition to establishing utility of current biomarkers, these resources may support development of new kidney safety and disease biomarkers that provide more sensitive detection and/or localization of ongoing injury, injury resolution, and ultimately enhanced nephrology patient care.
Funding
- Other U.S. Government Support (U.S. Food and Drug Administration)