Qualification of Biomarker-Total Kidney Volume in Studies for Treatment of Autosomal Dominant Polycystic Kidney Disease; Guidance for Industry; Availability

Zusammenfassung

The Food and Drug Administration (FDA or Agency) is announcing the availability of a guidance for industry entitled "Qualification of Biomarker--Total Kidney Volume in Studies for Treatment of Autosomal Dominant Polycystic Kidney Disease." This guidance provides a qualified context of use (COU) for total kidney volume (TKV), measured at baseline, to be used as a prognostic enrichment biomarker to select patients with autosomal dominant polycystic kidney disease (ADPKD) at high risk for a "progressive decline" in renal function, defined as a confirmed 30 percent decline in the patient's estimated glomerular filtration rate (eGFR), for inclusion in interventional clinical trials. This guidance also describes the experimental conditions and constraints for which this biomarker is qualified through the Center for Drug Evaluation and Research (CDER) Biomarker Qualification Program. This biomarker can be used by drug developers for the qualified COU in submissions of investigational new drug applications (INDs), new drug applications (NDAs), and biologics license applications (BLAs)without the relevant CDER review group reconsidering and reconfirming the suitability of the biomarker.