Japanese PMDA Announces First Biomarker Qualification

The unprecedented sharing of data by the industry has facilitated regulatory agencies to receive, review and approve new methods as qualified for use in drug development. The use of these new tests can now be used in laboratory research to predict the safety of experimental drugs, allowing drugs to reach the market faster and with greater confidence in their safety.

Kidney Biomarker Submissions

Following several years of extensive work and collaboration between 16 member companies & the Critical Path Institute’s PSTC leadership, the FDA (US Food and Drug Administration), EMA (European Medicines Agency) and PMDA (the Japanese Pharmaceuticals and Medical Devices Agency) have each qualified seven novel laboratory tests on urine which signal kidney injury. A first for both agencies, the FDA and EMA jointly announced qualification in May 2008. In May 2010, PMDA announced the first ever biomarker qualification decision under PMDA’s new consultation process on pharmacogenomics/biomarkers for use in Japan. The biomarkers were evaluated in data from rat studies submitted to the regulatory agencies by the PSTC. The newly accepted biomarkers are KIM-1, Albumin, Total Protein, B2-Microglobulin, Cystatin C, Clusterin and Trefoil factor-3.

EMA Announcement:
First EMA-FDA joint biomarker qualification review

FDA Press Release:
FDA announces joint EMA/FDA biomarker review

C-Path Technical Summary:
PSTC’s technical summary of the biomarker review

Click below to view EMA, FDA, & PMDA’s summary approval documents.




In reviewing the kidney qualification data, the regulatory agencies (FDA, EMA and PMDA) came to the conclusion that:

    • the kidney biomarkers are acceptable in the context of non-clinical drug development for the detection of acute drug-induced kidney toxicity;

    • the kidney biomarkers provide additional and complementary information to the currently available standards;

    • the use of kidney biomarkers in clinical trials is to be considered on a case-by-case basis in order to gather further data to qualify their usefulness in monitoring drug-induced kidney toxicity in humans.

  • PSTC is actively involved in research to further qualify biomarkers for use in clinical drug development.