TUCSON, Ariz.--(BUSINESS WIRE)--The Critical Path Institute (C-Path) announced today that the European Medicines Agency (EMA) rendered a positive qualification opinion to C-Path’s Polycystic Kidney Disease Outcomes Consortium (PKDOC) for total kidney volume (TKV) as a prognostic biomarker to select patients for clinical trials of new therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD).
ADPKD affects approximately 12 million people worldwide. There is only one medication developed to treat ADPKD, called tolvaptan, which has not yet been approved in the U.S.
TKV is a measurement of the impact of ADPKD on kidney size and is considered to be predictive of decline in kidney function. The EMA opinion states, “CHMP supports baseline total kidney volume, in combination with patient age and eGFR as a prognostic biomarker to identify patients likely to experience a progressive decline in renal function, as characterized by a decline in eGFR or progression to end-stage renal disease.
“From the data provided it is reasonable to expect that baseline TKV can predict disease progression and is a biomarker valuable for risk stratification."
”This qualification, along with a similar one from FDA, confirms the relationship between TKV and ADPKD disease progression, and will help in the design of clinical trials for new therapies for ADPKD” says C-Path COO and PKDOC Co-Director Steve Broadbent.
The PKDOC created a Clinical Data Interchange Standards Consortium (CDISC) data standard for ADPKD and used it to remap data from patient registries and observational studies. The database was used to develop a joint model linking baseline TKV with clinical outcomes.
About the organizations:
C-Path, a nonprofit, was established in 2005 to deliver on FDA’s Critical Path Initiative. C-Path has established 12 global, public-private partnerships that include regulatory agencies, academia, patient advocacy organizations, and pharmaceutical companies. C-Path has headquarters in Tucson, AZ, and has been funded by public and private support. For information, visit www.c-path.org.
The PKD Foundation is the only US organization solely dedicated to finding treatments and a cure for PKD, through promoting programs of research, education, advocacy, support, and awareness on a national level, along with services in local communities. Visit pkdcure.org to learn more.
