ProKidney Corp has announced the confirmation of alignment with the US Food and Drug Administration (FDA) on the accelerated approval pathway for rilparencel.
Rilparencel is an autologous cellular therapy that received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA and currently is being evaluated in the ongoing Phase 3 REGEN-006 (PROACT 1) trial to demonstrate the therapy’s potential to preserve kidney function in patients with advanced chronic kidney disease (CKD) and type 2 diabetes.
In a recent Type B meeting, the FDA confirmed that the slope of estimated glomerular filtration rate (eGFR) in patients from the ongoing Phase 3 PROACT 1 study can serve as the surrogate endpoint and primary basis for a Biologics License Application (BLA) submission of rilparencel under the accelerated approval pathway. The FDA agreed that a rilparencel effect size (versus sham controls) of at least 1.5 mL/min/1.73m2/year improvement would be an acceptable demonstration of efficacy in the setting of patients receiving appropriate standard of care therapies.
ProKidney anticipates topline data readout of eGFR slope as the surrogate endpoint to support an application for accelerated approval in Q2 2027. To date, ProKidney has enrolled nearly half of the patients required for the accelerated approval analysis.
The FDA also confirmed that the ongoing Phase 3 PROACT 1 study may serve as the confirmatory study to support full approval of rilparencel based on the primary time-to-event composite endpoint specified in the protocol. Updated guidance on the expected timing of the confirmatory readout will be provided in 1H 2026.
“We are very encouraged by the FDA’s support of the accelerated approval pathway for rilparencel using eGFR slope, marking a critical regulatory milestone for ProKidney,” said Bruce Culleton, chief executive officer of ProKidney. “This alignment allows us to move forward with clarity and conviction as we advance enrolment in our pivotal Phase 3 PROACT 1 study on the heels of the positive Phase 2 REGEN-007 topline results announced last week. We look forward to our ongoing dialogue with the FDA, under RMAT designation, to bring rilparencel to patients with advanced CKD and diabetes, a patient population with high unmet clinical and societal needs.”
