ProKidney Secures FDA Alignment on Accelerated Approval Pathway for Rilparencel in Chronic Kidney Disease
核心洞察
ProKidney Corp (搜索). has secured FDA alignment on the accelerated approval pathway for rilparencel, with eGFR (搜索) slope confirmed as an acceptable surrogate endpoint for patients with chronic kidney disease and type 2 diabetes.
The FDA agreed that a rilparencel effect size of at least 1.5 mL/min/1.73m²/year improvement versus sham controls would demonstrate acceptable efficacy in the accelerated approval setting.
The ongoing Phase 3 PROACT 1 study can serve both for accelerated approval and as the confirmatory study for full approval, with topline data for accelerated approval anticipated in Q2 2027.
ProKidney Corp (搜索). has achieved a significant regulatory milestone with FDA confirmation of alignment on the accelerated approval pathway for rilparencel, an autologous cellular therapy for chronic kidney disease (CKD) patients with type 2 diabetes. The FDA confirmed that the slope of estimated glomerular filtration rate (eGFR (搜索)) can serve as an acceptable surrogate endpoint for accelerated approval in this patient population.
FDA Confirms Regulatory Framework
In a recent Type B meeting, the FDA established clear parameters for rilparencel's accelerated approval pathway. The agency confirmed that eGFR (搜索) slope from the ongoing Phase 3 PROACT 1 study can serve as both the surrogate endpoint and primary basis for a Biologics License Application (BLA) submission under the accelerated approval pathway.
The FDA agreed that a rilparencel effect size of at least 1.5 mL/min/1.73m²/year improvement versus sham controls would constitute an acceptable demonstration of efficacy in patients receiving appropriate standard of care therapies. This threshold provides ProKidney with a clear regulatory target for demonstrating clinical benefit.
Dual-Purpose Clinical Trial Design
The FDA also confirmed that the ongoing Phase 3 PROACT 1 study can serve dual purposes: supporting both accelerated approval based on the eGFR (搜索) slope surrogate endpoint and serving as the confirmatory study for full approval based on the primary time-to-event composite endpoint specified in the protocol.
ProKidney anticipates topline data readout of eGFR (搜索) slope to support an application for accelerated approval in Q2 2027. The company has enrolled nearly half of the patients required for the accelerated approval analysis. Updated guidance on the expected timing of the confirmatory readout will be provided in the first half of 2026.
Addressing Significant Unmet Medical Need
The regulatory alignment comes as ProKidney targets a substantial patient population with high unmet clinical needs. An estimated 37 million adults in the U.S. have CKD, though many remain undiagnosed in the early stages. Diabetes is the leading cause of CKD, and individuals with both conditions face significantly elevated risks of cardiovascular events, hospitalization, and mortality.
ProKidney is developing rilparencel specifically for patients with Stage 3b/4 CKD and diabetes, a population that includes 1 to 2 million people in the U.S. While current treatment options aim to slow disease progression, there remains a substantial unmet need for therapies that can stabilize kidney function and delay or prevent the need for dialysis in patients with advanced CKD.
Phase 3 PROACT 1 Trial Details
The REGEN-006 (PROACT 1) trial is an ongoing Phase 3, randomized, blinded, sham-controlled safety and efficacy study of rilparencel in subjects with advanced CKD and type 2 diabetes. The study protocol was amended in the first half of 2024 to focus on a subset of patients with Stage 4 CKD (eGFR (搜索) 20-30 mL/min/1.73m²) and late Stage 3b CKD (eGFR 30-35 mL/min/1.73m²) with accompanying albuminuria.
The total planned enrollment is approximately 685 subjects, randomized 1:1 to the treatment group and sham control group prior to kidney biopsy or sham biopsy procedure, respectively. The primary objective is to assess the efficacy of up to two rilparencel injections (one in each kidney) using a minimally invasive percutaneous approach.
The primary composite endpoint for full approval is the time from first injection to the earliest of: at least 40% reduction in eGFR (搜索); eGFR <15 mL/min/1.73m²; chronic dialysis; renal transplant; or renal or cardiovascular death.
Company Leadership Perspective
"We are very encouraged by the FDA's support of the accelerated approval pathway for rilparencel using eGFR (搜索) slope, marking a critical regulatory milestone for ProKidney," said Bruce Culleton, M.D., CEO of ProKidney. "This alignment allows us to move forward with clarity and conviction as we advance enrollment in our pivotal Phase 3 PROACT 1 study on the heels of the positive Phase 2 REGEN-007 topline results announced last week."
Rilparencel, also known as REACT®, is a first-in-class, patented, proprietary autologous cellular therapy that has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. The therapy is being evaluated for its potential to preserve kidney function in diabetic patients at high risk of kidney failure.