- FDA confirms eGFR slope as a surrogate endpoint for accelerated approval of ProKidney's rilparencel.
- The Phase 3 PROACT 1 study will serve dual purposes for both accelerated and full approval.
- Nearly 50% of required patients are enrolled, with topline data expected in Q2 2027.
ProKidney Corp. (PROK, Financial) has announced a major milestone in its pursuit of regulatory approval for rilparencel, its innovative autologous cellular therapy aimed at treating chronic kidney disease (CKD) in patients with type 2 diabetes. The U.S. Food and Drug Administration (FDA) has confirmed that the estimated glomerular filtration rate (eGFR) slope from the ongoing Phase 3 PROACT 1 trial can be used as a surrogate endpoint for accelerated approval.
The FDA has agreed that an improvement in the rilparencel effect size of at least 1.5 mL/min/1.73m²/year compared to controls is an acceptable measure of efficacy. This agreement provides a clear and measurable pathway for ProKidney's Phase 3 PROACT 1 trial, with topline data to support accelerated approval anticipated in the second quarter of 2027.
As of now, ProKidney has enrolled nearly half of the necessary patients for this analysis, marking significant progress towards meeting the trial's objectives. Additionally, the FDA has confirmed that the ongoing Phase 3 PROACT 1 study can also serve as a confirmatory study for full approval, ensuring a streamlined pathway from accelerated to full market authorization.
This development follows the FDA's grant of the Regenerative Medicine Advanced Therapy (RMAT) designation to rilparencel, which acknowledges the potential of this therapy to meet serious unmet medical needs for patients with CKD compounded by diabetes. The RMAT designation enhances the interaction with the FDA, facilitating a clearer regulatory path.
ProKidney's CEO, Dr. Bruce Culleton, expressed optimism about the FDA's support, stating, "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 positive Phase 2 results." The ongoing dialogue with the FDA under the RMAT designation is set to expedite the process of making rilparencel available to a patient population in dire need of effective treatment options.
About Chronic Kidney Disease: CKD is a progressive disease affecting millions of adults in the U.S., often leading to end-stage kidney disease. Diabetes is the primary cause of CKD, further elevating the risk of severe health complications. ProKidney's rilparencel is targeted at patients with advanced stages of the disease, where current therapies are inadequate in slowing progression.
