Core Viewpoint - Akebia Therapeutics does not plan to initiate the VALOR clinical trial for vadadustat to treat anemia in patients with late-stage chronic kidney disease (CKD) not on dialysis, following feedback from the U.S. FDA indicating a need for a larger patient population and increased time and costs for the trial [1][2][3] Company Overview - Akebia Therapeutics, Inc. is a biopharmaceutical company focused on improving the lives of individuals affected by kidney disease, founded in 2007 and headquartered in Cambridge, Massachusetts [4] Product Information - Vafseo® (vadadustat) is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor approved for treating anemia due to CKD in adults who have been on dialysis for at least three months, with approval in 37 countries [5] Regulatory Feedback - The FDA's feedback from the Type C meeting suggests that regulatory alignment for the VALOR trial would necessitate a significantly larger patient cohort than initially proposed, leading to increased time and costs [2][3] Future Directions - Despite the setback with the VALOR trial, the company remains committed to addressing the unmet need for oral anemia treatment in CKD patients not on dialysis and is exploring potential clinical trial designs for smaller subgroups of CKD patients [3]

Core Insights - Akebia Therapeutics has initiated a post-marketing study, the VOCAL trial, to evaluate the efficacy and safety of Vafseo (vadadustat) in patients with anemia due to chronic kidney disease (CKD) undergoing hemodialysis [1][2] - The trial aims to compare the three times per week dosing of Vafseo against standard erythropoiesis-stimulating agents (ESA) [1][2] - Vafseo was approved by the FDA in March 2024 and became available in the U.S. in January 2025 for treating anemia in adults on dialysis for at least three months [4] Study Design and Objectives - The VOCAL trial will enroll approximately 350 patients across 18 DaVita hemodialysis clinics, utilizing a 1:1 randomization method [2] - The study duration will be up to 33 weeks, including screening, treatment, and safety follow-up [2] - Primary endpoint is the change in hemoglobin levels, with secondary endpoints including serious adverse events, target hemoglobin range, and RBC transfusion rates compared to ESA treatment [2] Sub-Study and Research Focus - A sub-study will involve around 28 patients from three clinics, focusing on the impact of Vafseo on red blood cell (RBC) quality [3] - The sub-study aims to analyze RBC phenotypes, including deformability and resistance to oxidative stress, in collaboration with Vitalant Research Institute [3] Company Overview - Akebia Therapeutics is a biopharmaceutical company dedicated to improving the lives of individuals affected by kidney disease, founded in 2007 and headquartered in Cambridge, Massachusetts [5] - Vafseo is a once-daily oral medication that stimulates endogenous erythropoietin production to manage anemia [6] Regulatory and Safety Information - Vafseo is indicated for treating anemia due to CKD in adults on dialysis for at least three months, but it has not been shown to improve quality of life or fatigue [7] - The drug carries warnings for increased risks of death, myocardial infarction, stroke, and thrombotic events [8][10]