FDA Approves Vafseo for Anemia in Kidney Disease

The FDA has approved Vafseo (vadadustat) tablets to treat anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. About 500,000 adult patients in the United States on dialysis suffer from anemia due to chronic kidney disease. Anemia is often treated with injectable erythropoiesis-stimulating agents mostly administered at dialysis centers.

Developed by Akebia Therapeutics, Vafseo is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor. It was designed to mimic the physiologic effect of altitude on oxygen availability. At higher altitudes, the body responds to lower oxygen availability with stabilization of hypoxia-inducible factor, which can lead to increased red blood cell production and improved oxygen delivery to tissues.

Nicholas Grund

Vafseo will be available in January 2025. The price has not been released, but the price will align with its value, Nicholas Grund, senior vice president and chief commercial officer, said during an investor call.

Most patients on dialysis are in Medicare and Medicaid plans that are reimbursed through bundled payments. About 40% of patients who could be treated with Vafseo have coverage through Medicare Advantage plans.

The company plans to apply for Transitional Drug Add-on Payment Adjustment (TDAPA), which CMS has established to support access to new therapies for patients with end-stage renal disease who are on dialysis. TDAPA was established in 2016 to provide payment for new therapies into the bundled reimbursement.

Once available, Grund said they expect rapid adoption of Vafseo.

The approval of Vafseo is based on efficacy and safety data from the INNO2VATE program and an assessment of postmarketing safety data from Japan where Vafsep was launched in August 2020. Results from INNO2VATE program were published in papers in The New England Journal of Medicine in April 2021. (Here and here.)

The label for Vafseo includes a boxed warning about the increased risk of death, myocardial infarction, stroke, venous thromboembolism and thrombosis of vascular access.

Akebia had first submitted an application to the FDA for vadadustat in March 2021. The agency, however, had issued a complete response letter in which regulators questioned the benefit-risk assessment of Vafseo for dialysis and non-dialysis patients. The FDA expressed concerns about safety, noting that the data submitted in the NDA did not show non-inferiority in major adverse cardiovascular events (MACE) in the non-dialysis patient population. They also pointed to the increased risk of thromboembolic events and the risk of drug-induced liver injury.

Akebia resubmitted the application in October 2023 with postmarketing safety data from patients in Japan where vadadustat is approved and has been in market for more than three years. In Japan, it is marketed by Mitsubishi Tanabe Pharma also as Vafseo.

Akebia will partner with CSL Vifor to market Vafseo in the United States.