The U.S. Food and Drug Administration (FDA) has granted approval for vadadustat (brand name Vafseo®) as a treatment for anemia resulting from chronic kidney disease (CKD) in adult patients undergoing dialysis. This approval, announced by Akebia Therapeutics on March 27, 2024, follows a rigorous evaluation process based on efficacy and safety data from the INNO2VATE program, as well as post-marketing safety data from Japan, where vadadustat has been in use since August 2020. Learn more about vadadustat/Vafseo® for anemia on dialysis.*

Early setbacks and a warning 

The drug finally earned acceptance from the FDA after encountering setbacks in 2022, including a Complete Response Letter (CRL) due to concerns over risk-benefit assessment and a 17% increased risk of major adverse cardiovascular events compared to the standard treatment, darbepoetin alfa. 

The manufacturer resubmitted its application with additional safety data from more than 10,000 vadadustat users in Japan, and after a rigorous six-month review the drug was approved.

The approval of vadadustat, however, includes a Boxed Warning highlighting potential risks such as:

• Increased mortality, 
• Myocardial infarction, 
• Stroke,  
• Venous thromboembolism, and 
• Thrombosis of vascular access. 

How vadadustat works

Anemia, the condition of having too few red blood cells carrying oxygen throughout the body, causes a strain on the heart because it has to pump more blood than normal. This can lead to an enlarged heart and/or heart failure. Anemia also typically causes fatigue and other health complications. Medication and iron supplements are a common treatment for anemia.

Vafseo® is a once-daily oral medication that operates by mimicking the physiological effects of altitude, enhancing red blood cell production and oxygen delivery to tissues.

Akebia Therapeutics’ CEO, John P. Butler, emphasized the significance of this approval as an alternative treatment for the sizable population (about 500,000), of Americans on dialysis who experience anemia due to CKD. 

Vafseo® has already been approved in 37 countries.

*Akebia Therapeutics. (2024, March 27). Akebia Receives FDA Approval of Vafseo® (vadadustat) Tablets for the Treatment of Anemia due to Chronic Kidney Disease in Adult Patients on Dialysis [Press Release]. https://ir.akebia.com/news-releases/news-release-details/akebia-receives-fda-approval-vafseor-vadadustat-tablets  

Responsum Health closely vets all sources to ensure that we always provide you with high-quality, reliable information. We do not, however, endorse or recommend any specific providers, treatments, or products, and the use of a given source does not imply an endorsement of any provider, treatment, medication, or procedure discussed within.