Don Tracy, Associate Editor

Approval of Vafsen (vadadustat) based on promising results from the INNO2VATE program and additional safety data from use in Japan in adults with chronic kidney disease.Akebia Therapeutics announced that the FDA has approved Vafsen (vadadustat) for the treatment of anemia in adults with chronic kidney disease (CKD) who are on dialysis. According to a company press release, the once-daily, oral tablet works as a hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor, activating the physiologic response to hypoxia to stimulate endogenous production of erythropoietin to manage anemia. In other words, it copies the body's response to low oxygen levels.
The FDA based the approval was on positive outcomes from the INNO2VATE program and additional safety data from its use in Japan, where it was launched in August 2020. Now approved in 37 countries, Akebia stated that Vafsen has the potential to improve overall outcomes for patients struggling with CKD-related anemia on dialysis.1
"With the approval of Vafseo in the US, we're proud to deliver an alternative treatment option for the hundreds of thousands of Americans on dialysis who are diagnosed with anemia due to CKD," said John P. Butler, CEO, Akebia, in a press release. "At Akebia we are committed to kidney patients, a dedication that has driven our team to achieve this milestone. We believe this commitment uniquely positions the company to execute a successful launch designed to drive toward a potential new oral standard of care for dialysis patients. We are tremendously grateful for the patients, physicians, investigators, and site coordinators who participated in our clinical trials that led to this important approval. This milestone is the culmination of years of perseverance by Akebia employees and partners committed to bettering the lives of people impacted by kidney disease."
In March 2022, Akebia received a Complete Response Letter (CRL) from the FDA after submitting a New Drug Application (NDA) for Vafseo. At the time, the agency determined that the data didn’t properly support a positive assessment for the treatment. Additionally, the FDA suggested additional clinical trials to discover a potentially beneficial assessment for Vafsen.2
"We are extremely disappointed to receive a CRL for vadadustat, a therapy that has the potential to help patients with anemia due to CKD. We continue to believe the data are supportive of a positive benefit-risk assessment of vadadustat for patients with anemia due to CKD, particularly in dialysis patients," said Butler, in a 2022 press release. "Despite this setback, we continue to work toward our purpose to better the lives of people impacted by kidney disease."
According to UCLA Health, approximately 12% of CKD patients develop anemia. Further, it is more likely to manifest in individuals of African descent, females, and those struggling with diabetes. Overall, a half-million people in the United States on dialysis suffer from the condition.1,3
"Anemia is a debilitating condition that significantly impacts our daily lives,” said Lori Hartwell, founder, president, Renal Support Network, in the press release. “It is promising to see the introduction of innovative treatment options for people fighting anemia."