Akebia Therapeutics announced that the FDA has approved Vafseo (vadadustat) for treating anemia caused by chronic kidney disease (CKD) in adults on dialysis for at least three months.
Vafseo, a once-daily oral medication, functions as a hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor. This class of drug stimulates the body's response to low oxygen levels, enhancing the natural production of erythropoietin, a hormone essential for managing anemia. The drug is now authorized in 37 countries.
In the U.S., an estimated 500,000 adults undergoing dialysis are affected by anemia due to CKD. Traditionally, most patients with CKD-related anemia are treated with injectable erythropoiesis-stimulating agents, which are often administered at dialysis centers. Vafseo offers an oral alternative that could help manage hemoglobin levels within the target ranges recommended by guidelines.
The FDA first rejected vadadustat in 2022, issuing a complete response letter citing insufficient data for a favorable benefit-risk balance and safety concerns, including thromboembolic risks and potential liver injury. Despite the setback, Akebia, expressing belief in vadadustat's potential, planned to seek further FDA dialogue. Meanwhile, the drug's review continued in Europe, and it had already been approved in Japan for CKD-related anemia in adults.
A year later, the FDA issued a second CRL, this time citing that due to agency resource and staffing constraints the deciding authority for the appeal will now be Peter Stein, Director of the Office of New Drugs. At the time, Dr. Stein indicated the need to seek internal consultation with nephrology, cardiology and liver safety experts in the OND before rendering a decision.
Back in 2016, Akebia and Japan's Otsuka Pharmaceutical formed a collaboration and license agreement to fund vadadustat. Otsuka pledged $265 million to support the late-stage trials of vadadustat, including an upfront payment of $125 million and approximately $35 million in early 2017. As part of the deal, Akebia agreed to split both the costs and profits of vadadustat in the U.S. with Otsuka.
Following the issuing of the first CRL in 2022, Otsuka decided to terminate its global license agreements with Akebia Therapeutics for vadadustat for alleged "material breaches."
