Akebia Therapeutics has scrapped plans for a trial that would have potentially led to an expansion of the patient population for its new anemia drug Vafseo, the company said.
In March 2024, the FDA endorsed the oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor for patients who have been on dialysis for at least three months and have anemia and chronic kidney disease (CKD). Akebia was working with regulators to design a study for patients with anemia and late-stage CKD who are not on dialysis.
But after meeting with the FDA, Akebia said Tuesday that it couldn't “come to alignment on a path forward” for the design of the trial.
The FDA requested a study with “a significantly larger number of patients” than the company had proposed, Akebia said, adding that it would have been too costly and take too much time to conduct the trial.
“Because we remain steadfast in our belief of the significant unmet need for an oral option to treat anemia in CKD patients not on dialysis, we are disappointed in the outcome of the meeting,” John Butler, the CEO of Akebia, said in a release.
Butler didn’t close the book on eventually expanding the patient population for Vasfeo, however, saying that the company was “encouraged by the discussion with FDA on smaller subgroups of CKD patients where we may be able to align on a potential clinical trial design and path forward.”
While Vafseo is approved in 37 countries—including Japan, where it has been on the market for five years to treat dialysis and non-dialysis patients—getting a nod in the U.S. was difficult, as the FDA rejected it in 2022 due to concerns about liver toxicity. Shortly after the setback, the Cambridge, Mass.-based company laid off 200 employees, which was 42% of its staff.
Eventually, based on safety data from Japan, Akebia won its FDA nod for Vafseo. The company launched Vafseo in the U.S. in January of this year and reported sales of $25 million in the first half.
Vafseo was not the first drug from its class approved in the indication in the U.S. In 2023, GSK earned a nod from the FDA for Jesduvroq to treat CKD patients on dialysis, but the company pulled it from the market in the U.S. in December of last year for “business reasons,” and not for safety issues.
Akebia is not solely dependent on sales of Vafseo. The company also sells Auryxia, a CKD drug that was approved in 2014 and has generated increasing sales over the last decade. In the first half of this year, Akebia reported sales of $91 million for the treatment, which addresses hyperphosphatemia and the iron deficiency that accompanies anemia.