Akebia Therapeutics Halts Plans for Vafseo Trial Expansion

Akebia Therapeutics has announced the discontinuation of its planned clinical trial aimed at expanding the patient population for its anemia drug Vafseo. This decision comes after discussions with the U.S. Food and Drug Administration (FDA) failed to reach an agreement on the study design.

FDA Approval and Current Indications

Vafseo, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor, received FDA approval in March 2024 for the treatment of anemia in chronic kidney disease (CKD) patients who have been on dialysis for at least three months. The drug is currently approved in 37 countries, including Japan, where it has been marketed for five years to treat both dialysis and non-dialysis patients.

Challenges in Trial Design

Akebia had been working with regulators to design a study for patients with anemia and late-stage CKD who are not on dialysis. However, the company cited inability to "come to alignment on a path forward" with the FDA regarding the trial design. The regulatory agency requested a study involving "a significantly larger number of patients" than Akebia had proposed, making the trial prohibitively expensive and time-consuming.

John Butler, CEO of Akebia, expressed disappointment in the outcome, stating, "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."

Future Prospects and Market Performance

Despite this setback, Akebia remains optimistic about potential opportunities to expand Vafseo's indications. Butler mentioned that discussions with the FDA had been encouraging regarding smaller subgroups of CKD patients, where alignment on a potential clinical trial design might be possible.

Vafseo was launched in the U.S. in January 2025, reporting sales of $25 million in the first half of the year. This follows a challenging period for the company, including an FDA rejection in 2022 due to liver toxicity concerns, which led to significant layoffs.

Akebia's portfolio also includes Auryxia, a CKD drug approved in 2014, which generated sales of $91 million in the first half of 2025. This diversification provides some stability for the company as it navigates the challenges with Vafseo's expansion plans.