Akebia's Vafseo Launches in US, Plans Expansion into Non-Dialysis CKD Patients

Akebia Therapeutics has initiated the US launch of its chronic kidney disease (CKD) anemia drug Vafseo, while simultaneously unveiling plans to broaden the therapy's reach through a new phase 3 clinical trial. This strategic move comes as the company aims to capitalize on a potentially lucrative market opportunity in non-dialysis CKD patients.

Vafseo's US Debut and Initial Market Performance

Vafseo, a hypoxia-inducible factor–prolyl hydroxylase inhibitor (HIF-PHI), received FDA approval in March 2024 for the treatment of CKD-associated anemia in patients who have been on dialysis for at least three months. The drug's US market launch began on January 9, 2025, with Akebia projecting first-quarter sales of $10 million to $11 million.

Early adoption indicators are promising, with three of the nation's top four dialysis organizations placing orders for Vafseo. By the end of February, more than 500 prescribers had written prescriptions for the drug, signaling growing acceptance among healthcare providers.

Expansion Plans: The Valor Trial

In a bid to expand Vafseo's market reach, Akebia has announced plans for a new phase 3 clinical trial named Valor. This study aims to evaluate Vafseo as a potential treatment for anemia in late-stage CKD patients who are not on dialysis.

The Valor trial is designed to recruit approximately 1,500 US patients with stage 4 or 5 CKD not undergoing dialysis. Akebia has submitted a trial protocol to the FDA and anticipates discussions with the regulator regarding study design. If approved, the company aims to initiate the trial in the second half of 2025.

This expansion strategy targets a significant patient population, with Akebia estimating around 550,000 anemic patients with stage 4 or 5 CKD who are not receiving dialysis. The company notes that only 25% of these patients are currently treated for anemia, presenting a substantial market opportunity.

Market Dynamics and Strategic Considerations

Akebia's push into the non-dialysis CKD patient segment is driven by several factors:

1. Declining use of erythropoietin stimulating agents (ESAs) in non-dialysis patients
2. Potential advantages of oral HIF-PH inhibitors over existing treatments
3. Physician interest in HIF-PH inhibitors for non-dialysis CKD patients

The company cites a Spherix Global Insights report indicating that 78% of doctors would increase anemia treatment for non-dialysis CKD patients if an HIF-PH inhibitor were approved.

Akebia's CEO, John Butler, has described the potential label expansion as a "multi-billion-dollar US market opportunity." The company's pricing strategy for Vafseo, which was set higher than competitor GSK's Jesduvroq, takes into account the possibility of future label expansions under the constraints of the Inflation Reduction Act.

As Akebia advances its plans for Vafseo, the pharmaceutical industry will be closely watching the outcome of the Valor trial and its potential impact on the CKD anemia treatment landscape.