New drug application for treatment of anemia due to chronic kidney disease

Akebia Therapeutics®, Inc. today announced that the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) to Akebia’s New Drug Application (NDA) for vadadustat, an investigational oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor under review for the treatment of anemia due to chronic kidney disease (CKD). The FDA issues a CRL to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form.

The FDA concluded that the data in the NDA do not support a favorable benefit-risk assessment of vadadustat for dialysis and non-dialysis patients. The FDA expressed safety concerns noting failure to meet non-inferiority in MACE in the non-dialysis patient population, the increased risk of thromboembolic events, driven by vascular access thrombosis in dialysis patients, and the risk of drug-induced liver injury. The CRL stated that Akebia could explore ways to potentially demonstrate a favorable benefit-risk assessment through new clinical trials. Akebia will discuss the details of the CRL with its collaboration partners and request a meeting with the FDA.

“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 John P. Butler, Chief Executive Officer of Akebia. “Despite this setback, we continue to work toward our purpose to better the lives of people impacted by kidney disease.”

In October 2021, Akebia’s collaboration partner, Otsuka Pharmaceutical Co., Ltd. (Otsuka), submitted an initial marketing authorization application (MAA) for vadadustat to the European Medicines Agency for vadadustat, for the treatment of anemia due to CKD in adults; the review is ongoing. In Japan, vadadustat is approved as a treatment for anemia due to CKD in both dialysis-dependent and non-dialysis dependent adult patients.

Akebia will host a conference call Wednesday, March 30 at 6:00 p.m. Eastern Time to discuss the CRL and next steps.