The biopharma Akebia Therapeutics (Nasdaq:AKBA) has received a dreaded complete response letter from FDA for vadadustat, the experimental oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor proposed as a treatment for anemia associated with chronic kidney disease (CKD).
A complete response letter communicates the agency’s rejection of an application in its present form. In the case of vadadustat, the FDA rejected the drug candidate due to safety concerns. Specifically, the agency decided the new drug application (NDA) for vadadustat did not support a favorable benefit-risk assessment for dialysis and non-dialysis patients.
In particular, the FDA was concerned about the drug candidate’s potential association with major adverse cardiovascular events (MACE), vascular access thrombosis in dialysis patients and drug-induced liver injury.
Akebia submitted an NDA for vadadustat for anemia associated with CKD in March 2021. The company announced that FDA had accepted the application in June.
AKBA shares fell 14.04% in mid-day trading to $0.71.
FDA recommended strategies Akebia could pursue in new clinical trials to show a beneficial benefit-risk assessment.
“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, Akebia CEO, in a statement. “Despite this setback, we continue to work toward our purpose to better the lives of people impacted by kidney disease.”
Akebia developed vadadustat in collaboration with Otsuka Pharmaceutical Co. Otsuka is pursuing authorization for the drug candidate in the European Union.
Vadadustat is already approved in Japan as a treatment for anemia due to CKD regardless of dialysis dependence.
Filed Under: clinical trials, Drug Discovery, Hematology, Nephrology/urology