Dive Brief:
- Akebia Therapeutics said Tuesday the Food and Drug Administration has outlined a “path forward” for the biotechnology company’s anemia pill following a rejection last year, though the regulator denied a formal appeal of its decision.
- In a written response to the company’s request for an appeal, Akebia said the FDA indicated it can resubmit an application for the drug, vadadustat, without generating new clinical data. The request would be for use in chronic kidney disease patients on dialysis, and would involve adding risks for heart or liver-related side effects to the medicine’s labeling.
- Akebia plans to meet with the regulator “as soon as possible” and file a new application in the second half of 2023, it said in a statement. The drug is approved in 33 countries but has not passed muster with the FDA, which has been hesitant to clear medicines like vadadustat due to safety concerns.
Dive Insight:
Following vadadustat’s U.S. rejection last spring, Akebia laid off 42% of its workforce and began to refocus its strategy around a different, marketed drug for kidney disease. The company lost a partnership with Otsuka Pharmaceutical involving U.S. drug rights, and shares once worth nearly $30 apiece traded at less than $1, raising the risk that Akebia might be delisted from the Nasdaq stock exchange.
But Akebia held out hope vadadustat might get a second chance in the U.S. Though the FDA had struck down vadadustat and another drug like it from FibroGen over safety concerns, it cleared a rival medicine from GSK for use in kidney disease patients on dialysis. Akebia appealed the FDA’s rejection of its medicine, and while that effort has now failed, the company sees an opportunity to bring vadadustat back to the FDA without running another trial.
“This is really the best outcome we could have hoped for,” said CEO John Butler, on a conference call with analysts on Tuesday.
The FDA rejected vadadustat because of the risk of heart problems as well as drug-induced liver damage. During the appeal process, the company gave the FDA newer analyses of the data.
According to Butler, the agency said in a written letter that the risk of clotting events is “not large” and may be managed by updating the drug’s labeling. The regulator also advised the company to include real-world data from use of vadadustat in Japan, where “tens of thousands” of patients have been treated over the last two years with no reports of drug-related liver injury, he said.
Once Akebia gets a meeting and finalizes its application, Butler expects vadadustat would undergo a six-month review. “We’re getting ready to go as quickly as we can,” he said.
Vadadustat is one of a group of medicines that were once billed as convenient and safer alternatives to widely used injectable medicines for anemia associated with chronic kidney disease. Prior to the drug’s rejection, analysts at Piper Sandler estimated the drug could reach $370 million in annual revenue in the U.S. by its second full year on the market if approved for people on dialysis.
Akebia has a distribution and profit-sharing deal in place with Vifor Pharma that could help the company access many of the dialysis patients in the U.S.
Shares climbed higher on Tuesday’s news, to about $1.20 a share by late morning.