- Akebia Therapeutics is taking a chance on its closely watched pill for anemia, announcing Tuesday the submission of an application for approval to the Food and Drug Administration despite mixed study data disclosed last fall that lowered expectations for the drug.
- In two Phase 3 studies, Akebia's experimental drug matched the approved injection Aranesp in raising and sustaining hemoglobin levels among trial participants with anemia due to their chronic kidney disease. But the pill, called vadadustat, appeared worse than Aranesp on a measure of heart safety, with a greater risk of major adverse cardiovascular events.
- Akebia said then the company still intended to ask the FDA for approval, citing the totality of the data from those and other studies. But analysts who follow the company were more skeptical the drug could obtain a clearance for a broad patient population that includes both dialysis and non-dialysis patients.
For several years now, Akebia has been chasing rival anemia drug developer Fibrogen, which appeared to be ahead in its development of a similar acting treatment and would-be competitor to vadadustat.
Fibrogen applied for FDA approval last year, only to have a potential December decision delayed until March. Then, at the beginning of the month, the company and its investors were taken by surprise when the agency indicated it would call an advisory committee meeting to review Fibrogen's drug.
At least twice before, Fibrogen claimed, the FDA had indicated it wouldn't convene a panel, making the last-minute communication an unexpected and potentially costly twist. No date has yet been set, and the FDA has now missed its March 20 goal. The company has not issued any update.
Now that Akebia has submitted its application, the FDA may seek to convene an advisory committee meeting covering both drugs, wrote Christopher Raymond, an analyst at Piper Sandler, in a note to clients.
Both drugs are designed to replace current injectable therapies with a more convenient pill option. They are known as HIF-PH inhibitors and work by promoting red blood cell production in a way that mimics the body's response to low-oxygen environments.
Studies have shown both pills can match the efficacy of injectable drugs. But their safety, particularly for the heart, has become a key concern, especially following Akebia's results last year. Those findings could make the approval case for both drugs harder to regulators, given both are meant to be at least as safe, if not safer, than the injectable medicines like Aranesp that are already tied to potential heart problems.
Akebia's submission came one to two months ahead of expectations, and the company indicated it did not include in its filing a priority review voucher. Such vouchers are a kind of a regulatory fast-pass and can be used to shorten FDA review times by four months.
In early 2020, Akebia had struck a deal with partner Vifor Pharma to hold onto a voucher Vifor bought for about $100 million. The agreement specified the companies would either use it on vadadustat or resell it and share the proceeds.
But the delay facing rival Fibrogen, and resulting uncertainty on the FDA's view of HIF-PH blockers, may have weakened the case for seeking a speedier review. Once a rare commodity, vouchers are now more common but can still fetch around $100 million in a sale.
The FDA now has 60 days to determine whether Akebia's application is complete and ready for an official review.