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Akebia’s Vafseo: Japanese Postmarketing Data, Narrowed Indication Eased Liver Toxicity Worries

 
• By Sue Sutter

The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.

Drug Review Profile: Vafseo
The FDA approved Akebia's Vafseo for treatment of anemia in chronic kidney disease patients on dialysis. • Source: Shutterstock

Akebia Therapeutics, Inc. turned to Japanese postmarketing data to assuage the US Food and Drug Administration’s hepatoxicity concerns about Vafseo (vadadustat), an anemia treatment for chronic kidney disease patients.

After a complete response letter questioned the drug’s benefit and cited multiple safety concerns, most notably the risk of drug-induced liver injury (DILI), Akebia proposed a

Key Takeaways

  • The FDA’s first-cycle review of Akebia’s vadadustat resulted in a complete response letter that cited several major safety issues, including drug-induced liver injury.

  • In the NDA resubmission, Akebia narrowed the indication and included safety data from two new randomized trials and several types of postmarketing data from Japan, where the drug has been available since August 2020

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