FDA approves Akebia anemia pill, two years after rejection

2024-03-28

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Dive Brief:

The Food and Drug Administration has approved Akebia Therapeutics’ blood-boosting pill Vafseo for people with anemia from chronic kidney disease, the company said Wednesday, reversing a rejection the agency made two years ago over the drug’s safety.

The decision came after a review of safety data from Japan, where Vafseo has been used since 2020, in addition to clinical trials Akebia completed before its initial application. Last year, the regulator approved a similarly acting pill called Jesduvroq from GSK with extensive safety restrictions and warnings.

Vafseo can only be used in kidney disease patients who have been on dialysis at least three months. The label includes a “black box” warning about the risk of cardiovascular complications from blood clots, stating there is no dose, dosing protocol or level of hemoglobin that doesn’t increase the risk.

Dive Insight:

Drug developers have studied pills like Jesduvroq and Vafseo as alternatives to injectable biologic drugs like Amgen’s Epogen in the hopes they would be safer and more convenient. Rather than mimicking a natural blood-boosting protein called erythropoietin as the biologics do, Jesduvroq and Vafseo trick the body into responding as if it were in a high-altitude environment by blocking a protein called HIF-PH. That, in turn, stimulates red blood cell production.

But the FDA has viewed the pills as no safer than biologics, stating in the black box warning that targeting a hemoglobin level above 11 grams per deciliter with Vafseo adds similar risk as with the biologics. Normal hemoglobin levels are 12 to 16 for women and 14 to 18 for men.

Doctors using Vafseo should use a low dose that can reduce the need for blood transfusions, the FDA said.

The FDA’s initial rejection of Vafseo prompted Akebia to lay off 42% of its workers in a restructuring that reoriented the company around marketing its approved drug Auryxia, an iron replacement and phosphorus control agent for people with anemia from chronic kidney disease.

Akebia got a lifeline when the FDA approved Jesduvroq and then accepted a resubmission for Vafseo, known scientifically as vadadustat. With the Jesduvroq approval and the Vafseo resubmission, the agency only considered use for patients on dialysis, as recommended by FDA’s outside experts, and not those patients whose disease hadn’t progressed enough to need dialysis.

The outlook for these drugs in only dialysis patients is unclear. GSK reported sales of only 26 million pounds, or about $33 million, for Jesduvroq in its annual report for 2023.

Akebia shares fell by double digits in morning trading Thursday.

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