- After seeing two competitors turned back by the Food and Drug Administration, GSK on Wednesday secured the agency’s approval for the first pill to treat anemia in patients with chronic kidney disease.
- The approval, while a welcome win for GSK, comes with notable limitations. The FDA only cleared use of the drug, which will be sold as Jesduvroq, for patients who are on dialysis, excluding those who don’t yet need the blood-filtering treatment. In that group, the FDA said Jesduvroq’s safety is not established.
- Jesduvroq will also carry a black box warning on its labeling, citing the increased risk of blood clotting that can lead to death, heart attack and stroke. The treatment’s safety was a point of debate at an advisory committee meeting the FDA held last October, when advisers backed Jesduvroq’s use only in patients on dialysis.
Jesduvroq belongs to a new class of oral treatments that GSK, along with rivals FibroGen and Akebia Therapeutics, saw as more convenient alternatives to the injectable medicines that are currently standard for treating kidney disease-related anemia. But in August 2021 and then again in March last year, the FDA rejected Fibrogen's and Akebia’s therapies over safety concerns, dimming the drug class’ potential.
The drugs, called HIF-PH inhibitors, work by tricking the body into responding as if it is in a high-altitude environment, spurring production of a hormone called erythropoietin. Higher erythropoietin production leads to increased levels of red blood cells, treating patients’ anemia.
Approval of Jesduvroq in patients on dialysis was based on a large GSK study called ASCEND-D that found it comparable to existing drugs known as erythropoiesis-stimulating agents.
“With an oral drug option in addition to the FDA-approved injection options, adults with chronic kidney disease on dialysis now have multiple ways to treat their anemia,” said Ann Farrell, head of the FDA’s division of non-malignant hematology, in an agency statement.
In their review, FDA staff determined that Jesduvroq treatment did not “unacceptably increase the risk” of heart complications when used in patients on dialysis, but noted greater risks in patients who are not. At the October advisory meeting, the panel of independent experts voted 13-3 that Jesduvroq’s benefits outweighed its risks in the former population, and 11-5 against the medicine in the latter group.
GSK in June 2020 won approval of the drug in Japan, where it’s sold as Duvroq, and is awaiting a decision from the European Medicines Agency this year.
The company hasn’t yet set a price in the U.S. and is currently submitting documentation to the Centers for Medicare & Medicaid Services, as 87% of dialysis patients are on Medicare, a company spokesperson said.