As GSK gears up for high-stakes daprodustat panel review, FDA flags some safety concerns

As an FDA expert panel gears up to review GSK’s oral anemia prospect daprodustat, the agency released a briefing document that outlines safety concerns for consideration. This week's adcomm could be pivotal for the drug's approval chances, given safety issues scuppered rival drugs from AstraZeneca, plus Akebia and Otsuka.

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will hold a meeting Wednesday to discuss whether the benefits of the drug outweigh its risks in treating anemia from chronic kidney disease, both in patients on dialysis and those not on dialysis.

For its pre-panel briefing document (PDF), the FDA highlighted the drug's performance in several clinical trials, including two late-stage studies in patients on dialysis and those who are not. The trials compared the drug with approved erythropoiesis-stimulating agents (ESAs).

In the trials, daprodustat increased hemoglobin levels to a "similar extent” as the approved ESAs. The FDA reviewers didn't flag any other benefits such as patients’ feelings, function, or survival rate, but they did see safety risks.

In the dialysis-dependent patient group, risks included hospitalization for heart failure and bleeding gastric erosions. While the drug did not “unacceptably increase” the risk of major adverse cardiac events, the same can’t be said for the non-dialysis-dependent population, the FDA staff said.

In that patient group, the drug appears to have “several other” risks on top of the risks of heart failure and bleeding gastric erosions. Those included "elevated estimated" risks for heart attack and stroke, among other potential complications. Since approved ESAs already carry some of the risks, a further risk increase is “concerning," the reviewers said.

GSK is confident about its trial data, a company spokesperson told Fierce Pharma in an emailed statement.

“We believe the innovative science behind daprodustat offers not only the benefit of a positive efficacy and safety profile, but also the convenience of an oral option with flexible dosing for patients living with anemia of chronic kidney disease,” the spokesperson said, adding that the company looks forward to this week's dialogue with the FDA.

The advisory committee experts will evaluate if the drug's benefits outweigh its risks. The FDA doesn't have to follow their recommendation.

The FDA is due to make an approval decision on GSK's drug by Feb. 1, 2023. Outside the U.S., daprodustat is also in the running for marketing authorization in Europe. The med boasts a green light in Japan, where it’s approved as Duvroq to treat patients with renal anemia.

If daprodustat wins FDA approval, it will be the first oral treatment available for the treatment of anemia from CKD in the U.S., plus the first HIF prolyl hydroxylase inhibitor to be brought to the market. Already, two other medicines in the class—Akebia and Otsuka’s vadadustat and AstraZeneca and Fibrogen’s roxadustat—have suffered FDA rejections.

CKD is estimated to impact 15% of U.S. adults. Currently available treatments for anemia of CKD include iron, ESAs and red blood cell transfusions.