The Food and Drug Administration (FDA)’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted 13 to 1 against the approval of roxadustat for the treatment of anemia in chronic kidney disease (CKD) in non-dialysis dependent adult patients, and 12 to 2 against the approval in dialysis-dependent adult patients.
The FDA panel raised several questions about the roxadustat development program, particularly regarding the safety analyses. Concerns related to the potential for thromboembolic events and major adverse cardiovascular events were discussed during the meeting.
Roxadustat is an investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor that promotes erythropoiesis by increasing endogenous erythropoietin production. Additionally, it improves iron regulation and eases the negative impact of inflammation on hemoglobin (Hb) synthesis and red blood cell production by downregulating hepcidin. The NDA submission is supported by data from a global phase 3 program that included over 8000 patients.
“While we are disappointed with today’s outcome,” said Enrique Conterno, Chief Executive Officer, FibroGen, “we believe the scientific evidence supports roxadustat approval in the US and will work with the FDA as it completes its review of the New Drug Application for roxadustat.”
Although not bound by the committee’s recommendations, the FDA does take them into consideration when making decisions on approval.
- Status on FDA Advisory Committee vote on roxadustat in anemia of chronic kidney disease. News release. AstraZeneca. Accessed July 16, 2021. https://www.astrazeneca.com/media-centre/press-releases/2021/status-on-us-fda-advisory-committee-for-roxadustat.html.
- FibroGen announces outcome of FDA advisory committee review of roxadustat for treatment of anemia of chronic kidney disease. News release. FibroGen. Accessed July 16, 2021. https://www.globenewswire.com/news-release/2021/07/15/2264019/33525/en/FibroGen-Announces-Outcome-of-FDA-Advisory-Committee-Review-of-Roxadustat-for-Treatment-of-Anemia-of-Chronic-Kidney-Disease.html.