A complete response letter (CRL) has been issued by the US Food and Drug Administration (FDA) regarding roxadustat for the treatment of anaemia of chronic kidney disease (CKD), in both non-dialysis dependent (NDD) and dialysis-dependent (DD) adult patients.
Oral HIF-PH inhibitors promote erythropoiesis, or RBC production, through increased endogenous production of erythropoietin, improved iron absorption and mobilisation, and reduction of hepcidin. Roxadustat is an oral medicine that could be the first in this new class of treatments.
The CRL requested an additional clinical trial on the safety of roxadustat in both the NDD and DD patient populations.
The CRL follows concerns raised by the FDA last month over serious thrombotic events and seizures with roxadustat treatment, which were detected against a placebo background in the NDD patient population.
In briefing documents published ahead of a meeting of the Cardiovascular and Renal Drugs Advisory Committee meeting on 15 July, the FDA said that “roxadustat’s efficacy is not in question”, however “the principal issues before the committee are the drug’s safety, and safety with respect to the specific CKD populations”.
The FDA advisory committee then voted against approval of roxadustat.
AstraZeneca and FibroGen have said that they are working together to “evaluate next steps”.
Cardiovascular, renal and metabolism (CVRM) is a key growth driver for AstraZeneca and is one of the company’s main disease areas.
Roxadustat is approved in a number of countries, including China under the brand name Evrenzo. It has recently received a positive CHMP opinion in the EU and is under regulatory review in other jurisdictions.
The drug is also in clinical development for anaemia associated with MDS and for chemotherapy-induced anaemia.