FDA panel votes overwhelmingly against approval of FibroGen's roxadustat

An FDA advisory panel on Thursday voted overwhelmingly against recommending approval of FibroGen's oral HIF-PH inhibitor roxadustat to treat anaemia in chronic kidney disease (CKD) for both non-dialysis-dependent (NDD) and dialysis-dependent (DD) patients. The company, whose shares plunged up to 45% after the news, is developing the drug with AstraZeneca.

Specifically, only one member of the 14-person advisory committee thought that roxadustat should be authorised for use in NDD patients, with all others voting against. Meanwhile, two panel members supported approval in DD patients, with the remaining 12 people voting against clearance in this indication. Enrique Conterno, CEO of FibroGen, remarked "while we are disappointed with today's outcome, we believe the scientific evidence supports roxadustat approval in the US and will work with the FDA as it completes its review."

In briefing documents released ahead of the meeting, FDA staff said that while roxadustat's efficacy is "not in question," the drug is associated with "important risks of serious thromboembolic events, as well as other risks." The agency reviewers noted that while it was "clear-cut" to interpret the results of safety analyses for the dialysis population, the same could not be said for the NDD studies because of "considerable disparity" in drop-out rates between the roxadustat and placebo arms.

Roxadustat, which is also partnered with Astellas, is currently approved in Japan, under the name Evrenzo, for the treatment of anaemia in CKD in NDD and DD adults, whilst it is also authorised in China, Chile and South Korea in the two patient populations. Late last month, roxadustat also received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use, with the drug set to be approved in both NDD and DD patients.

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