In December 2018, roxadustat, developed by FibroGen, in collaboration with Astellas and AstraZeneca, received its first global approval in China for the treatment of anaemia in patients with dialysis-dependent chronic kidney disease (DD-CKD).[1] Roxadustat is expected to launch in China in the second half of 2019.[2]

The New Drug Application (NDA) accepted by the National Medical Products Administration (NMPA/China FDA) in October 2017 was granted priority review and approval was based on a phase III study (FGCL-4592-806), which evaluated the safety and efficacy of roxadustat for the treatment of anaemia in patients with CKD on dialysis.[3] This randomized, open-label, active-controlled (epoetin alfa) trial was initiated in December 2015, enrolling 304 patients in China, and was completed in June 2017.[3] The primary endpoint of mean change in haemoglobin levels from baseline to weeks 23-27 was met.[3] Adverse events observed in the clinical trial were consistent with underlying diseases in CKD patients.[2]

Roxadustat is an orally-administered, first-in-class inhibitor of hypoxia-inducible factor (HIF) prolyl hydroxylase that increases iron availability and haemoglobin levels.[3][4]  HIF is responsible for the regulation of gene expression in response to reduced oxygen levels.[3] Inhibition of HIF-proyl hydroxylase enzymes prevents HIF breakdown and promotes HIF transcriptional activity.[1][3]

According to Sean Bohen, Executive Vice President, Global Medicines Development, and Chief Medical Officer at AstraZeneca, “This first approval of roxadustat in China is a significant step towards achieving our ambition to transform care in a condition where prevalence in China is increasing.”[4]

Worldwide, CKD affects more than 200 million people[5] and in China it is estimated that 500,000 dialysis patients with CKD are anaemic.[4]

Roxadustat is under regulatory review in Japan for anaemia in dialysis-dependent CKD patients.[1] Phase III trials assessing the safety and efficacy of roxadustat in the treatment of anaemia in non-dialysis-dependent CKD patients are ongoing.[1] Evaluation of the treatment of anaemia with roxadustat in patients with lower risk myelodysplastic syndrome (MDS) is currently underway.[1][3]

Chief Executive Officer Thomas B. Neff of FibroGen stated, “We believe roxadustat will make a significant difference […] by addressing a substantial unmet medical need in the treatment of anemia associated with chronic kidney disease. This is an exciting milestone, as we are realizing our decade-long commitment to bringing innovative medicine to people in China. We look forward to bringing roxadustat to patients worldwide.”[2]

For additional information related to the first approval of roxadustat please visit Drugs[1], or to learn more about the overall development of this drug across all indications visit AdisInsight[3].

Image credit: Tanushree Rao – Unsplash

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