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). Roxadustat is expected to launch in China in the second half of 2019.
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. 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. The primary endpoint of mean change in haemoglobin levels from baseline to weeks 23-27 was met. Adverse events observed in the clinical trial were consistent with underlying diseases in CKD patients.
Roxadustat is an orally-administered, first-in-class inhibitor of hypoxia-inducible factor (HIF) prolyl hydroxylase that increases iron availability and haemoglobin levels. HIF is responsible for the regulation of gene expression in response to reduced oxygen levels. Inhibition of HIF-proyl hydroxylase enzymes prevents HIF breakdown and promotes HIF transcriptional activity.
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.”
Roxadustat is under regulatory review in Japan for anaemia in dialysis-dependent CKD patients. Phase III trials assessing the safety and efficacy of roxadustat in the treatment of anaemia in non-dialysis-dependent CKD patients are ongoing. Evaluation of the treatment of anaemia with roxadustat in patients with lower risk myelodysplastic syndrome (MDS) is currently underway.
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.”
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