A first-in-class drug for anaemia caused by chronic kidney disease (CKD) developed by FibroGen and AstraZeneca has cleared two phase III studies in China and will be filed for approval before the end of 2017.
Roxadustat is currently leading the pack among a new class of orally-active hypoxia-inducible prolyl hydroxylase inhibitors (HIF-PHIs) and – if approved for marketing – could become a $400m product by 2020 and bring in $2bn or more at peak, according to analysts.
The drug was able to correct anaemia caused by CKD in the two trials, achieving a significant increase in haemoglobin levels compared to placebo in one eight-week study and matching the efficacy of an injectable erythropoietin (EPO) product in a second six-month trial. A year-long safety assessment in around 100 patients is ongoing and due to generate results in June.
The positive results in Chinese patients means that AZ and FibroGen can press ahead with plans to file for approval in China this year, although they will have to wait for results from other trials – due in 2018 – before submitting the new drug in Europe and the US. Roxadustat works by turning on the body’s machinery for producing red blood cells that is usually stimulated by reduced oxygen levels in the blood.
The drug and other HIF-PHIs in the pipeline – which include GlaxoSmithKline’s daprodustat and Akebia/Takeda’s vadadustat, which are also in phase III testing – could provide the first oral alternative to injectable EPO for anaemia and iron supplementation.
FibroGen chief executive Tom Neff said: ”We are very encouraged by the top line results of these two pivotal phase III studies for roxadustat, which bring us closer to the first oral anaemia therapy that does not require injectable administration or intravenous iron supplementation.
“In addition, use of roxadustat is free of the burden of cold-chain storage and maintaining sterility, and costly distribution infrastructure.”
If approved, AZ will market the drug while FibroGen will be responsible for manufacturing and medical affairs under an agreement agreed between the two companies in 2013.
Astellas has commercialisation rights in Japan. AZ agreed to pay up to $815m for rights to the drug, including $350m in upfront and non-contingent payments and to date FibroGen has received around $185m from AZ.