Despite a high-profile FDA rejection, FibroGen and its partners have been able to conquer new territories outside of the U.S. with novel oral anemia drug Evrenzo.
Evrenzo, also known as roxadustat, has won reimbursement backing from England’s drug cost watchdog for treating some patients with symptomatic anemia caused by chronic kidney disease. Astellas holds Evrenzo’s rights in Europe.
The coverage decision (PDF) from the National Institute for Health and Care Excellence (NICE) “is a vote of confidence in roxadustat as a cost-effective oral treatment option,” Timir Patel, medical director at Astellas UK & Ireland, said in a statement.
NICE’s recommendation comes with some limitations, which Astellas said are in line with Evrenzo’s local marketing authorization. Evrenzo is only covered in patients with stage 3 to 5 chronic kidney disease (CKD) with no iron deficiency and in those who are not on dialysis.
In its evaluation, NICE noted that Evrenzo can serve as an alternative to existing erythropoiesis-stimulating agents (ESAs), which are injectables. As NICE’s reviewers noted, “many people with anemia find injecting themselves unpleasant and difficult.” As a result, these patients would welcome an oral treatment, the reviewers said. Evrenzo marks the first oral HIF-PH inhibitor approved anywhere.
But perhaps more importantly, Astellas offered a confidential discount to make the drug a cost-effective use of NHS resources. Astellas offers five dosing forms of Evrenzo in 12-tablet packs. The lowest dosing, of 20-mg tablets, carries the list price of 59.24 pounds ($74.3). The highest, 150-mg regimen comes with a list price of 444.32 pounds (557.27). Those prices don't reflect the discounts offered by Astellas.
FibroGen and partners conducted four randomized clinical trials for Evrenzo in CKD anemia patients who were not on dialysis. But NICE only considered one trial, dubbed Dolomites, because the others pitted Evrenzo against a placebo.
In Dolomites, Evrenzo showed it was comparable to Amgen’s ESA therapy Aranesp in achieving and maintaining target hemoglobin levels. Further, the novel drug was superior at reducing the need for iron infusions.
Evrenzo nabbed its first-in-class approval in China in late 2018 under a partnership between FibroGen and AstraZeneca. At that time, it looked on track to be first to market in the U.S. as well. But the FDA rejected the med last year, taking issue with its side effect profile, especially some cardiovascular disease risks such as blood clots and deaths. AZ and FibroGen still can’t agree on whether or how to conduct a new clinical trial potentially supporting a resubmission with the FDA.
Meanwhile, European regulators are OK with the drug’s side effects. The European Commission cleared the drug in symptomatic anemia associated with CKD last year, a decision that was quickly followed by a nod from the U.K.’s Medicines and Healthcare products Regulatory Agency.
Last month, Astellas rolled out a pooled analysis of four trials, saying Evrenzo didn’t increase the risk of cardiovascular events or deaths compared with traditional ESAs.