Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) and FibroGen, Inc. (Nasdaq: FGEN, CEO: Enrique Conterno, “FibroGen”) today announced that the European Commission (EC) has approved EVRENZO™ (roxadustat) for the treatment of adult patients with symptomatic anemia associated with chronic kidney disease (CKD).
“We are very pleased EVRENZO has been approved as the first oral HIF-PH inhibitor to treat adult patients with symptomatic anemia associated with CKD in the European Union,” said Steven Benner, M.D., M.H.S., President of Development, Astellas. “Today’s approval provides patients, regardless of dialysis status, with a first-in-class treatment option to address the multifaceted nature of this condition. We look forward to making roxadustat available to adult patients with anemia of CKD in countries across the European Union.”
CKD impacts one in 10 people globally, of whom one in five are affected by anemia.1, 2 Anemia of CKD is often untreated or not treated to target, and is associated with reduced quality of life and progression to adverse cardiovascular (CV) and renal outcomes.3-5
“Anemia is a significant and early complication of CKD that occurs with greater frequency and impact as CKD worsens, affecting patients’ day-to-day living, self-care and mobility,” said Jonathan Barratt, Ph.D., FRCP, Consultant Nephrologist and the Mayer Professor of Renal Medicine at the University of Leicester, United Kingdom. “This approval represents a step forward in providing patients with an efficient and simple option to manage anemia symptoms and maintain target hemoglobin levels to minimize the impact on their quality of life.”
Roxadustat is the first orally administered HIF-PH inhibitor available in the European Union. Roxadustat increases hemoglobin (Hb) levels through a different mechanism of action compared to injectable erythropoiesis-stimulating agents (ESAs) which are typically co-administered with intravenous iron. As a HIF-PH inhibitor, roxadustat activates the body’s natural response to reduced oxygen levels in the blood. This response involves the regulation of multiple, coordinated processes that allow management of anemia with a reduced use of intravenous iron.
“HIF-PH inhibitors represent a major advance in the treatment of anemia of CKD,” said Mark Eisner, M.D., M.P.H., Chief Medical Officer, FibroGen. “Roxadustat provides a novel breakthrough for patients who suffer from this condition.”
This approval follows the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) positive opinion to authorize roxadustat in June6 based on results from a comprehensive pivotal Phase 3 program comprising of eight multicenter and randomized studies, which involved 9,600 patients worldwide.7-12 The results of this program showed roxadustat was efficacious in achieving and maintaining target Hb levels (10-12g/dL) in patients with symptomatic anemia of CKD regardless of dialysis status and irrespective of prior ESA treatment.7-11 The safety profile observed in the roxadustat development program is reflective of the CKD populations studied and comparable to ESAs.7-12
The EC has the authority to approve medicines for European Union member states, as well as in the European Economic Area (EEA) countries Iceland, Norway, Liechtenstein.13
The EC approval of roxadustat triggers a milestone payment of $120 million by Astellas to FibroGen, and FibroGen will also receive royalties based upon European net sales.
About CKD and Anemia of CKD
Chronic kidney disease (CKD) is a progressive disease characterized by gradual loss of kidney function that may eventually lead to kidney failure or end-stage renal disease, requiring dialysis or kidney transplant.14 Many patients with CKD die of cardiovascular complications before progressing to kidney failure and as such the prevalence of early kidney disease is much greater than end-stage disease.14, 15 CKD impacts one in 10 people globally and is predicted to become the fifth most common cause of premature death globally by 2040.1, 16
Anemia, a serious medical condition in which patients have insufficient red blood cells and low levels of hemoglobin, is a common early complication of CKD affecting approximately 20% of CKD patients.2, 17 Anemia of CKD is associated with an increased risk of hospitalization, cardiovascular complications and death, and can also cause significant fatigue, cognitive dysfunction and reduced quality of life.4, 18 Blood transfusions are used for treating severe anemia, however, they may reduce a patient’s opportunity for kidney transplant and can increase the risk of infection and/or complications such as heart failure and allergic reactions.19, 20
Roxadustat, an oral medicine, is the first in a new class of medicines, HIF-PH inhibitors, that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin; improved iron absorption and mobilization; and downregulation of hepcidin. Roxadustat is also in Phase 3 clinical development for anemia associated with myelodysplastic syndromes (MDS) and Phase 2 for chemotherapy-induced anemia (CIA).
Roxadustat is approved in EU member states, including the EEA countries, as well as in Japan, China, Chile and South Korea for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). Several other licensing applications for roxadustat have been submitted by Astellas and AstraZeneca to regulatory authorities across the globe and are currently in review.
Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia of CKD in territories including Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia of CKD in the U.S., China, other markets in the Americas, in Australia/New Zealand, and Southeast Asia.
Important Safety Information
The full European Summary of Product Characteristics (SPC/SmPC) for roxadustat will be available from the European Medicines Agency at www.ema.europa.eu 21