pharma · Asthma · Type 2 Diabetes
AstraZeneca is a pharma organization headquartered in Södertälje, SE. Primary therapeutic focus areas include Asthma, Type 2 Diabetes, Chronic Obstructive Pulmonary Disease (COPD), Chronic Obstructive Pulmonary Disease,
Phase 3 · small molecule · Anemia
Roxadustat (EVRENZO) is a small-molecule hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor developed by AstraZeneca for the treatment of anemia. The drug operates through a novel mechanism distinct from traditional erythropoiesis-stimulating agents, targeting the HIF pathway to promote endogenous erythropoiet
Internal code D5740C00001
Roxadustat (EVRENZO) is a small-molecule hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor developed by AstraZeneca for the treatment of anemia. The drug operates through a novel mechanism distinct from traditional erythropoiesis-stimulating agents, targeting the HIF pathway to promote endogenous erythropoietin production. AstraZeneca's program has completed Phase 3 clinical development, with the most recent milestone recorded in December 2019. The compound has achieved regulatory approval in multiple jurisdictions: Japan approved roxadustat in September 2019, and the European Union approved EVRENZO in May 2026 under marketing authorization holder Astellas Pharma Europe B.V. In China, the drug remains in clinical trials as of the latest available data. The program represents a strategic entry into the anemia treatment market, which is dominated by established erythropoiesis-stimulating agents and iron supplementation therapies. Roxadustat's oral formulation and distinct mechanism offer potential differentiation in patient populations with chronic kidney disease-related anemia and other anemia indications.
Anemia represents a significant unmet medical need affecting millions of patients globally, particularly those with chronic kidney disease, cancer, and other chronic conditions. Traditional treatments rely on erythropoiesis-stimulating agents (ESAs) such as epoetin alfa and darbepoetin alfa, which carry cardiovascular and thromboembolic risks, and iron supplementation, which has limited efficacy in certain patient populations. Roxadustat's HIF prolyl hydroxylase inhibitor mechanism offers a pharmacologically distinct approach that stimulates endogenous erythropoietin production, potentially reducing the cardiovascular risks associated with exogenous ESA administration. The oral route of administration provides convenience advantages over intravenous or subcutaneous ESA therapies, addressing patient preference and adherence challenges. The competitive landscape includes multiple established players—Amgen (ARANESP, MIRCERA via Roche partnership), Hoffmann-La Roche (MIRCERA, NEORECORMON), and Takeda (FERAHEME, OMONTYS)—making market penetration dependent on demonstrable clinical and safety advantages. Roxadustat's approval in Japan and Europe validates the HIF inhibitor class and establishes commercial viability, though the program's completion status and absence of disclosed peak sales projections suggest AstraZeneca may be evaluating strategic options or managing market entry sequentially across regions.
Drug Class: Hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor
Modality: Small-molecule oral therapeutic
Indication: Anemia (chronic kidney disease-related anemia and other anemia indications)
Therapeutic Classification: Blood and blood forming organs (ATC B03)
Brand Name: EVRENZO
International Nonproprietary Name (INN): Roxadustat
Route of Administration: Not yet disclosed in available data
Mechanism of Action: HIF prolyl hydroxylase inhibition, promoting endogenous erythropoietin production and iron mobilization
Target: Not yet disclosed in available data
Related Therapies: Erythropoiesis-stimulating agents (epoetin alfa, darbepoetin alfa), iron supplements, and other HIF inhibitors in development
First Approval: Japan, September 2019; European Union, May 2026
Patent Status: Not yet disclosed in available data
Also known as: anaemia (disease), anemia (disease)
A reduction in the number of red blood cells, the amount of hemoglobin, and/or the volume of packed red blood cells. Clinically, anemia represents a reduction in the oxygen-transporting capacity of a designated volume of blood, resulting from an imbalance between blood loss (through hemorrhage or hemolysis) and blood production. Signs and symptoms of anemia may include pallor of the skin and mucous membranes, shortness of breath, palpitations of the heart, soft systolic murmurs, lethargy, and fatigability.
ClinicalTrials.gov lists 98 registered studies for Anaemia, (AACT aggregate).
Phase breakdown: NA (35), PHASE3 (21), PHASE1 (18), PHASE2 (12), PHASE4 (11), PHASE2/PHASE3 (1)
Common investigational therapies:
Disease data sourced from MONDO Disease Ontology (MONDO:0002280), NCT00466297, NCT00767702, NCT01043133, NCT01317979, NCT01477281, AACT (ClinicalTrials.gov aggregate), ClinicalTrials.gov, NCT00140517, NCT00238043, NCT00258024, NCT00259142, NCT00276224, Open Targets Platform (CC BY 4.0).
Phase 3 completion milestone
Latest disclosed milestone for the roxadustat program; specific trial outcome not yet disclosed.
Japan regulatory approval
Roxadustat approved in Japan for anemia treatment.
European Union regulatory approval
EVRENZO approved by EMA under marketing authorization holder Astellas Pharma Europe B.V.
Roxadustat enters a mature anemia treatment market dominated by established erythropoiesis-stimulating agents and iron therapies. Amgen's ARANESP (darbepoetin alfa) and Hoffmann-La Roche's MIRCERA (methoxy polyethylene glycol-epoetin beta) and NEORECORMON (epoetin beta) represent the ESA standard of care. Takeda markets FERAHEME (ferumoxytol) and OMONTYS (peginesatide), providing alternative iron and ESA options. Teva Pharma GmbH offers EPORATIO (epoetin theta), and additional approved competitors include JESDUVROQ, FERACCRU, DYNEPO, REBLOZYL, and RETACRIT, collectively representing diverse mechanisms and formulations. Roxadustat's HIF prolyl hydroxylase inhibitor mechanism differentiates it from traditional ESAs by stimulating endogenous erythropoietin production rather than providing exogenous hormone replacement, potentially offering improved cardiovascular safety profiles. The oral formulation contrasts with predominantly parenteral competitors, addressing patient convenience and adherence. However, competitive intensity remains high, with established market share, clinical familiarity, and reimbursement pathways favoring incumbent therapies. Roxadustat's regulatory approvals in Japan and Europe validate the HIF inhibitor class but do not guarantee rapid market adoption absent compelling clinical trial data demonstrating superiority in efficacy, safety, or patient outcomes versus existing standards.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| FERAHEME | Takeda | — | approved |
| OMONTYS | Takeda | — | approved |
| EPORATIO | Teva Pharma GmbH | — | approved |
| EPOSTIM | — | — | approved |
| JESDUVROQ | — | — | approved |
| FERACCRU | — | — | approved |
| ARANESP | Amgen | — | approved |
| MIRCERA | Hoffmann-La Roche | — | approved |
| DYNEPO | — | — | approved |
| NEORECORMON | Hoffmann-La Roche | — | approved |
| REBLOZYL | — | — | approved |
| RETACRIT | — | — | approved |
| VOXELOTOR | — | Hemoglobin HbA positive modulator | Approved |
| TRIAMCINOLONE ACETONIDE | — | Glucocorticoid receptor agonist | Approved |
| SUTIMLIMAB | — | Complement C1s inhibitor | Approved |
| RUXOLITINIB PHOSPHATE | — | Tyrosine-protein kinase JAK2 inhibitor | Approved |
| RAVULIZUMAB | — | Complement C5 inhibitor | Approved |
| PREDNISONE | — | Glucocorticoid receptor agonist | Approved |
| PREDNISOLONE SODIUM PHOSPHATE | — | Glucocorticoid receptor agonist | Approved |
| PREDNISOLONE ACETATE | — | Glucocorticoid receptor agonist | Approved |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
Japan (PMDA): Roxadustat approved in September 2019, representing the first regulatory approval for the compound and validation of the HIF prolyl hydroxylase inhibitor mechanism in anemia treatment.
European Union (EMA): EVRENZO approved on 11 May 2026 under EMA product number EMEA/H/C/004871, with marketing authorization held by Astellas Pharma Europe B.V. This approval follows Phase 3 program completion and represents the second major regulatory jurisdiction to approve roxadustat.
China (NMPA): Roxadustat remains in clinical trials as of the latest available data (NCT05010460 ongoing), with no regulatory approval yet disclosed. Clinical trial status suggests ongoing development and potential future submission to Chinese regulatory authorities.
United States (FDA): Regulatory status not yet disclosed in available data; no approval or filing information is currently available.
Summary: Roxadustat has achieved regulatory approval in two major markets (Japan and Europe) but remains in clinical development in China. U.S. regulatory status is not yet disclosed.
Roxadustat is used to treat anemia, including anemia associated with chronic kidney disease and other chronic conditions. It works by stimulating the body's natural production of erythropoietin through HIF prolyl hydroxylase inhibition.
Yes. Roxadustat was approved in Japan in September 2019 and by the European Union in May 2026 under the brand name EVRENZO. It remains in clinical trials in China. U.S. regulatory status is not yet disclosed.
Roxadustat is a hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor that promotes endogenous erythropoietin production and iron mobilization, stimulating red blood cell production through a mechanism distinct from traditional erythropoiesis-stimulating agents.
AstraZeneca developed roxadustat. In Europe, marketing authorization is held by Astellas Pharma Europe B.V. as of the May 2026 approval. Regional commercialization arrangements may vary.
Phase 3 development was completed as of December 2019. Key trial NCT02174627 is referenced, though detailed results are not yet disclosed in available data. Additional trial NCT05010460 is ongoing in China.
Roxadustat is a small-molecule therapeutic. The specific route of administration is not yet disclosed in available data, though oral formulation is implied by its classification as a small-molecule HIF inhibitor.
The internal development code is D5740C00001.
Competitors include erythropoiesis-stimulating agents such as Amgen's ARANESP and Hoffmann-La Roche's MIRCERA and NEORECORMON, as well as iron therapies like Takeda's FERAHEME and OMONTYS, and other approved anemia treatments including JESDUVROQ, FERACCRU, REBLOZYL, and RETACRIT.
Roxadustat is classified under ATC code B03 (Blood and blood forming organs), reflecting its role in treating anemia and blood disorders.
Phase 3 clinical development has been completed. The program is now in regulatory and commercialization phases following approvals in Japan and Europe.
No partner is disclosed in available data for the development program, though Astellas Pharma Europe B.V. holds European marketing authorization, suggesting a regional commercialization arrangement.
Traditional anemia treatments carry cardiovascular and thromboembolic risks, and some patient populations have limited response to iron supplementation. Roxadustat's oral HIF inhibitor mechanism offers an alternative approach with potentially improved safety and convenience.
First disclosure date is not yet disclosed in available data. The earliest documented milestone is the December 2019 Phase 3 completion.
Projected peak sales are not yet disclosed in available data.
Consensus analyst position is not yet disclosed in available data.
Patent status is not yet disclosed in available data.
Expected next milestones are not yet disclosed in available data. Potential catalysts include U.S. regulatory submission, China NMPA approval, and publication of Phase 3 trial results.
Roxadustat → Drug → Target → Indication → Company → Trials → Competitors
Strategic Positioning: AstraZeneca's completion of Phase 3 development and subsequent regulatory approvals in Japan (2019) and Europe (2026) indicate a deliberate, sequential market entry strategy. The seven-year gap between Japan approval and European approval suggests either regulatory complexity, manufacturing scale-up, or strategic timing considerations. The transfer of European marketing authorization to Astellas Pharma Europe B.V. may reflect partnership arrangements, regional commercialization strategies, or portfolio optimization decisions not yet disclosed.
Competitive Implications: Roxadustat's oral HIF inhibitor mechanism represents genuine differentiation from parenteral ESAs and iron therapies, addressing known cardiovascular and thromboembolic risks associated with traditional ESA use. However, market penetration will depend critically on Phase 3 trial data demonstrating non-inferiority or superiority in efficacy and safety endpoints versus standard of care. The absence of disclosed peak sales projections and consensus analyst positions suggests either limited commercial confidence or ongoing evaluation of market potential.
Future Catalysts: Key catalysts include (1) publication or disclosure of Phase 3 trial results from NCT02174627, (2) regulatory submission and approval decision in the United States, (3) advancement of NCT05010460 in China toward potential NMPA approval, (4) real-world evidence and post-marketing surveillance data from Japan and European markets, and (5) potential label expansions to additional anemia indications or patient populations.
Development Status Implications: The program's completed Phase 3 status combined with regulatory approvals in two major markets validates the clinical and regulatory pathway. However, the absence of recent milestones (latest disclosed December 2019) and lack of disclosed commercial metrics suggest the program may be in a consolidation or evaluation phase, with commercialization efforts potentially constrained by competitive pressures, reimbursement challenges, or internal strategic prioritization.
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Intelligence compiled from public regulatory and clinical sources (FDA, EMA, ClinicalTrials.gov and company disclosures). Figures may be editorial or analyst estimates; verify against primary sources before relying on them.