Roxadustat, sold under the brand name Evrenzo, is an anti-

anemia Anemia (also spelt anaemia in British English) is a blood disorder in which the blood has a reduced ability to carry oxygen. This can be due to a lower than normal number of red blood cells, a reduction in the amount of hemoglobin availabl ...

medication. Roxadustat is a

HIF prolyl-hydroxylase inhibitor Hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHIs) are a novel class of oral medications developed for the treatment of anemia in chronic kidney disease (CKD). These drugs work by inhibiting hypoxia-inducible factor-proline diox ...

that increases endogenous production of

erythropoietin Erythropoietin (; EPO), also known as erythropoetin, haematopoietin, or haemopoietin, is a glycoprotein cytokine secreted mainly by the kidneys in response to cellular hypoxia; it stimulates red blood cell production ( erythropoiesis) in th ...

and stimulates production of

hemoglobin Hemoglobin (haemoglobin, Hb or Hgb) is a protein containing iron that facilitates the transportation of oxygen in red blood cells. Almost all vertebrates contain hemoglobin, with the sole exception of the fish family Channichthyidae. Hemoglobin ...

and

red blood cell Red blood cells (RBCs), referred to as erythrocytes (, with -''cyte'' translated as 'cell' in modern usage) in academia and medical publishing, also known as red cells, erythroid cells, and rarely haematids, are the most common type of blood cel ...

s. It was investigated in clinical trials for the treatment of anemia caused by

chronic kidney disease Chronic kidney disease (CKD) is a type of long-term kidney disease, defined by the sustained presence of abnormal kidney function and/or abnormal kidney structure. To meet criteria for CKD, the abnormalities must be present for at least three mo ...

(CKD). It is taken

by mouth Oral administration is a route of administration whereby a substance is taken through the Human mouth, mouth, swallowed, and then processed via the digestive system. This is a common route of administration for many medications. Oral administ ...

. The drug was developed by FibroGen, in partnership with

AstraZeneca AstraZeneca plc () (AZ) is a British-Swedish multinational pharmaceutical and biotechnology company with its headquarters at the Cambridge Biomedical Campus in Cambridge, UK. It has a portfolio of products for major diseases in areas includi ...

. The most common side effects include

hypertension Hypertension, also known as high blood pressure, is a Chronic condition, long-term Disease, medical condition in which the blood pressure in the artery, arteries is persistently elevated. High blood pressure usually does not cause symptoms i ...

(high blood pressure), vascular access thrombosis (formation of blood clots in the blood vessels associated with dialysis),

diarrhea Diarrhea (American English), also spelled diarrhoea or diarrhœa (British English), is the condition of having at least three loose, liquid, or watery bowel movements in a day. It often lasts for a few days and can result in dehydration d ...

peripheral edema Peripheral edema is edema (accumulation of fluid causing swelling) in tissues perfused by the peripheral vascular system, usually in the lower limbs. In the most dependent parts of the body (those hanging distally), it may be called dependent ede ...

(swelling especially of the ankles and feet),

hyperkalemia Hyperkalemia is an elevated level of potassium (K+) in the blood. Normal potassium levels are between 3.5 and 5.0 mmol/L (3.5 and 5.0 mEq/L) with levels above 5.5mmol/L defined as hyperkalemia. Typically hyperkalemia does not cause symptoms. Oc ...

(high blood potassium levels) and

nausea Nausea is a diffuse sensation of unease and discomfort, sometimes perceived as an urge to vomit. It can be a debilitating symptom if prolonged and has been described as placing discomfort on the chest, abdomen, or back of the throat. Over 30 d ...

(feeling sick). Roxadustat received its first global approval in China on 17 December 2018, for the treatment of anemia caused by CKD in patients who are dialysis-dependent. It was approved in Japan in 2019, for the treatment of anemia caused by CKD in patients on dialysis, and in 2020 for patients not on dialysis. Roxadustat was approved for medical use in the European Union in August 2021.

Medical uses

Roxadustat is indicated for treatment of adults with symptomatic anemia associated with

(CKD). Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

Adverse effects

Roxadustat is reported to increase

VEGF Vascular endothelial growth factor (VEGF, ), originally known as vascular permeability factor (VPF), is a signal protein produced by many cells that stimulates the formation of blood vessels. To be specific, VEGF is a sub-family of growth factors ...

, a signal protein that can activate tumor growth and also is considered to cause

pulmonary hypertension Pulmonary hypertension (PH or PHTN) is a condition of increased blood pressure in the pulmonary artery, arteries of the lungs. Symptoms include dypsnea, shortness of breath, Syncope (medicine), fainting, tiredness, chest pain, pedal edema, swell ...

. In phase 3 trial conducted at 29 sites in China, roxadustat treatment was found to cause

, i.e., increase in serum potassium, and metabolic acidosis in patients.

Society and culture

Legal status

In July 2021, the Cardiovascular and Renal Drugs Advisory Committee of the US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) voted against the use of roxadustat in people with anemia in chronic kidney disease, both for those that are non-dialysis-dependent and those that are on dialysis. Significant safety concerns were raised that the panelists believed could not be addressed without further study. Notably, prior to the vote of the FDA committee, FibroGen and AstraZeneca announced that the company had changed parameters used to analyze cardiovascular safety data, which made the drug appear safer than it is.

Usage as a doping product

Due to the potential applications of roxadustat in

athletic doping In competitive sports, doping is the use of banned athletic performance-enhancing drugs (PEDs) by athletes, as a way of cheating. As stated in the World Anti-Doping Code by WADA, doping is defined as the occurrence of one or more of the anti-d ...

, such as raising haemoglobin levels and stimulating the production of red blood cells, it has been incorporated into screens for

performance-enhancing drugs Performance-enhancing substances (PESs), also known as performance-enhancing drugs (PEDs), are substances that are used to improve any form of activity performance in humans. Many substances, such as anabolic steroids, can be used to improve at ...

, as it has already been detected being used illicitly by athletes. In March 2024, following a lengthly process, former world No. 1 tennis player

Simona Halep Simona Halep (; born 27 September 1991) is a Romanian former professional tennis player. She was ranked as the List of WTA number 1 ranked singles tennis players, world No. 1 in women's singles by the Women's Tennis Association (WTA) for 64 w ...

received a nine month ban for unintentional use of roxadustat that was sourced to a contaminated supplement that she had ingested.

Medical uses

Adverse effects

Society and culture

Legal status

Usage as a doping product

References

Further reading