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

anemia Anemia or anaemia (British English) is a blood disorder in which the blood has a reduced ability to carry oxygen due to a lower than normal number of red blood cells, or a reduction in the amount of hemoglobin. When anemia comes on slowly, th ...

medication. Roxadustat is a

HIF prolyl-hydroxylase inhibitor Not to be confused with '' Factor Inhibiting HIF Asparaginyl Hydroxylase Inhibitors'' Hypoxia-inducible factor prolyl hydroxylase Inhibitors (HIF-PHIs) also known as hypoxia-inducible factor stabilizers (HIF stabilizers) are members of a mostl ...

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 the bo ...

and stimulates production of

hemoglobin Hemoglobin (haemoglobin BrE) (from the Greek word αἷμα, ''haîma'' 'blood' + Latin ''globus'' 'ball, sphere' + ''-in'') (), abbreviated Hb or Hgb, is the iron-containing oxygen-transport metalloprotein present in red blood cells (erythrocyte ...

and

red blood cell Red blood cells (RBCs), also referred to as red cells, red blood corpuscles (in humans or other animals not having nucleus in red blood cells), haematids, erythroid cells or erythrocytes (from Greek ''erythros'' for "red" and ''kytos'' for "holl ...

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 kidney disease in which a gradual loss of kidney function occurs over a period of months to years. Initially generally no symptoms are seen, but later symptoms may include leg swelling, feeling tired, vo ...

(CKD). It is taken

by mouth Oral administration is a route of administration where a substance is taken through the mouth. Per os abbreviated to P.O. is sometimes used as a direction for medication to be taken orally. Many medications are taken orally because they are i ...

. The drug was developed by FibroGen, in partnership with

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

. The most common side effects include

hypertension Hypertension (HTN or HT), also known as high blood pressure (HBP), is a long-term medical condition in which the blood pressure in the arteries is persistently elevated. High blood pressure usually does not cause symptoms. Long-term high bl ...

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

diarrhea Diarrhea, also spelled diarrhoea, is the condition of having at least three loose, liquid, or watery bowel movements each day. It often lasts for a few days and can result in dehydration due to fluid loss. Signs of dehydration often begin wi ...

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 edem ...

(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 5.0mmol/L (3.5 and 5.0mEq/L) with levels above 5.5mmol/L defined as hyperkalemia. Typically hyperkalemia does not cause symptoms. Occasi ...

(high blood potassium levels) and

nausea Nausea is a diffuse sensation of unease and discomfort, sometimes perceived as an urge to vomit. While not painful, it can be a debilitating symptom if prolonged and has been described as placing discomfort on the chest, abdomen, or back of the ...

(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 © 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. 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

Due to its potential applications in

athletic doping In competitive sports, doping is the use of banned athletic performance-enhancing drugs by athletic competitors as a way of cheating in sports. The term ''doping'' is widely used by organizations that regulate sporting competitions. The use of ...

, it has been incorporated into screens for

performance-enhancing drugs Performance-enhancing substances, also known as performance-enhancing drugs (PEDs), are substances that are used to improve any form of activity performance in humans. A well-known example of cheating in sports involves doping in sport, where bann ...

, as it has already been detected being used illicitly by athletes despite not having yet been approved for medical use. In October 2022, it was announced that Romanian former world No. 1 tennis player

Simona Halep Simona Halep (; born 27 September 1991) is a Romanian professional tennis player. She has been ranked world No. 1 in singles twice between 2017 and 2019, for a total of 64 weeks, which ranks eleventh in the history of the Women's Tennis Assoc ...

had tested positive for roxadustat at the 2022 US Open.

FDA rejection

On July 15, 2021, the FDA Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted against roxadustat's use in patients 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 FDA committee's vote, FibroGen and AstraZeneca announced that the company had changed parameters used to analyze cardiovascular safety data, which made the drug apprear safer than it is.

References

External links