Deferiprone, sold under the brand name Ferriprox among others, is a

medication A medication (also called medicament, medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy) is an important part of the medical field and re ...

that

chelate Chelation is a type of bonding of ions and molecules to metal ions. It involves the formation or presence of two or more separate coordinate bonds between a polydentate (multiple bonded) ligand and a single central metal atom. These ligands are ...

iron Iron () is a chemical element with symbol Fe (from la, ferrum) and atomic number 26. It is a metal that belongs to the first transition series and group 8 of the periodic table. It is, by mass, the most common element on Earth, right in f ...

and is used to treat iron overload in thalassaemia major. It was first approved and indicated for use in treating

thalassaemia Thalassemias are inherited blood disorders characterized by decreased hemoglobin production. Symptoms depend on the type and can vary from none to severe. Often there is mild to severe anemia (low red blood cells or hemoglobin). Anemia can result ...

major in 1994 and had been licensed for use in the European Union for many years while awaiting approval in Canada and in the United States. On October 14, 2011, it was approved for use in the US under the

FDA The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food ...

's accelerated approval program. The most common side effects include red-brown urine (showing that iron is being removed through the urine), nausea (feeling sick), abdominal pain (stomach ache) and vomiting. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Less common but more serious side effects are agranulocytosis (very low levels of granulocytes, a type of white blood cell) and neutropenia (low levels of neutrophils, a type of white blood cell that fights infections).

Medical uses

Deferiprone monotherapy is indicated in the European Union for the treatment of iron overload in those with thalassaemia major when current chelation therapy is contraindicated or inadequate. Deferiprone in combination with another chelator is indicated in the European Union in those with thalassaemia major when monotherapy with any iron chelator is ineffective, or when prevention or treatment of life-threatening consequences of iron overload (mainly cardiac overload) justifies rapid or intensive correction.

Controversy

Deferiprone was at the center of a protracted struggle between Nancy Olivieri, a Canadian

haematologist Hematology ( always spelled haematology in British English) is the branch of medicine concerned with the study of the cause, prognosis, treatment, and prevention of diseases related to blood. It involves treating diseases that affect the produc ...

and

researcher Research is "creative and systematic work undertaken to increase the stock of knowledge". It involves the collection, organization and analysis of evidence to increase understanding of a topic, characterized by a particular attentiveness t ...

, and the Hospital for Sick Children and the

pharmaceutical company The pharmaceutical industry discovers, develops, produces, and markets drugs or pharmaceutical drugs for use as medications to be administered to patients (or self-administered), with the aim to cure them, vaccinate them, or alleviate sympto ...

Apotex Apotex Inc. is a Canadian pharmaceutical corporation. Founded in 1974 by Barry Sherman, the company is the largest producer of generic drugs in Canada, with annual sales exceeding . By 2016, Apotex employed over 10,000 people as one of Canada's ...

, that started in 1996, and delayed approval of the drug in North America. Olivieri's data suggested deferiprone leads to progressive hepatic fibrosis.

History

Deferiprone was approved for medical use in the European Union in August 1999. It was approved for medical use in the United States in October 2011. Generic versions were approved in August 2019. The safety and effectiveness of deferiprone is based on an analysis of data from twelve clinical studies in 236 participants. Participants in the study did not respond to prior iron chelation therapy. Deferiprone was considered a successful treatment for participants who experienced at least a 20 percent decrease in serum ferritin, a protein that stores iron in the body for later use. Half of the participants in the study experienced at least a 20 percent decrease in ferritin levels.

References

External links

* {{Portal bar , Medicine Chelating agents Chelating agents used as drugs Antidotes 4-Pyridones