Deferiprone | Mechanism of Action, Uses & Side effects

What is Deferiprone?

Deferiprone (3-hydroxy-1,2-dimethylpyridin-4-one) is an iron-chelating agent that is synthetic and can be consumed orally. It belongs to the category of compounds known as 4-pyridones and has the structure of a pyridin-4(1H)-one that has methyl groups attached to positions 1 and 2 and a hydroxyl group attached to position 3. It is a crystalline powder that ranges in color from white to pinkish-white. It’s solubility in deionized water is low and has a melting point ranging anywhere from 272 to 278 degrees celsius.

It is used to treat the patients having transfusional iron overload. Patients with thalassemia, sickle cell disease or anemia are prescribed with Deferiprone to remove the excess iron from the body. It works by inhibiting a rate limiting enzyme in DNA synthesis- the intracellular ribonucleotide reductase.

Chemical Structure and Properties

Chemical Structure of Deferiprone

Name of Product Deferiprone
IUPAC Name 3-hydroxy-1,2-dimethylpyridin-4-one
Synonyms 3-Hydroxy-1,2-dimethyl-4(1H)-pyridone; Ferriprox; 1,2-dimethyl-3-hydroxy-4-pyridone; Deferiprona; défériprone
CAS No 30652-11-0
Molecular Formula C7H9NO2
Molecular Weight 139.15 g/mol
Appearance White to pinkish-white powder
Taste Very Bitter taste
Solubility Maximum water solubility of 16–18 g/L at 24°
Melting Point 272-278°C
Pubchem CID 2972
Pubchem SID 463821092


In 1981, Deferiprone was originally designed and screened in vitro and in vivo by Kontoghiorghes G. J. The safety and efficacy of deferiprone is based on an examination of twelve clinical investigations involving 236 individuals. Initially the journey of deferiprone was a difficult one in the treatment of thalassemia but after many complications and the usage of suboptimal dose, the aim of the drug to remove the excessive iron in thalassemia patients was achieved.

It was first approved and indicated for use in treating thalassaemia major in 1994. It was approved for medical use in the European Union in August 1999 and in the United States in October 2011. In August of 2019, approval was granted for generic versions.

Mechanism of Action

Mechanism of Action of Deferiprone

Deferiprone is a chelating agent that has a high affinity to ferric ion (iron III). It binds to ferric ions (iron III) and forms a stable and neutral 3:1  complex with the iron, which is excreted in the urine. That is why, the use of Deferiprone is commonly associated with orange discoloration of the urine. The red-brown urine actually is an indication of iron being removed through the urine.

The deferiprone molecule has a greater affinity for iron than for other metals such as zinc, copper, and aluminum.


Numerous individuals with Thalassemia, Sickle cell disease (SCD) or other anemias require chronic blood transfusions, which frequently results in iron overload necessitating chelation therapy. Blood transfusions aid in the treatment of blood diseases, but they also introduce excess iron into the body. It is critical to eliminate excess iron since high amounts of iron can create health concerns such as heart failure, liver disease, diabetes, and delayed growth in children.

When standard chelation therapy is insufficient to treat individuals with transfusional iron overload caused by thalassemia syndromes, deferiprone tablet is recommended as an alternative treatment option in adult and pediatric patients 8 years of age and older.

It may also be utilized to remove excess iron from the bodies of adults and children older than three years of age in the form of an oral solution in patients suffering from sickle cell disease (a form of inherited blood disease) or other forms of anemia.


Both tablet (500 mg and 1000 mg) and an oral solution (liquid) form (40g/400mL or  20g/250mL, having strength of 80 mg/mL and 50g/500mL, having strength of 100mg/mL) of deferiprone are available for oral administration. It is recommended to take this medication two or three times per day. One can take deferiprone with or without food, but taking with food assists in reducing the risk of experiencing nausea and vomiting as a side effect of the medicine.

The dosage recommended for the patients with transfusional iron overload varies with age and weight. It is taken in oral form and the starting dosage is 25mg/kg, thrice a day. It is recommended according to the body weight like 20kg person can have 500mg i.e., 1 tablet, 30 kg person can have 750mg i.e.,1.5 number of tablet . 250mg dose increases with every 10kg.

Side effects

Some unwanted effects can be commonly observed with this medicine, varying from person to person. The following list contains the side effects that are most frequently associated with the use of deferiprone:

  • Gastrointestinal symptoms such as nausea, vomiting, and abdominal pain
  • Chromaturia (reddish/brown discoloration of the urine)
  • Heartburn
  • Increased or decreased appetite
  • Pain in the arms, legs, back or joints

The adverse effects that are clinically significant are as follows:

  • Agranulocytosis (extremely low amounts of granulocytes, a kind of white blood cell)
  • Neutropenia (low levels of neutrophils, a type of white blood cell that fights infections)
  • Liver Enzyme Elevations
  • Zinc Deficiency


  • Women should be warned of the potential risk to the baby and advised to avoid pregnancy while taking this medication.
  • Check the concentration of serum ferritin once every two to three months in order to evaluate the effects on body iron storage.
  • Between using Deferiprone and any other prescription or supplement that contains polyvalent cations like iron, aluminum, or zinc, there should be at least a four-hour gap.
  • Patients who are known to have a hypersensitivity to deferiprone should not receive treatment with deferiprone because hypersensitivity reactions such as Henoch-Schonlein purpura, urticaria, and periorbital edema with skin rash have been reported.
  • Consult with your doctor if you are losing weight, facing unnecessary sweats, fever, stuffy nose, blurred vision, headache, etc.
  • Deferiprone can sometimes lower the count of white blood cells increasing the infection chances, avoid getting sick.

Comparison with other compounds

Deferiprone vs Deferoxamine

  • Deferiprone is an iron chelator that has the ability to remove intracellular iron from the heart more effectively than deferoxamine.
  • In addition, research indicates that long-term therapy with deferiprone has a greater cardioprotective effect against the toxicity of iron overload than subcutaneous deferoxamine. This means that intake of deferiprone reduces the side-effect to heart.
  • A study was conducted where, Deferiprone (75 mg/kg/day) was given to 71 patients with thalassemia major and deferoxamine (50
    mg/kg/day) to 73 patients with thalassemia major. Deferiprone was taken orally three times a day eight hours apart, whereas deferoxamine was administered subcutaneously five to seven days a week over eight to twelve hours. The deferiprone group had a serum ferritin reduction of 222 plus/- 783 ng/ml while the deferoxamine group had a reduction of 232 plus/- 619 ng/ml (P = 0.81).

Deferiprone vs Deferasirox

  • Both Deferiprone and Deferasirox are tridentate iron chelator and functions by forming a stable complex with iron, which is then eliminated from the body. However, the Deferiprone-iron complex is excreted through urine while the Deferasirox-iron complex is excreted via feces.
  • Deferiprone is a less expensive iron-chelator as compared to Deferasirox.
  • Deferiprone is much more superior in controlling or reducing myocardial iron load as compared to Deferasirox.


Q. How often do you take Deferiprone?

Deferiprone is given in two different forms:

  • Oral liquid dosage for the adults and children of 3 years of age and older depending on the body weight. Initially 25mg/kg 3 times a day.
  • Oral 1000mg tablets for the adults and children of 8 years and older depending on the body weight with 75mg/kg.

Q. How is Deferiprone excreted?

It is excreted in urine, with approximately 4% of single dose excreted in urine having heavy iron load.

Q. Who manufactures Deferiprone?

Macsen Labs is a GMP and ISO-certified manufacturer and supplier of high-quality Deferiprone.

Q. How much iron does deferiprone remove?

The efficacy of deferiprone is associated with a decrease in iron content of about 24.9% in the liver, but an increase in liver weight and water content.

Q. Is Deferiprone FDA approved?

Ferriprox, which is the brand name of the medication deferiprone, has received approval from the FDA, for the treatment of thalassemia, iron overload, and sickle cell anemia.


The information provided here is based on general knowledge, articles, research publications etc. and we do not claim the authenticity of any of the information provided above. We do not claim or suggest/advise any medical, therapeutic, health or nutritional benefits of Deferiprone. We do not supply or promote our Deferiprone product for the applications which are covered by valid patents and which are not approved by the FDA.

Macsen Labs is a manufacturer and supplier of high-quality Deferiprone.

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