Deferoxamine
[CAS# 70-51-9]

Identification

• Classification: API >> Special medicine >> Antidote
• Name: Deferoxamine
• Synonyms: N'-[5-[[4-[[5-(Acetylhydroxyamino)pentyl]amino]-1,4-dioxobutyl]hydroxyamino]pentyl]-N-(5-aminopentyl)-N-hydroxysuccinamide
• Molecular Formula: C₂₅H₄₈N₆O₈
• Molecular Weight: 560.68
• CAS Registry Number: 70-51-9
• EC Number: 200-738-5
• SMILES: CC(=O)N(CCCCCNC(=O)CCC(=O)N(CCCCCNC(=O)CCC(=O)N(CCCCCN)O)O)O

Properties

• Density: 1.2±0.1 g/cm³ Calc.^*
• Index of refraction: 1.537 (Calc.)^*

^* Calculated using Advanced Chemistry Development (ACD/Labs) Software.

Safety Data

• Hazard Symbols: GHS07 Warning
• Risk Statements: H302
• Safety Statements: P264-P270-P301+P317-P330-P501

Hazard Classification

Hazard	Class	Category Code	Hazard Statement
Acute toxicity	Acute Tox.	4	H302
Skin sensitization	Skin Sens.	1	H317

Discovery and Applications

Deferoxamine is a natural siderophore and a clinically important iron-chelating agent used primarily to treat iron overload conditions. It was originally isolated from the bacterium Streptomyces pilosus and later developed for medical applications due to its strong affinity for ferric iron (Fe³⁺).

The compound is a trihydroxamic acid that forms highly stable complexes with ferric ions, effectively binding and removing excess iron from the body. Deferoxamine’s molecular structure enables it to selectively chelate iron, preventing iron-catalyzed formation of harmful free radicals that contribute to tissue damage in conditions of iron overload.

Clinically, deferoxamine is used to manage acute and chronic iron overload resulting from repeated blood transfusions in patients with diseases such as thalassemia major, sickle cell anemia, and other chronic anemias. Iron overload can cause severe damage to organs including the liver, heart, and endocrine glands. Deferoxamine binds excess iron in plasma and tissues, facilitating its excretion primarily through the urine and bile.

The development of deferoxamine as a therapeutic agent represented a major advance in the treatment of iron toxicity. Prior to its use, options for managing iron overload were limited, and patients faced progressive organ damage and reduced life expectancy. Deferoxamine therapy significantly improves clinical outcomes by reducing iron burden and associated complications.

In addition to its clinical use, deferoxamine is applied in laboratory research to study iron metabolism and oxidative stress. Its iron-binding properties make it a useful tool to manipulate iron availability in cell culture and animal models.

Deferoxamine is typically administered parenterally, either by subcutaneous infusion or intramuscular injection, due to poor oral bioavailability. Its safety profile includes potential side effects such as injection site reactions, allergic responses, and, rarely, auditory and ocular toxicity when used in high doses or prolonged therapy.

In summary, deferoxamine is a natural siderophore developed into an essential iron-chelating drug used to treat iron overload disorders. Its strong and selective binding to ferric iron underlies its therapeutic efficacy, improving the prognosis of patients with transfusional hemosiderosis and other iron accumulation diseases.

References

1979. Effects of Desferrioxamine on 67Ga Distributions in Tumor-Bearing Mice. Proceedings of the Society for Experimental Biology and Medicine, 161(4).
DOI: 10.3181/00379727-161-40561

1990. Comparison of oral iron chelator L1 and desferrioxamine in iron-loaded patients. Lancet, 336(8726).
DOI: 10.1016/0140-6736(90)92962-h

1991. Regulation of ferritin and heme oxygenase synthesis in rat fibroblasts by different forms of iron. Proceedings of the National Academy of Sciences of the United States of America, 88(3).
DOI: 10.1073/pnas.88.3.688