Iron biology
Medical management of iron
The medical management of iron to date has been too simplistic given the tools available, but our current and growing understanding of the nature of iron dysregulation and how it is at the core of various diseases has highlighted the large unmet need for more advanced iron therapeutics.
1968
In 1968, Deferoxamine (Desferal; Novartis) was approved for subcutaneous, intramuscular, or intravenous injections for treating acute iron intoxication and chronic iron overload due to excess body iron loading from repeated blood transfusions needed to treat transfusion-dependent anemia. The body has no excretion route for excess body iron loads. Deferoxamine provides an excretion route for iron and several generic forms of deferoxamine have been approved.
2005
In 2005, Deferasirox (Exjade; Novartis), was approved as a tablet for oral suspension and is indicated for the treatment of chronic iron overload due to blood transfusions in patients aged 2 years and older. Under the accelerated approval program, the FDA expanded the indications for deferasirox in 2013 to include the treatment of patients age 10 and older with chronic iron overload due to non-transfusion-dependent thalassemia syndromes and specific liver iron concentration and serum ferritin levels. In 2015, an oral tablet formulation for deferasirox (Jadenu™) was approved by FDA. All formulations of deferasirox carry a black box warning because it may cause serious and fatal renal toxicity and failure, hepatic toxicity and failure, and gastrointestinal hemorrhage. As a result, treatment with deferasirox requires close patient monitoring, including laboratory tests of renal and hepatic function.
2011
In 2011, the iron chelator Deferiprone (Ferriprox®) was approved for the treatment of patients with transfusional overload due to thalassemia syndromes when other chelation therapy is inadequate. Deferiprone is available in tablet and oral solution. Ferriprox® carries a black box warning because it can cause agranulocytosis that can lead to serious infections and death. As a result, absolute neutrophil count should be monitored before and during treatment. In a June 2014 warning to consumers, FDA advised that FDA-approved chelating agents are available by prescription only. There are no FDA-approved over-the-counter chelation products.
These agents are toxic and though they have been investigated as potential infectious disease agents, they have shown poor potential, sometimes acting to deliver iron from within cells to bacteria or fungi, worsening infections.
Our Science
Medicine needs a new approach to iron management.