Novartis today welcomed the Scottish Medicines Consortium’s acceptance of deferasirox (Exjade®) for use within NHS Scotland for the treatment of iron overload associated with the treatment of rare acquired or inherited anaemias. The Scottish Medicines Consortium (SMC) provides advice to the NHS in Scotland about the use of all newly licensed medicines.
Exjade is the first and only once-daily oral iron chelator for the treatment of iron overload in patients needing frequent blood transfusions for genetic blood disorders such as thalassaemia, sickle cell disease and other rare anaemias.1 Iron chelation is necessary to prevent the potentially life-threatening complications of excess iron in the body which can cause damage to the heart, liver and endocrine glands if left untreated.2
Prior to the approval of Exjade, the most common way of performing iron chelation was a painful nightly infusion by needle and pump that can last from eight to 12 hours every night for five to seven nights a week. As a result of the pain and inconvenience, many patients stop or avoid iron chelation therapy, thus risking the toxic effects of iron overload.3
“Exjade is effective and well tolerated across a range of conditions where chronic transfusions result in iron overload,” said Professor John Porter, Consultant Haematologist, University College London Hospitals. “Exjade offers a much needed alternative to patients who find traditional iron chelation therapy unacceptable. The SMC decision is an important indicator that this treatment should be made available to all patients who need it.”
Exjade was launched in the UK in September 2006 but since launch, patients in some areas have been having difficulty gaining access to treatment.
“As there are no current plans for NICE to review Exjade, patients in some areas of the UK are having difficulty accessing this new therapy,” said Mike Michael, President of the United Kingdom Thalassaemia Society. “Recently issued guidance from the Department of Health states it is not acceptable to cite a lack of NICE guidance as a reason for not providing treatment and points to other sources such as the SMC that should be used to make an informed decision. We hope this positive SMC decision sends a clear signal to local funding bodies to provide this much needed therapy.”
Sobia Afridi, from Oxford, whose daughter Sabrena has thalassaemia said: “My local Primary Care Trust has so far denied my three-year old daughter access to Exjade, so she has to suffer painful nightly injections which are a traumatic experience for the whole family. I hope this decision by the SMC will encourage PCTs to enable patients like Sabrena to receive this potentially life changing treatment.”
Exjade is approved in the EU for the treatment of chronic iron overload due to frequent blood transfusions in patients age six and older with beta thalassaemia major. It is also indicated in the EU for the treatment of chronic iron overload when desferrioxamine is contraindicated or considered inadequate in patients with other anaemias, children aged two to five years and patients with beta thalassaemia major with iron overload due to infrequent blood transfusions.
The SMC did not recommend the use of Exjade for patients with myelodysplastic syndromes and Novartis will be resubmitting an application to the SMC with additional data to support the use of Exjade in this patient population.
New data published to further support Exjade use in sickle cell disease
New data has recently been published in the British Journal of Haematology that further supports the use of Exjade in sickle cell patients. A randomised comparison of Exjade versus desferrioxamine (the current standard iron chelator) for the treatment of transfusional iron overload in sickle cell disease found that over one year, once-daily oral deferasirox has acceptable tolerability and appears to have similar efficacy to desferrioxamine in reducing iron burden in transfused patients with sickle cell disease.4
The authors concluded that:
“When combined with appropriate laboratory monitoring, the availability of deferasirox as a once-daily, oral option for safe and effective chelation therapy has the potential to prevent complications of iron overload. In paediatric and adult patients with sickle cell disease, this should consequently help prevent serious complications, such as stroke and organ failure, by facilitating the appropriate use of blood transfusions.”4
Iron Overload
Iron overload is a cumulative, potentially life threatening and unavoidable consequence of frequent blood transfusions used to treat certain chronic blood disorders such as thalassaemia, sickle cell disease and myelodysplastic syndromes (MDS).
As excess iron is highly toxic, the human body has a sophisticated mechanism to balance dietary intake and natural loss in order to maintain the proper concentration of iron in the blood which meets the body’s requirements.5 The body absorbs iron when we eat food. Transport proteins – called transferrin and ferritin – then move the absorbed iron through the body before incorporating it into haemoglobin.5
However, while the human body has a complex mechanism to absorb iron, it has no means to excrete excess iron.5 The only way to decrease iron overload is to remove it physically or by a process called chelation.6
Chelating agents bind iron, and the chelated iron is excreted from the body, preventing the toxic build-up of excess iron associated with transfusion therapy.7
The standard chelating agent is typically administered as a nightly infusion by needle and pump, often lasting eight to 12 hours per night for five to seven nights per week. As a result, many patients stop or avoid iron chelation, exposing themselves to the dangers of iron overload.8
Thalassaemia
Thalassaemia refers to a group of genetic blood disorders that affect the body’s production of haemoglobin, the oxygen-carrying component of the red blood cells.
Sickle Cell Disease
Sickle cell disease is an inherited blood disorder whereby the red blood cells become deformed (‘sickle’ shaped) and tend to get stuck in small blood vessels. This blockage can cause pain, damage tissue and organs and lead to stroke if it occurs in the brain.
Advice from the Scottish Medicines Consortium
The full advice is available on the SMC website:
scottishmedicines
References
1. Exjade summary of product characteristics, Novartis, August 2006
2. Kushner JP, Porter JP, Olivieri NF. Secondary Iron Overload. Hematology (Am Soc Hematol Educ Program) 2001;:47-61
3. Nisbet-Brown E, et al. Effectiveness and safety of ICL670 in iron overloaded patients with thalassaemia; a randomised, double-blind placebo controlled, dose escalation trial. Lancet. 2003, May 10; 361(9369): 1597-602
4. Vichinsky E, et al. A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. British Journal of Haematology. 2006; 136: 501-508
5. Iron Absorption. Last retrieved on 15 January 2007 from sickle.bwh.harvard/iron_absorption.html
6. Porter J. A risk-benefit assessment of iron-chelation therapy. Drug Saf. 1997 Dec; 17(6):407-21
7. Exjade: Basic Prescribing Information. Novartis. August 2006
8. About Thalassaemia. Cooley’s Anemia Foundation. Last retrieved on 15 January 2007 from thalassaemia/sections.php?sec=1
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