Promising New Data for Patients with Iron Overload

Main Category: Pediatrics / Children's Health
Article Date: 19 May 2005 - 11:00 PDT

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Data presented at two major medical meetings this week demonstrate that investigational agent ICL670 (Exjade ™), a once daily oral iron chelator, has a well-tolerated safety profile in patients with thalassaemia and with myelodysplastic syndromes (MDS).

Thalassaemia Clinical data from the first ICL670 trial in 40 paediatric thalassaemia patients, presented today at the American Society of Pediatric Hematology/Oncology's (ASPH/O) Annual Meeting in Washington DC, indicates that ICL670 had no observed harmful effects on the sexual or physical development of children with thalassaemia1.

Elaine Miller, from the UK Thalassaemia Society, welcomed the data, saying, "These are interesting data which provide hope for a potential new chelator for Thalassaemia, a condition often currently treated with multiple lengthy sub-cutaneous infusions. We look forward to seeing further data." Thalassaemia affects mainly people of Mediterranean origin, but it also occurs in people with African, Asian and Arab origins. It is an inherited disorder, which in its more severe forms does not allow the body to produce enough healthy, mature red blood cells. Children usually become unwell at about six months and very often have reduced sexual and physical development thereafter. Untreated, the condition may become fatal.

Myelodysplastic Syndromes (MDS)

Data from a Phase II study in 47 MDS patients presented at the 8th International Symposium on MDS between May 12-15 in Nagasaki, Japan, indicated that ICL670 appears to be a convenient, effective and well-tolerated oral chelator for the treatment of iron overload in MDS patients2. MDS is a group of diseases where the production of blood cells by the bone marrow is disrupted. It can affect the production any type of, and sometimes all types, of blood cells. It is most commonly seen in adults with a higher proportion of men developing it. It affects about 3,250 people per year in the UK3. The current standard of care in iron chelation typically requires a subcutaneous infusion lasting eight to 12 hours per day for five to seven days a week, for as long as the patient continues to receive transfusions. For many patients, the need for transfusion and chelation therapy is life-long.

References

1 Piga A et al. Growth and Sexual Development in pediatric patients during one year of treatment with the once-daily, iron chelator ICL670. Pediatric Academic Societies 2005 Annual Meeting. May 2005 2 Gatterman N et al. The efficacy and tolerability of ICL670, a once-daily iron chelator, in patients with myelodysplastic syndrome (MDS) and iron overload. 8th International Symposium on myelodysplastic syndrome. Japan. May 2005 3 Leukaemia Research Fund. lrf.org.uk/en/1/dismdshome.html

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For further information please contact:
Joanna Lewis Novartis Pharmaceuticals UK Ltd Tel: 01276 698691
Email: joanna.lewis@novartis.com
Georgina Pinnington Ruder Finn UK Ltd Tel: 0207 462 8900
Email: gpinnington@ruderfinn.co.uk

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