Two and a half year efficacy and safety data in a wide range of underlying diseases in patients as young as two years confirm initial findings.
Additional results from ESCALATOR trial show once daily dosing of Exjade provides sustained 24 hour removal of toxic excess iron
Patients in the studies included those with sickle cell disease, myelodysplastic syndromes, thalassemia, and other rare blood disorders
Amman, Jordan: New long term efficacy and safety data indicate that once-daily Exjade (deferasirox) continues to reduce total body iron over 2.5 years of treatment in a range of patients with chronic iron overload due to blood transfusions. The data further support the use of Exjade in patients who may need life-long iron chelation therapy.
The analysis, reported at the 48th American Society of Hematology meeting in Orlando, Florida also found that the safety and tolerability profiles of patients being treated with Exjade are consistent with one-year observations.
"These long-term data are important because they confirm that the safety and efficacy profile of Exjade is similar to that seen at one year," said Maria Cappellini, MD, University di Milano, Milan, Italy, who led the long-term safety analysis. " We now also have confirmation that for patients who need ongoing blood transfusions, the dosing of Exjade can be adjusted up or down -- either to achieve iron reduction where there is a high iron burden, or to maintain iron levels within a target range. "
Additional research findings presented at the meeting demonstrate that one year of treatment with once-daily Exjade produced a sustained reduction in levels of labile plasma iron (LPI). LPI is a type of freely circulating iron believed to be responsible for damage to key organs such as the liver and the heart. This sub-study from the ESCALATOR trial suggests that since LPI is produced 24 hours a day, the constant presence of Exjade may help avoid this type of damaging iron from accumulating. Exjade is the only oral iron chelator to provide continuous 24-hour chelation coverage with once-daily dosing.
Iron overload is a cumulative and potentially life threatening condition that results from frequent blood transfusions required to treat certain types of rare chronic blood disorders, including sickle cell disease, thalassemia, and myelodysplastic syndromes. Signs of iron overload may be detected after as few as ten blood transfusions (20 units of blood). If left undiagnosed or untreated, the excess iron in the body may eventually lead to organ damage. The body has no inherent mechanism to remove excess iron, so iron chelation is used as an effective treatment for transfusion-related iron overload.
Exjade was developed to meet the high unmet need for an oral iron chelator despite the availability of deferoxamine (Desferal). While effective, compliance with deferoxamine is poor due to its burdensome administration by needle and pump, often lasting 8 to 12 hours per night 5 to 7 nights a week.
"Exjade has transformed the treatment of iron overload for many adults and children around the world by offering an effective once-daily oral iron chelator," said Diane Young, MD, Vice President, Head, Global Medical Affairs, Novartis Oncology. "These longer-term results strengthen our belief that Exjade will quickly become the new standard of care in Iron chelation around the world."
Study Details
The long term efficacy and safety studies are from the first analysis of the extension phase of five core trials that led to the approval of Exjade in the US, the EU, and other countries. More than 1,000 patients - approximately 40% of whom were between the ages of 2 and 16 at enrollment - have now been followed for a median of 2.5 years. The type and severity of adverse events were consistent with those observed in the core trials.
A separate analysis from the extension phase followed approximately 400 pediatric patients with a variety of underlying diseases. Follow-up of 2.5 years of treatment revealed that once-daily doses of 20-30 mg/kg/day of Exjade led to clinically relevant and continuous reductions in iron burden, as measured by serum ferritin.
Another analysis from the extension phase of two core trials found that serum ferritin steadily decreased following dose increases in regularly transfused patients who initially received Exjade doses of 5 to 10 mg/kg/day. This analysis confirmed that 30 mg/kg/day effectively reduced body iron over the long term, whereas 20 mg/kg/day was effective in maintaining body iron levels in regularly transfused patients. Therefore, Exjade dosing can be tailored accordingly, depending on the goal of therapy and transfusion burden.
In the ESCALATOR sub-study, 14 patients with high baseline iron burdens received 20-30 mg/kg/day and were followed for one year. After four weeks, levels of LPI were close to normal, within the normal range by week 16 and therefore for the entire study period. The decreases in LPI were accompanied by decreases in liver iron concentration and serum ferritin, indicating effective iron removal.
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About Exjade
Now approved in more than 70 countries, including the US and the EU, Exjade is the first once-daily oral iron chelator approved for use in patients with transfusional iron overload and who have a wide range of underlying diseases such as sickle cell disease, thalassemia, and myelodysplastic syndromes. Exjade is administered as a drink after the tablets are dispersed in a glass of water, apple, or orange juice.
© Press Release 2007
