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Exjade: FDA Panel recommends approval for once-daily oral treatment of chronic iron overload due to blood transfusions


Blood Products Advisory Committee ( BPAC ) of the FDA ( Food and Drug Administration ) has given a positive review of Exjade ( Deferasirox ).

Exjade is once-daily oral iron chelator for the treatment of chronic iron overload due to blood transfusions in adults and children.

The Committee unanimously voted to recommend approval for use of Exjade for patients with chronic iron overload due to blood transfusions.

Iron overload is a cumulative, potentially life-threatening, unavoidable consequence of frequent blood transfusions used to treat certain rare, chronic blood disorders, including thalassemia, sickle cell disease, other rare anemias and myelodysplastic syndromes.
Signs of iron overload may be detected after 10 to 20 blood transfusions.
If left undiagnosed or untreated, the excess iron in the body leads to damage to the liver, heart and endocrine glands.
Iron chelation has been demonstrated to be the only effective treatment for transfusion-related iron overload.

The Exjade filings were based on the results of a pivotal clinical trials program, including a Phase III head-to-head trial vs. Desferal ( Deferoxamine ), which showed that Exjade significantly reduced liver iron concentration ( LIC ) at doses of 20-30 mg/kg/day.
These clinical trials, which included more than 1,000 adults and children, were part of the largest prospective global clinical trials program ever implemented for an investigational iron chelator.
LIC is the accepted indicator for body iron content in patients receiving blood transfusions.

The studies demonstrated that Exjade at 20-30 mg/kg/day led to the maintenance or reduction of iron burden in transfused patients with thalassemia, sickle cell disease, other rare anemias and myelodysplastic syndromes. In the clinical studies, Exjade was generally well tolerated, with the most frequently reported adverse events being nausea, vomiting, diarrhea, abdominal pain, skin rash and increases in serum creatinine. As with Deferoxamine, cases of ocular and auditory disturbances have been reported.

In iron chelation an agent binds to iron in the body and helps remove it through the urine and/or feces.
To date, only Deferoxamine is globally available for the first-line treatment of transfusional iron overload.
While Deferoxamine is effective, it typically requires subcutaneous infusion lasting eight to twelve hours per day, for five to seven days a week for as long as the patient continues to receive blood transfusions or has excess iron within their body.
In many patients the need for transfusions may be life-long.
However, due to the inconvenience and discomfort associated with the administration of Deferoxamine, many patients choose not to undergo iron chelation therapy, exposing themselves to the dangers of iron overload.

Source: Novartis, 2005


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