Exjade

Exjade can only be obtained with a prescription and treatment should be started and supervised by a doctor who is experienced in the treatment of chronic iron overload.

It is available as film-coated tablets, dispersible tablets and granules. The film-coated tablets are to be swallowed with water while the dispersible tablets are to be mixed with a liquid to make up a suspension that the patient can drink. The granules are sprinkled on soft food such as yogurt or apple sauce that the patient can eat.

The starting dose of Exjade depends on the patient’s body weight, on which form of the medicine is being taken, what the medicine is used for, and on the level of iron overload. The dose is then adjusted as needed, every 3 to 6 months, according to the iron levels in the blood.

Exjade is taken once a day at around the same time. The dispersible tablets should be taken on an empty stomach (at least 30 minutes before food), and the film-coated tablets and granules can be taken on an empty stomach or with a light meal.

For more information about using Exjade, see the package leaflet or contact your doctor or pharmacist.

The body cannot remove iron effectively and excess iron can cause damage. The active substance in Exjade, deferasirox, is an ‘iron chelator’. It attaches to excess iron in the body to form a compound called a ‘chelate’ that can be removed by the body, mainly in the stool. This helps to correct the iron overload and prevent damage to organs such as the heart or liver from excess iron.

In chronic iron overload due to blood transfusions, one main study compared Exjade with deferoxamine in 591 patients with beta thalassaemia major. About half of the patients were under the age of 16 years, and 56 were less than 6 years old. Effectiveness was determined by the level of iron in the liver before and after one year of treatment. The iron level was reduced satisfactorily in 53% of the patients receiving Exjade, compared with 66% of the patients receiving deferoxamine. Exjade was not as effective overall as the comparator medicine. However, in the 381 patients who had particularly high levels of iron at the beginning of the study and who received comparable amounts of Exjade and deferoxamine, the two medicines were as effective as each other.

Another study involved 184 patients who could not be treated with deferoxamine, including patients with beta thalassaemia major and with other types of anaemia. In more than half of these patients the iron level was reduced satisfactorily after a year of treatment with Exjade, including patients aged between 2 and 5 years.

In a further main study involving 166 patients from 10 years of age (including 21 patients aged 10 to 18 years) with non-transfusion-dependent thalassaemia syndromes and iron overload, Exjade was more effective than placebo (a dummy treatment). The main measure of effectiveness was the change in iron levels in the liver after 12 months of treatment. In patients treated with Exjade, liver iron levels decreased on average by 3.8 mg per gram of liver compared with an average increase of 0.4 mg per gram of liver in patients treated with placebo.

The most common side effect with Exjade (which may affect more than 1 in 10 people) is increased blood creatinine (a marker of kidney problems). Other common side effects (in up to 1 patient in 10) include nausea (feeling sick), vomiting, diarrhoea, indigestion, abdominal (belly) pain, constipation, headache, rash, itching, blood tests showing raised transaminases (which may suggest liver damage) and protein in urine. For the full list of side effects of Exjade, see the package leaflet.

Cases of kidney and liver failure have been reported with Exjade. Kidney and liver tests should be done before starting treatment and at regular intervals during treatment with Exjade. Treatment may need to be interrupted or stopped depending on the results of the tests.

Cases of metabolic acidosis (when the body produces more acid than it gets rid of) have been reported with Exjade, mostly in patients with kidney impairment. Interruption of treatment with Exjade should be considered in patients who develop this condition.

Exjade must not be used in people whose creatinine clearance is less than 60 ml per minute. It must not be used in combination with other iron chelators. For the full list of restrictions, see the package leaflet.

The European Medicines Agency decided that Exjade’s benefits are greater than its risks and it can be authorised for use in the EU.

The company that markets Exjade must issue an education pack to healthcare professionals. This pack aims to inform them about the treatment recommendations with Exjade, including choosing the right dose, that the doses are different for dispersible tablets compared to film-coated tablets or granules, and the need to monitor the patient’s health, especially kidney function. The company will also prepare a similar pack for patients.

The company will also perform the following studies: a study on the long-term effects of the film-coated tablets and dispersible tablets in children aged over 10 years with non-transfusion-dependent thalassaemia; and a study to assess the safety of the film-coated tablets (particularly when they are crushed) in children.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Exjade have also been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Exjade are continuously monitored. Side effects reported with Exjade are carefully evaluated and any necessary action taken to protect patients.

Exjade received a marketing authorisation valid throughout the EU on 28 August 2006