Swedish Orphan Biovitrum distributes this product in the UK and Ireland, in Spain, Portugal, Malta, Switzerland, France, Belgium, Luxembourg, the Netherlands, Denmark, Norway, Sweden, Finland, Estonia, Latvia, Lithuania and Algeria.
What is Ferriprox?
Ferriprox is a medicine containing the active substance deferiprone. It is available as white, capsule- shaped tablets (500 mg) and as an oral solution (100 mg/ml).
What is Ferriprox used for?
Ferriprox is used to treat iron overload (an excess of iron in the body) in patients with thalassaemia major (an inherited disease where patients are unable to make enough haemoglobin, the protein found in red blood cells that carries oxygen around the body). Ferriprox is used when deferoxamine (another medicine used to treat iron overload) cannot be used. The medicine can only be obtained with a prescription.
How is Ferriprox used?
Ferriprox treatment should be started and maintained by a doctor who has experience in the treatment of patients with thalassaemia. The total daily dose of Ferriprox is 75 -100 mg per kilogram body weight given in three separate doses per day. If tablets are used, the number of tablets for each dose is adjusted to the nearest half tablet. If the oral solution is used, the dose is adjusted to the nearest 2.5 ml. For example, a 70 kg patient will take three and a half tablets or 17.5 ml three times a day. Doses above 100 mg/kg per day are not recommended because of an potentially increased risk of side effects. Patients taking Ferriprox, or their carers, must be given a reminder card that reminds the patient how to take the medicine safely.
How does Ferriprox work?
Patients with thalassaemia major need frequent blood transfusions. When patients receive repeated transfusions, the transfused red cells bring iron. However, the body does not have a natural way of removing excess iron, so it builds up. Over time, the excess iron can damage important organs such as the heart or liver. The active substance in Ferriprox, deferiprone, is an ‘iron chelator’. It binds to iron in the body to form a compound (a ‘chelate’) that can be excreted by the body, mainly in the urine, and to a lesser extent in the faeces (stools). This helps to correct the iron overload and prevent damage due to excess iron.
How has Ferriprox been studied?
Ferriprox was originally studied in three studies involving 247 patients over 10 years of age with thalassaemia major, with the main study comparing the effectiveness of Ferriprox with that of deferoxamine (the standard treatment to remove iron) in 71 patients over two years. The study was an ‘open label’ study, meaning that the doctor and patients knew which medicine they were using, because Ferriprox is given by mouth, whereas deferoxamine is given by subcutaneous infusion (a very slow injection under the skin) overnight. A later study compared treatment alternating Ferriprox and deferoxamine (five days’ Ferriprox plus two days’ deferoxamine each week) with continuous treatment with deferoxamine on its own, in 60 patients over 12 months. In all studies, effectiveness was measured by looking at the levels of ferritin in the blood before and during treatment. Ferritin is a protein that stores iron in the body. The serum ferritin level (the amount of ferritin in the blood) is indicative of the amount of iron stored in the body.
What benefit has Ferriprox shown during the studies?
In the initial comparative study, the mean serum ferritin levels were not significantly different in the two treatment groups, but average iron concentration in the liver of Ferriprox-treated patients seemed to increase more than in deferoxamine-treated patients. In the alternating treatment study, the treatment schedule combining Ferriprox for five days with deferoxamine for two days reduced ferritin levels to the same extent as deferoxamine taken on its own. However, the number of patients in the study was not sufficient to prove that such a schedule is as effective as deferoxamine taken on its own.
What is the risk associated with Ferriprox?
The most common side effects with Ferriprox (seen in more than 1 patient in 10) are reddish/brown urine (showing that iron is being excreted), nausea (feeling sick), abdominal (tummy) pain and vomiting. An uncommon but serious side effect is agranulocytosis (very low levels of a type of white blood cell). Ferriprox should not be used in people who may be hypersensitive (allergic) to deferiprone or any of the other ingredients. Ferriprox should not be used in people who have had neutropenia (low levels of a type of white blood cell) repeatedly or agranulocytosis. Ferriprox should also not be used with medicines that might cause neutropenia or agranulocytosis. Ferriprox should not be used in women who are pregnant or breast-feeding.
Why has Ferriprox been approved?
The Committee for Medicinal Products for Human Use (CHMP) decided that Ferriprox’s benefits are greater than its risks for the treatment of iron overload in patients with thalassaemia major when deferoxamine therapy is contraindicated or inadequate. They recommended that Ferriprox be given marketing authorisation. Ferriprox was originally authorised under ‘Exceptional Circumstances’, because, as the disease is rare, limited information was available at the time of approval. As the company had supplied the additional information requested, the ‘Exceptional Circumstances’ ended on 12 April 2002.
Other information about Ferriprox:
The European Commission granted a marketing authorisation valid throughout the European Union for Ferriprox on 25 August 1999. The marketing authorisation was renewed on 25 August 2004. The marketing authorisation holder is Apotex Europe B.V.