What is Ferriprox?
Ferriprox is a medicine that contains 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 indicated for the treatment of iron accumulation (an excess of iron in the body) in patients with thalassemia major. It is an inherited disease in which patients are unable to produce a sufficient amount of hemoglobin, the protein contained in red blood cells that carries oxygen into the body. Ferriprox is used when deferoxamine therapy (the standard therapy for iron accumulation) is contraindicated or inadequate.
The medicine can only be obtained with a prescription.
How is Ferriprox used?
Ferriprox therapy should be started and maintained by a doctor who has experience in the treatment of thalassemia patients.
The total daily dose of Ferriprox is 75-100 mg per kilogram of body weight, divided into three separate doses. If tablets are used, the number of tablets per dose should be calculated by rounding to half a tablet. If the oral solution is used, the dose should be rounded to 2.5 ml. For example, a patient weighing 70 kg will have to take three and a half tablets or 17.5 ml of oral solution three times a day. Doses above 100 mg / kg per day are not recommended due to the potential increased risk of side effects.
Patients taking Ferriprox, or their carers, must be given a memorandum to remind the patient how to take the drug safely.
How does Ferriprox work?
Patients with thalassemia major must undergo frequent blood transfusions. When patients receive repeated transfusions, the transfused red blood cells bring iron into the body, which however does not have a natural way to remove excess iron, which therefore tends to accumulate. Over time, excess iron can damage important organs such as the heart or liver. The active ingredient in Ferriprox, deferiprone, is an "iron chelator". It binds to the iron present in the body to form a compound that can be excreted by the body, mainly through the urine, and to a lesser extent through the faeces. This helps correct iron overload and prevent damage due to excess iron.
How has Ferriprox been studied?
Ferriprox was initially investigated in three studies involving 247 patients over the age of 10 with thalassemia major. In the main study the effectiveness of Ferriprox was compared over two years to that of deferoxamine in 71 patients. The study was "open-ended", meaning the doctor and patients knew which drug they were using, as Ferriprox is taken by mouth, while deferoxamine is given by subcutaneous infusion (an injection done very slowly under the skin) all night long. In a subsequent study the alternate use of Ferriprox and deferoxamine (every week Ferriprox was given for five days and deferoxamine for two days) was compared in 60 patients with continuous treatment with deferoxamine monotherapy (alone), over a period of 12 months.
In all the studies, the main measure of effectiveness was the change in ferritin levels in the blood. Ferritin is a protein that stores iron in the body. The level of ferritin in the blood indicates the amount of iron accumulated in the body.
What benefit has Ferriprox shown during the studies?
In the initial study comparing Ferriprox with deferoxamine, mean serum ferritin levels were similar in the two treated groups. However, the average iron concentration in the liver of patients treated with Ferriprox appeared to increase more than that in patients treated with deferoxamine.
In the alternate treatment study, the dosing regimen that included the combination of Ferriprox for five days with deferoxamine for two days showed the same reduction in the blood ferritin levels of deferoxamine monotherapy treatment. However, the number of patients who participated in the study was too small to show whether this regimen is as effective as deferoxamine therapy alone or not.
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 (a sign that iron is being excreted), nausea, abdominal pain (stomach ache) and vomiting. An uncommon but serious side effect is agranulocytosis (very low concentrations of granulocytes, 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 by people who have suffered repeated episodes of neutropenia (decreased number of neutrophils, a type of white blood cell) or who have suffered from agranulocytosis. Ferriprox should also not be taken with medicines that could cause neutropenia or agranulocytosis. The medicine should not be used during pregnancy or during breast-feeding.
Why has Ferriprox been approved?
The Committee for Medicinal Products for Human Use (CHMP) has determined that Ferriprox's benefits are greater than its risks for the treatment of iron overload in patients with thalassemia major when deferoxamine therapy is contraindicated or inadequate and has therefore recommended granting of the marketing authorization for Ferriprox.
Ferriprox was initially authorized in "exceptional circumstances" because incomplete information on the medicine was available at the time of approval for treatment of a rare disease. Since the pharmaceutical company provided the additional information requested, the condition referred to "exceptional circumstances" was removed on 12 April 2002.
Other information on Ferriprox:
On 25 August 1999, the European Commission issued a marketing authorization for Ferriprox, valid throughout the European Union. The marketing authorization was renewed on 25 August 2004 and 25 August 2009. The marketing authorization holder is Apotex Europe BV
The full EPAR for Ferriprox can be found here.
Last update of this summary: 08-2009.
