Soliris - eculizumab
What is Soliris?
Soliris is a concentrate to be made up into a solution for infusion. Contains the active ingredient eculizumab.
What is Soliris used for?
Soliris is indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening genetic disease that causes red blood cells to be broken down (destroyed) too quickly. The consequences are anemia (decrease in the number of red blood cells), thrombosis (formation of clots in the blood vessels) and dark urine.
Because the number of patients with PNH is low, the disease is considered 'rare', and Soliris was designated an 'orphan medicine' (a medicine used in rare diseases) on 17 October 2003.
The medicine can only be obtained with a prescription.
How is Soliris used?
Soliris must be administered by a healthcare professional, such as a doctor or nurse, under the supervision of a physician experienced in the treatment of patients with blood disorders.
Treatment with Soliris consists of an infusion (drip into a vein) of 600 mg of product for 25-45 minutes once a week, followed by a dose of 900 mg during the fifth week. Thereafter the dose should be maintained at 900 mg, given approximately every two weeks. At least two weeks before starting Soliris treatment, all patients should be vaccinated against meningitis caused by the bacterium. Neisseria meningitidis and must be revaccinated in accordance with current guidelines.
Patients receiving Soliris must be given a special card explaining the symptoms of certain types of infections, with the warning to seek immediate medical attention if such symptoms occur.
How does Soliris work?
The active substance in Soliris, eculizumab, is a monoclonal antibody. A monoclonal antibody is an antibody (a type of protein) designed to recognize and attach to a specific structure (the antigen) in the body. Eculizumab was designed to bind to complement protein C5, a part of the body's defense system called "complement." In PNH, patients have a defect in the CD59 protein located on the surface of red blood cells, which normally prevents complement from attack the cells. As a consequence of this defect, complement destroys red blood cells. By blocking the complement protein C5, eculizumab prevents complement from attacking cells, reducing cell lysis and alleviating disease symptoms.
How has Soliris been studied?
The effects of Soliris were first tested in experimental models before being studied in humans.
Soliris was studied in one main study involving 88 adults with PNH who had received at least four transfusions for anemia in the previous year. Soliris was compared with placebo (a dummy treatment). The main measures of effectiveness were the number of patients. patients whose levels of hemoglobin (a protein found in red blood cells) remained above the individual target level and the number of red blood cell transfusions required during the first 26 weeks of treatment.
What benefit has Soliris shown during the studies?
Soliris was more effective than placebo in improving PNH symptoms. In the main study, 49% of patients (21 out of 43) treated with Soliris had stable hemoglobin levels and, on average, did not need transfusions. In comparison, none of the 44 patients who took placebo had stable hemoglobin levels and they needed, on average, 10 transfusions.
What is the risk associated with Soliris?
The most common side effect with Soliris (seen in more than 1 in 10 patients) is headache. For the full list of side effects reported with Soliris, see the package leaflet.
Soliris must not be used in people who may be hypersensitive (allergic) to eculizumab, mouse proteins or any of the other substances, or who have or are suspected to have inherited complement deficiencies. Due to the increased risk of meningitis, Soliris should not be given to people infected with Neisseria meningitidis or who have not been vaccinated against this bacterium.
Why has Soliris been approved?
The Committee for Medicinal Products for Human Use (CHMP) decided that Soliris's benefits are greater than its risks for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), but noted that the evidence for the benefit of Soliris is limited to individuals. who have previously undergone blood transfusions. The committee recommended the granting of a marketing authorization for Soliris.
What measures are being taken to ensure the safe use of Soliris?
The Soliris company will agree on the details of a system in each Member State which ensures that the drug is only distributed after verifying that the patient has been properly vaccinated. It will also provide prescribers and patients with information about the safety of the medicine and verify that prescribers use it safely.
More information about Soliris
On 20 June 2007, the European Commission granted Alexion Europe SAS a "Marketing Authorization" for Soliris, valid throughout the European Union.
For the summary of the opinion of the Committee for Orphan Medicinal Products for Soliris click here.
For the complete version of the Soliris EPAR click here.
Last update of this summary: 04-2009
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