Soliris - eculizumab

What is Soliris?

Soliris is a concentrate that is made up into a solution for infusion. It contains the active ingredient eculizumab.

What is Soliris used for?

Soliris is indicated in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare genetic disease that is life-threatening to the patient, causing the lysis (destruction) of red blood cells too fast. The consequences are anemia (decrease in the number of red blood cells), thrombosis (clot formation in 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 should be given by a healthcare professional, such as a doctor or nurse, under the supervision of a doctor who is experienced in treating patients with blood disorders.

Soliris treatment consists of an infusion (a 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, administered 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 should be revaccinated according to current guidelines.

Patients receiving Soliris must be given a particular card that explains the symptoms of certain types of infections, with the warning to seek medical attention immediately if these 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 bind to a specific structure (the antigen) in the body. Eculizumab was designed to bind to the complement protein C5, a part of the body's defense system called "complement". In the EPN, patients have a defect in the CD59 protein located on the surface of red blood cells, which normally prevents the complement from attacking cells. As a result of this defect, the complement destroys red blood cells. By blocking the C5 protein from the complement, eculizumab prevents the complement from attacking the cells, reducing lysis and relieving the symptoms of the disease.

What studies have been carried out on Soliris?

The effects of Soliris were first tested in experimental models before being studied in humans.

Soliris has been studied in one main study involving 88 adults with PNH who had undergone at least four transfusions for anemia in the previous year. Soliris was compared with a placebo (a dummy treatment). The main indicators of effectiveness were the number of patients whose hemoglobin levels (a protein found in red blood cells) remained higher than the individual target level and the number of red blood cell transfusions needed during the first 26 weeks of treatment .

What benefit has Soliris shown during the studies?

Soliris was more effective than placebo in improving the symptoms of EPN. In the main study, 49% of patients (21 out of 43) treated with Soliris had stable hemoglobin levels and, on average, did not need red blood cell transfusions. In comparison, none of the 44 patients who took placebo had stable hemoglobin levels and these needed, on average, 10 transfusions.

What is the risk associated with Soliris?

The most common side effect with Soliris (seen in more than 1 patient in 10) is headache. For the full list of all side effects reported with Soliris, see the Package Leaflet.

Soliris should not be used in people who may be hypersensitive (allergic) to eculizumab, mouse proteins or any of the other substances, or who are suffering or are expected to have hereditary complement deficiency. 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 in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), but noted that evidence for the benefit of Soliris is limited to subjects previously subjected to blood transfusions. The committee recommended that Soliris be given marketing authorization.

What measures are being taken to ensure the safe use of Soliris?

The manufacturer of Soliris will agree on the details of a system in each Member State that ensures that the distribution of the medicine takes place only after verifying that the patient has been adequately vaccinated. It will also provide prescribers and patients with information about the safety of the medicine and will verify that the prescribers use it safely.