Soliris

eculizumab

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This is a summary of the European public assessment report (EPAR) for Soliris. It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Soliris.

What is Soliris?

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

What is Soliris used for?

Soliris is used to treat patients with paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS), two rare, life-threatening genetic diseases that cause the breakdown of red blood cells resulting in various medical complications. PNH results in anaemia (low red-blood-cell counts), thrombosis (formation of blood clots in the blood vessels), pancytopenia (low counts of blood cells) and dark urine, while aHUS results in anaemia, thrombocytopenia (a decrease in the number of platelets, components that help the blood to clot) and kidney failure.

Because the numbers of patients with these medical conditions are low, the diseases are considered ‘rare’, and Soliris was designated an ‘orphan medicine’ (a medicine used in rare diseases) for PNH on 17 October 2003 and for aHUS on 24 July 2009.

The medicine can only be obtained with a prescription.

How is Soliris used?

Soliris must be given by a healthcare professional, such as a doctor or nurse, under the supervision of a doctor who has experience in the management of patients with blood or kidney disorders.

In PNH, treatment with Soliris consists of an infusion (drip into a vein) of 600 mg over 25 to 45 minutes once a week, followed by 900 mg in the fifth week. After this, the dose should be maintained at 900 mg, given approximately every two weeks. At least two weeks before starting Soliris treatment, patients must be vaccinated against meningitis caused by the bacterium Neisseria meningitidis and revaccinated according to current guidelines.

In aHUS, patients aged 18 and over receive an infusion of 900 mg over 25 to 45 minutes once a week for four weeks, followed by 1,200 mg in the fifth week. After this, the dose should be maintained at 1,200 mg, given approximately every two weeks. 

PNH and aHUS patients under 18 years old receive lower doses based on their body weight, administered over 1 to 4 hours.

In patients who receive plasma exchange or infusion (the removal, treatment, and return of blood plasma from their blood circulation), additional doses of Soliris are required.

Patients who receive Soliris must be given a special card that explains the symptoms of certain types of infection, instructing them to seek medical care immediately if they experience them.

How does Soliris work?

The active substance in Soliris, eculizumab, is a monoclonal antibody. A monoclonal antibody is an antibody (a type of protein) that has been designed to recognise and attach to a specific structure (called an antigen) that is found in the body. Eculizumab has been designed to attach to the C5 complement protein, which is part of the body’s defence system called ‘complement’.

In PNH and aHUS, patients have a defect in certain proteins that normally stop complement from attacking blood cells. This defect results in complement inflaming white blood cells and platelets and destroying red blood cells. By blocking the C5 complement protein, eculizumab prevents complement from attacking the cells, reducing their destruction and relieving the symptoms of the disease.

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 87 adults with PNH who had had at least four blood transfusions for anaemia in the previous year. Soliris was compared with placebo (a dummy treatment). The main measures of effectiveness were the number of patients whose levels of haemoglobin (a protein found in red blood cells) remained above their individual target level, and the number of red-blood-cell transfusions that the patients needed during the first 26 weeks of treatment.

Soliris was studied in three main studies involving 67 patients with aHUS. The first study involved 17 patients with aHUS who did not respond to or could not be treated with plasma exchange or infusion. The main measures of effectiveness were the change in platelet count and the number of patients who achieved ‘platelet-count normalisation’ and ‘haematologic normalisation’ (their levels of platelets and lactate dehydrogenase, an enzyme normally found in red blood cells, were within normal levels).

The second study involved 20 patients with aHUS who were already receiving plasma exchange or infusion. The main measures of effectiveness were the numbers of patients who achieved ‘thrombotic microangiopathy event-free’ status (they did not have a decrease of over 25% in platelet count after starting Soliris and did not require plasma exchange or infusion, or dialysis) and the number of patients who achieved ‘haematologic normalisation’ while receiving Soliris.

The third study involved 30 patients with aHUS who had already received at least one dose of eculizumab. The effectiveness of Soliris treatment was assessed using a range of measurements, including the change in platelet count and other measures of effectiveness also used in the first two studies.

What benefit has Soliris shown during the studies?

Soliris was more effective than placebo in improving the symptoms of PNH. In the main study in PNH, 49% of the patients receiving Soliris had stable haemoglobin levels (21 out of 43), and, on average, they did not need any transfusions of red blood cells. In comparison, none of the 44 patients receiving placebo had stable haemoglobin levels, and they needed an average of 10 transfusions.

In the first study in aHUS, platelet counts increased and were within normal levels in 82% of the patients, while 87% (13 out of 15 patients) with initially low platelet counts achieved platelet count normalisation and 76% achieved haematologic normalisation. In the second study in aHUS, 80% of the patients achieved ‘thrombotic microangiopathy event-free’ status and 90% achieved haematologic normalisation. In the third study, platelet counts increased and were within normal levels in 83% of the patients, while 77% (10 out of 13 patients) with initially low platelet counts achieved platelet-count normalisation.

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 must not be used in people who are hypersensitive (allergic) to eculizumab, mouse proteins or any of the other ingredients. Because of an increased risk of meningitis, it must not be given to people who are infected with Neisseria meningitidis, or who have not been vaccinated against this bacterium, or in the case of aHUS who do not take appropriate antibiotics until two weeks after vaccination.

Why has Soliris been approved?

The CHMP decided that the benefits of Soliris are greater than its risks for the treatment of patients with PNH, but noted that evidence of the benefit of Soliris is limited to patients who have had blood transfusions in the past. The Committee also concluded that the benefits of Soliris are greater than its risks for patients with aHUS whether or not they responded to standard treatment. The Committee therefore recommended that Soliris be given marketing authorisation.

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

A risk management plan has been developed to ensure that Soliris is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Soliris, including the appropriate precautions to be followed by healthcare professionals and patients.

In addition, the company that makes Soliris will agree the details of a system in each Member State that will ensure that distribution of the medicine only occurs after checking that the patient has been vaccinated appropriately. It will also provide prescribers and patients with information on the safety of the medicine, and check that prescribers use the medicine safely.

Other information about Soliris

The European Commission granted a marketing authorisation valid throughout the European Union for Soliris on 20 June 2007.

For more information about treatment with Soliris, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Name Language First published Last updated
Soliris : EPAR - Summary for the public BG = bălgarski 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public ES = español 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public CS = čeština 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public DA = dansk 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public DE = Deutsch 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public ET = eesti keel 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public EL = elliniká 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public EN = English 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public FR = français 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public IT = italiano 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public LV = latviešu valoda 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public LT = lietuvių kalba 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public HU = magyar 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public MT = Malti 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public NL = Nederlands 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public PL = polski 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public PT = português 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public RO = română 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public SK = slovenčina 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public SL = slovenščina 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public FI = suomi 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public SV = svenska 01/07/2009 07/05/2014
Soliris : EPAR - Summary for the public HR = Hrvatski 01/07/2009 07/05/2014

This EPAR was last updated on 07/05/2014 .

Authorisation details

Product details

Product details for Soliris
NameSoliris
Agency product numberEMEA/H/C/000791
Active substance

eculizumab

International non-proprietary name (INN) or common name

eculizumab

Therapeutic area Hemoglobinuria, Paroxysmal
Anatomical therapeutic chemical (ATC) code L04AA25
Treatment of rare diseases

This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

Publication details

Publication details for Soliris
Marketing-authorisation holder

Alexion Europe SAS

Revision13
Date of issue of marketing authorisation valid throughout the European Union20/06/2007

Contact address:

Alexion Europe SAS
25 Boulevard de l’Amiral Bruix
75016 Paris
France

Product information

Product information

21/03/2014  Soliris -EMEA/H/C/000791 -II/0058/G

Name Language First published Last updated
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014
Soliris : EPAR - Product Information HR = Hrvatski 01/07/2009 11/04/2014

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  
Soliris : EPAR - All Authorised presentations HR = Hrvatski 29/06/2007  

Name Language First published Last updated
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012
Soliris : EPAR - Conditions imposed on member states for safe and effective use - Annex IV HR = Hrvatski 01/07/2009 09/07/2012

Pharmacotherapeutic group

Immunosuppressants

Therapeutic indication

Soliris is indicated in adults and children for the treatment of patients with:

  • paroxysmal nocturnal haemoglobinuria (PNH). Evidence of clinical benefit of Soliris in the treatment of patients with PNH is limited to patients with history of transfusions;
  • atypical haemolytic uraemic syndrome (aHUS).

Assessment History

Changes since initial authorisation of medicine

Initial marketing-authorisation documents

Name Language First published Last updated
Soliris : EPAR - Procedural steps taken before authorisation HR = Hrvatski 29/06/2007  
Soliris : EPAR - Scientific Discussion HR = Hrvatski 29/06/2007  

Authorised

This medicine is approved for use in the European Union

News

More information on Soliris