Elaprase

idursulfase

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This is a summary of the European public assessment report (EPAR). It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the studies performed, to reach its recommendations on how to use the medicine.

If you need more information about your medical condition or your treatment, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist. If you want more information on the basis for the CHMP recommendations, read the scientific discussion (also part of the EPAR).

What is Elaprase?

Elaprase is a concentrate that is made up into a solution for infusion (drip) into a vein. It contains the active substance idursulfase.

What is Elaprase used for?

Elaprase is used to treat patients with Hunter syndrome. It is designed for long-term use.

Hunter syndrome, which is also known as mucopolysaccharidosis II, is a rare, inherited disease that primarily affects male patients. Patients with Hunter syndrome do not produce an enzyme called iduronate-2-sulfatase. This enzyme is needed to break down substances in the body called glycosaminoglycans (GAGs). Since patients with Hunter syndrome cannot break these substances down, the GAGs gradually build up in most of the organs in the body and damage them. This causes a wide range of symptoms, particularly difficulty breathing and difficulty walking. Without treatment, these symptoms become more severe over time.

Because the number of patients with Hunter syndrome is low, the disease is considered ‘rare’, and Elaprase was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 11 December 2001.

The medicine can only be obtained with a prescription.

How is Elaprase used?

Elaprase treatment should be supervised by a doctor or other healthcare professional who has experience in the management of patients with Hunter syndrome or other inherited diseases affecting the metabolism.

Elaprase is given every week, as an infusion into a vein, at a dose of 0.5 mg per kilogram body weight. The infusion should last three hours. However, as long as the patient does not develop an infusion reaction (rash, itching, fever, headache, high blood pressure or flushing), the medicine can be infused more rapidly, by gradually reducing the duration of infusion to one hour.

Patients who tolerate the infusions well for several months in a clinic may be able to start having them at home. Home infusions must be done under the supervision of a doctor or nurse.

How does Elaprase work?

The active substance in Elaprase, idursulfase, is a copy of the human enzyme iduronate-2-sulfatase It replaces the enzyme that is missing or defective in patients with Hunter syndrome. Supplying the enzyme will help to break down GAGs and stop it building up in the body thereby helping to improve the symptoms of the disease.

How has Elaprase been studied?

The main study of Elaprase involved 96 male patients aged between five and 31 years, and compared it with placebo (a dummy treatment). The main measures of effectiveness were lung function (‘forced vital capacity’, the maximum amount of air the patient could breathe out), and the distance the patients could walk in six minutes, which measures the combined effects of the illness on the heart, lungs, joints and other organs. These measurements were taken before and after a year of treatment.

What benefit has Elaprase shown during the studies?

Elaprase improved lung function and the walking ability of the patients. At the start of the study, the patients could walk an average of around 395 metres in six minutes. After a year, the patients receiving Elaprase could walk a further 43 metres on average, and the patients receiving placebo could walk a further 8 metres. The medicine also produced an improvement in lung function, while the patients on placebo showed a slight worsening.

What is the risk associated with Elaprase?

The most common side effects with Elaprase (seen in more than 1 patient in 10) are related to the infusion, including skin reactions (rash or itching), pyrexia (fever), headache, hypertension (high blood pressure) and swelling at the site of the infusion. Other side effects seen in more than 1 patient in 10 are flushing (reddening of the skin), wheezing, dyspnoea (difficulty breathing), abdominal pain (stomach ache), nausea (feeling sick), dyspepsia (heartburn), diarrhoea, vomiting and chest pain. Severe allergic reactions have been seen in some patients taking Elaprase. For the full list of all side effects reported with Elaprase, see the package leaflet.

Elaprase must not be used in people who have had a severe or life-threatening allergic (anaphylactic) reactions to idursulfase or any of the other ingredients if their allergy is not controllable. If given to patients who have had severe allergic reactions in the past, Elaprase should be used with caution, and trained staff and equipment for emergency resuscitation should be available during infusion.

Why has Elaprase been approved?

The CHMP concluded that the improvements shown in the study, even if limited, represent a clinical benefit in the treatment of Hunter syndrome. The Committee decided that Elaprase’s benefits are greater than its risks and recommended that it be given marketing authorisation.

Elaprase has been authorised under 'Exceptional Circumstances'. This means that because Hunter syndrome is rare, it has not been possible to obtain complete information about Elaprase. Every year, the European Medicines Agency will review any new information that may become available and this summary will be updated as necessary.

What information is still awaited for Elaprase?

The company that makes Elaprase will investigate the long-term effects of the medicine, and whether it stimulates the body to produce antibodies that could affect the medicine’s safety and effectiveness. The company will also study the medicine’s effectiveness in patients below five years of age, and investigate whether it has any effects on the lungs, the heart or the blood vessels.

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

A risk management plan has been developed to ensure that Elaprase 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 Elaprase, including the appropriate precautions to be followed by healthcare professionals and patients.

Other information about Elaprase

The European Commission granted a marketing authorisation valid throughout the European Union for Elaprase on 8 January 2007.

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

Name Language First published Last updated
Elaprase : EPAR - Summary for the public BG = bălgarski 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public ES = español 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public CS = čeština 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public DA = dansk 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public DE = Deutsch 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public ET = eesti keel 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public EL = elliniká 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public EN = English 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public FR = français 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public IT = italiano 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public LV = latviešu valoda 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public LT = lietuvių kalba 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public HU = magyar 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public MT = Malti 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public NL = Nederlands 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public PL = polski 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public PT = português 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public RO = română 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public SK = slovenčina 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public SL = slovenščina 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public FI = suomi 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public SV = svenska 18/01/2007 15/12/2014
Elaprase : EPAR - Summary for the public HR = Hrvatski 18/01/2007 15/12/2014

This EPAR was last updated on 15/12/2014 .

Authorisation details

Product details

Product details for Elaprase
NameElaprase
Agency product numberEMEA/H/C/000700
Active substance

idursulfase

International non-proprietary name (INN) or common name

idursulfase

Therapeutic area Mucopolysaccharidosis II
Anatomical therapeutic chemical (ATC) code A16AB09
Additional monitoring

This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.

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.

Exceptional Circumstances

There were "exceptional circumstances" concerning the approval of this medicine. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicine for which authorisation is being sought, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.

Publication details

Publication details for Elaprase
Marketing-authorisation holder

Shire Human Genetic Therapies AB

Revision10
Date of issue of marketing authorisation valid throughout the European Union08/01/2007

Contact address:

Shire Human Genetic Therapies AB
Svärdvägen 11D
182 33 Danderyd
Sweden

Product information

Product information

25/09/2014  Elaprase -EMEA/H/C/000700 -II/0051

Name Language First published Last updated
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014
Elaprase : EPAR - Product Information HR = Hrvatski 25/03/2009 14/11/2014

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
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  
Elaprase : EPAR - All Authorised presentations HR = Hrvatski 18/01/2007  

Pharmacotherapeutic group

Other alimentary tract and metabolism products

Therapeutic indication

Elaprase is indicated for the long-term treatment of patients with Hunter syndrome (mucopolysaccharidosis II, MPS II). Heterozygous females were not studied in the clinical trials.

Assessment History

Changes since initial authorisation of medicine

Initial marketing-authorisation documents

Name Language First published Last updated
Elaprase : EPAR - Procedural steps taken before authorisation HR = Hrvatski 18/01/2007  
Elaprase : EPAR - Scientific Discussion HR = Hrvatski 18/01/2007  

Authorised

This medicine is approved for use in the European Union

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