Aldurazyme

laronidase

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This is a summary of the European public assessment report (EPAR) for Aldurazyme. 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 Aldurazyme.

What is Aldurazyme?

Aldurazyme is a solution for infusion (drip into a vein) that contains the active substance laronidase.

What is Aldurazyme used for?

Aldurazyme is used in patients with a confirmed diagnosis of mucopolysaccharidosis I (MPS I; α L iduronidase deficiency) to treat the non-neurological symptoms of the disease (symptoms that are not connected with the brain or nerves). MPS I is a rare, inherited disease, in which the level of α L iduronidase enzyme activity is much lower than normal. This means that substances called glycosaminoglycans (GAGs) are not broken down, so they build up in most of the organs in the body and damage them. The non-neurological symptoms of MPS I can be an enlarged liver, stiff joints that make moving more difficult, reduced lung volume, heart disease and eye disease.

The medicine can only be obtained with a prescription.

How is Aldurazyme used?

Aldurazyme treatment should be supervised by a doctor who has experience in the management of patients with MPS I or other inherited metabolic diseases. Aldurazyme should be given in a hospital or clinic where resuscitation equipment is available, and patients may need to receive some medicines before the infusion to prevent an allergic reaction. Aldurazyme is given once a week as an infusion. It is designed for long-term use.

How does Aldurazyme work?

The active substance in Aldurazyme, laronidase, is a copy of the human enzyme α L iduronidase. It is produced by a method known as ‘recombinant DNA technology’: the enzyme is made by cells into which a gene (DNA) has been introduced that makes them able to produce laronidase. Laronidase is used as ‘enzyme replacement therapy’, which means that it replaces the enzyme that is missing in patients with MPS I. This controls the symptoms of MPS I, improving the patient’s quality of life.

How has Aldurazyme been studied?

Aldurazyme has been compared with placebo (a dummy treatment) in 45 patients aged six years and over with a confirmed diagnosis of MPS I. The main measure of effectiveness was the forced vital capacity (FVC, a measure of how well the lungs are working) and the distance the patients could walk over six minutes. These were measured before and after 26 weeks of treatment. After this, the study continued for up to four years and all of the patients were treated with Aldurazyme.

Aldurazyme has also been studied in 20 children below the age of five years who received Aldurazyme for a year. The study was looking mainly at the safety of the medicine, but it also measured its ability to reduce the levels of GAGs in the urine and the size of the liver.

What benefit has Aldurazyme shown during the studies?

The study showed that Aldurazyme had improved both the FVC and the walking ability of patients at 26 weeks. This effect was maintained for up to four years.

In children under five years of age, Aldurazyme reduced the levels of GAGs in the urine by about 60%. Half of the children treated had a normal size liver at the end of the study.

What is the risk associated with Aldurazyme?

Most of the side effects seen with Aldurazyme are reactions caused by the infusion rather than the medicine itself. Some of these are severe, but the number of side effects tends to decrease with time. The most common side effects in patients over the age of five years (seen in more than 1 patient in 10) are headache, nausea (feeling sick), abdominal pain (stomach ache), rash, arthropathy (damage to the joints), arthralgia (joint pain), back pain, pain in the extremities (hands and feet), flushing, pyrexia (fever) and reactions at the site of the infusion. In patients under five years of age, the most common side effects (seen in more than 1 patient in 10) are increased blood pressure, decreased oxygen saturation (a measure of how well the lungs are working), tachycardia (rapid heart rate), pyrexia and chills. For the full list of all side effects reported with Aldurazyme, see the package leaflet.

Almost all patients who receive Aldurazyme develop antibodies (proteins that are produced in response to Aldurazyme). The effect of these on the safety and effectiveness of the medicine is not fully known.

Aldurazyme must not be used in people who are severely allergic to laronidase or any of the other ingredients.

Why has Aldurazyme been approved?

The CHMP decided that Aldurazyme gives effective control of the symptoms of MPS I. The Committee decided that Aldurazyme’s benefits are greater than its risks and recommended that Aldurazyme be given marketing authorisation.

Aldurazyme was originally designated an orphan medicine and has been authorised under ‘Exceptional Circumstances’. This means that, because the disease is rare, it has not been possible to obtain complete information about Aldurazyme. 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 Aldurazyme?

The company that makes Aldurazyme will monitor patients receiving Aldurazyme, looking at reactions to the infusion and the development of antibodies.

Other information about Aldurazyme

The European Commission granted a marketing authorisation valid throughout the European Union for Aldurazyme on 10 June 2003.

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

Name Language First published Last updated
Aldurazyme : EPAR - Summary for the public BG = bălgarski 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public ES = español 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public CS = čeština 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public DA = dansk 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public DE = Deutsch 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public ET = eesti keel 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public EL = elliniká 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public EN = English 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public FR = français 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public IT = italiano 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public LV = latviešu valoda 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public LT = lietuvių kalba 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public HU = magyar 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public MT = Malti 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public NL = Nederlands 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public PL = polski 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public PT = português 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public RO = română 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public SK = slovenčina 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public SL = slovenščina 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public FI = suomi 27/07/2009 20/11/2013
Aldurazyme : EPAR - Summary for the public SV = svenska 27/07/2009 20/11/2013

This EPAR was last updated on 20/11/2013 .

Authorisation details

Product details

Product details for Aldurazyme
NameAldurazyme
Agency product numberEMEA/H/C/000477
Active substance

laronidase

International non-proprietary name (INN) or common name

laronidase

Therapeutic area Mucopolysaccharidosis I
Anatomical therapeutic chemical (ATC) code A16AB05
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.

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 Aldurazyme
Marketing-authorisation holder

Genzyme Europe B.V.

Revision12
Date of issue of marketing authorisation valid throughout the European Union10/06/2003

Contact address:

Genzyme Europe B.V.
Gooimer 10
NL-1411 DD Naarden
The Netherlands

Product information

Product information

24/10/2013  Aldurazyme -EMEA/H/C/000477 -S/0047

Name Language First published Last updated
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013
Aldurazyme : EPAR - Product Information SV = svenska 27/07/2009 20/11/2013

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
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  
Aldurazyme : EPAR - All Authorised presentations SV = svenska 21/10/2005  

Pharmacotherapeutic group

Other alimentary-tract and metabolism products

Therapeutic indication

Aldurazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis I (MPS I; alpha-L-iduronidase deficiency) to treat the nonneurological manifestations of the disease.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated
Aldurazyme : EPAR - Procedural steps taken and scientific information after authorisation SV = svenska 27/07/2009 20/11/2013

Initial marketing-authorisation documents

Name Language First published Last updated
Aldurazyme : EPAR - Procedural steps taken before authorisation SV = svenska 21/10/2005  
Aldurazyme : EPAR - Scientific Discussion SV = svenska 21/10/2005  

Authorised

This medicine is approved for use in the European Union

More information on Aldurazyme

This product is no longer an orphan medicine. It was originally designated an orphan medicine on 14 February 2001. Aldurazyme was withdrawn from the Community register of orphan medicinal products in June 2013 at the end of the 10-year period of market exclusivity.