Fabrazyme

agalsidase beta

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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 of the CHMP recommendations, read the scientific discussion (also part of the EPAR).

What is Fabrazyme?

Fabrazyme is a solution for infusion (drip into a vein) containing the active substance agalsidase beta.

What is Fabrazyme used for?

Fabrazyme is used to treat patients who have Fabry disease, a rare inherited disorder. Patients with Fabry disease do not have enough of an enzyme called alpha-galactosidase A. This enzyme normally breaks down a fatty substance called globotriaosylceramide (GL-3). If the enzyme is not present, GL-3 cannot be broken down and it builds up in the body’s cells, such as kidney cells.

People with Fabry disease may have a wide range of signs and symptoms, including severe conditions such as kidney failure, heart problems and stroke.

Because the number of patients with Fabry disease is low, the disease is considered ‘rare’, and Fabrazyme was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 August 2000.

The medicine can only be obtained with a prescription.

How is Fabrazyme used?

Only a doctor who has experience in treating patients with Fabry disease or other inherited metabolic diseases should give Fabrazyme. It is used as an intravenous infusion of 1 mg per kilogram body weight given once every two weeks. The starting infusion rate should be no more than 0.25 mg per minute (15 mg per hour) to reduce the risk of infusion-related side effects. The infusion rate may be increased gradually with further infusions. One study has looked at what happens to Fabrazyme when given to children and suggested that Fabrazyme could be used in children between eight and 16 years of age at the same dose. Patients who have severe kidney damage may have a weaker response to treatment. Fabrazyme is intended for long-term use.

How does Fabrazyme work?

Fabrazyme is an enzyme replacement therapy. Enzyme replacement therapies provide patients with the enzyme they are lacking. Fabrazyme is designed to replace the human enzyme alpha-galactosidase A, which people with Fabry disease are lacking. The active substance in Fabrazyme, agalsidase beta, is a copy of the human enzyme, produced by a method known as ‘recombinant DNA technology’: it is made by a cell that has received a gene (DNA), which makes it able to produce the enzyme. The replacement enzyme helps to break down GL-3 and stops it building up (accumulating) in the patient’s cells.

How has Fabrazyme been studied?

Three clinical studies involving a total of 73 adult patients were presented. In the main study, Fabrazyme was compared with placebo (a dummy treatment) in 58 patients. The study looked at the effect of the medicines on clearing GL-3 from the kidney.

The effectiveness of Fabrazyme was also tested in 16 children aged between eight and 16 years who had Fabry disease.

What benefit has Fabrazyme shown during the studies?

In the main study, Fabrazyme produced a highly significant and almost complete clearance of GL-3 in the kidney cells after 20 weeks of treatment: 69% of the patients treated with Fabrazyme had the best score for clearance, compared with none of the patients in the placebo group. This might lead to the symptoms of the disease improving or the disease becoming stable.

Children treated with Fabrazyme also had decreases in levels of GL-3 in the blood, with all children having normal levels after 20 weeks of treatment. This was accompanied by improvements in the children’s symptoms and quality of life.

What is the risk associated with Fabrazyme?

The most common side effects with Fabrazyme (seen in more than 1 patient in 10) are caused by the infusion rather than the medicine. These reactions are mainly fever and chills. Other very common side effects include headache, paraesthesia (abnormal sensations like pins and needles), nausea (feeling sick), vomiting, flushing and feeling cold. Side effects reported in children are similar to those seen in adult patients. For the full list of all side effects reported with Fabrazyme, see the package leaflet.

Patients who receive Fabrazyme can develop antibodies (proteins that are produced in response to Fabrazyme and can affect treatment).

Fabrazyme should not be used in people who may be hypersensitive (allergic) to agalsidase beta or any of the other ingredients.

Why has Fabrazyme been approved?

The Committee for Medicinal Products for Human Use (CHMP) decided that, for patients with Fabry disease, treatment with Fabrazyme might provide long-term clinical benefits. The CHMP decided that Fabrazyme’s benefits are greater than its risks for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease. The Committee recommended that Fabrazyme be given marketing authorisation.

Fabrazyme was originally authorised under ‘exceptional circumstances’, because, as the disease is rare, limited information was available at the time of approval. As the company had supplied the additional information requested, the ‘exceptional circumstances’ ended on 6 February 2008.

Other information about Fabrazyme

The European Commission granted a marketing authorisation valid throughout the European Union for Fabrazyme to Genzyme Europe B.V. on 3 August 2001. The marketing authorisation was renewed on 3 August 2006.

Name Language First published Last updated
Fabrazyme : EPAR - Summary for the public BG = bălgarski 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public ES = español 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public CS = čeština 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public DA = dansk 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public DE = Deutsch 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public ET = eesti keel 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public EL = elliniká 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public EN = English 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public FR = français 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public IT = italiano 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public LV = latviešu valoda 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public LT = lietuvių kalba 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public HU = magyar 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public MT = Malti 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public NL = Nederlands 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public PL = polski 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public PT = português 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public RO = română 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public SK = slovenčina 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public SL = slovenščina 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public FI = suomi 2008-04-10 2013-03-19
Fabrazyme : EPAR - Summary for the public SV = svenska 2008-04-10 2013-03-19

This EPAR was last updated on 20/07/2016 .

Authorisation details

Product details

Product details for Fabrazyme
NameFabrazyme
Agency product numberEMEA/H/C/000370
Active substance

agalsidase beta

International non-proprietary name (INN) or common name

agalsidase beta

Therapeutic area Fabry Disease
Anatomical therapeutic chemical (ATC) code A16AB04

Publication details

Publication details for Fabrazyme
Marketing-authorisation holder

Genzyme Europe B.V.

Revision22
Date of issue of marketing authorisation valid throughout the European Union03/08/2001

Contact address:

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

Product information

Product information

22/01/2015  Fabrazyme -EMEA/H/C/000370 -P46/0062

Name Language First published Last updated
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10
Fabrazyme : EPAR - Product Information SV = svenska 2009-07-20 2014-10-10

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
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27
Fabrazyme : EPAR - All Authorised presentations SV = svenska 2005-12-20 2013-11-27

Name Language First published Last updated
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  
Fabrazyme : EPAR - Conditions imposed on member states for safe and effective use SV = svenska 2012-02-02  

Pharmacotherapeutic group

Other alimentary-tract and metabolism products

Therapeutic indication

Fabrazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (α-galactosidase-A deficiency).

Assessment History

Changes since initial authorisation of medicine

Initial marketing-authorisation documents

Name Language First published Last updated
Fabrazyme : EPAR - Procedural steps taken before authorisation SV = svenska 2005-12-20  
Fabrazyme : EPAR - Scientific discussion SV = svenska 2005-12-20  

Authorised

This medicine is approved for use in the European Union

More information on Fabrazyme

This product is no longer an orphan medicine. It was originally designated an orphan medicine on 8 August 2000. Fabrazyme was withdrawn from the Community register of orphan medicinal products in August 2011 at the end of the 10-year period of market exclusivity.