Replagal

agalsidase alfa

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

What is Replagal?

Replagal is medicine that contains the active substance agalsidase alfa. It is available as a concentrate to be made into solution for infusion (drip) into a vein.

What is Replagal used for?

Replagal 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 (Gb3 or GL-3). If the enzyme is not present, Gb3 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.

The medicine can only be obtained with a prescription.

How is Replagal used?

Only a doctor who has experience in treating patients with Fabry disease or other inherited metabolic diseases should give Replagal.

Replagal is given once every 2 weeks as an infusion of 0.2 mg per kilogram body weight over 40 minutes. It is intended for long-term use.

How does Replagal work?

Replagal is an enzyme-replacement therapy. Enzyme-replacement therapy provides patients with the enzyme they are lacking. Replagal is designed to replace the human enzyme alpha-galactosidase A, which is lacking in people with Fabry disease. The active substance in Replagal, agalsidase alfa, is a copy of the human enzyme, produced by a method known as ‘recombinant DNA technology’: it is made by cells that have received a gene (DNA), which makes them able to produce the enzyme. The replacement enzyme helps to break down the Gb3 and stops it building up in the cells.

How has Replagal been studied?

Replagal has been compared with placebo (a dummy treatment) in two main studies involving a total of 40 male patients. The first study measured the effects of Replagal on pain while the second study measured its effect on the mass of the left ventricle (heart muscle), a measure of the amount of Gb3 in the heart cells. The effect of giving doses weekly rather than every two weeks was also investigated.

A further study was carried out in 15 female patients. Replagal has also been evaluated in additional studies involving 38 children aged 7 years or above.

What benefit has Replagal shown during the studies?

After 6 months of treatment, Replagal significantly reduced pain in patients compared with placebo.

Replagal reduced left ventricle mass by an average of 11.5 g while patients receiving placebo had an increase in left ventricular mass of 21.8 g. The effects in female patients were comparable to those in male patients and weekly dosing had no advantage over standard doses.

Children who received Replagal had no unexpected increase in heart mass, and the levels of Gb3 in their blood were reduced.

What is the risk associated with Replagal?

The most common side effects with Replagal (seen in more than 1 patient in 10) are infusion-associated reactions. These include chills, headache, nausea, pyrexia (fever), pain and discomfort, flushing and fatigue (tiredness), and are rarely severe. For the full list of all side effects and restrictions with Replagal, see the package leaflet.

Why has Replagal been approved?

The CHMP decided that for patients with Fabry disease treatment with Replagal might provide long-term clinical benefits. The CHMP decided that Replagal’s benefits are greater than its risks and recommended that it be given marketing authorisation.

Replagal 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 20 July 2015.

Other information about Replagal

The European Commission granted a marketing authorisation valid throughout the European Union for Replagal on 3 August 2001.

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

Name Language First published Last updated
Replagal : EPAR - Summary for the public BG = bălgarski 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public ES = español 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public CS = čeština 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public DA = dansk 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public DE = Deutsch 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public ET = eesti keel 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public EL = elliniká 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public EN = English 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public FR = français 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public IT = italiano 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public LV = latviešu valoda 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public LT = lietuvių kalba 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public HU = magyar 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public MT = Malti 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public NL = Nederlands 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public PL = polski 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public PT = português 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public RO = română 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public SK = slovenčina 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public SL = slovenščina 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public FI = suomi 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public SV = svenska 12/04/2007 29/07/2015
Replagal : EPAR - Summary for the public HR = Hrvatski 12/04/2007 29/07/2015

This EPAR was last updated on 11/08/2016 .

Authorisation details

Product details

Product details for Replagal
NameReplagal
Agency product numberEMEA/H/C/000369
Active substance

agalsidase alfa

International non-proprietary name (INN) or common name

agalsidase alfa

Therapeutic area Fabry Disease
Anatomical therapeutic chemical (ATC) code A16AB03

Publication details

Publication details for Replagal
Marketing-authorisation holder

Shire Human Genetic Therapies AB

Revision20
Date of issue of marketing authorisation valid throughout the European Union03/08/2001

Contact address:

Shire Human Genetic Therapies AB
Vasagatan 7
111 20 Stockholm
Sweden

Product information

Product information

14/07/2016  Replagal -EMEA/H/C/000369 -IAIN/0093

Name Language First published Last updated
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016
Replagal : EPAR - Product Information HR = Hrvatski 03/11/2008 11/08/2016

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
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013
Replagal : EPAR - All Authorised presentations HR = Hrvatski 21/07/2006 11/11/2013

Pharmacotherapeutic group

Other alimentary-tract and metabolism products

Therapeutic indication

Replagal 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
Replagal : EPAR - Procedural steps taken before authorisation HR = Hrvatski 21/07/2006  
Replagal : EPAR - Scientific Discussion HR = Hrvatski 21/07/2006  

Authorised

This medicine is approved for use in the European Union

More information on Replagal

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