Myozyme

alglucosidase alfa

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

What is Myozyme?

Myozyme is a powder that is made up into a solution for infusion (drip into a vein). It contains the active substance alglucosidase alfa.

What is Myozyme used for?

Myozyme is used to treat patients who have Pompe disease, a rare inherited disorder. Patients with Pompe disease do not have enough of an enzyme called alpha-glucosidase. This enzyme normally breaks down sugar stored as glycogen into glucose that can be used for energy by the body’s cells. If the enzyme is not present, glycogen builds up in certain tissues, particularly the muscles, including the heart and diaphragm (the main breathing muscle under the lungs). The progressive build-up of glycogen causes a wide range of symptoms, including an enlarged heart, breathing difficulties and muscle weakness. The disease can appear at birth (the ‘infantile-onset’ form) but also later in life (the ‘late-onset’ form).

Because the number of patients with Pompe disease is low, the disease is considered ‘rare’, and Myozyme was designated as an ‘orphan medicine’ (a medicine used in rare diseases) on 14 February 2001.

The medicine can only be obtained with a prescription.

How is Myozyme used?

Myozyme treatment should be supervised by a doctor who has experience in the management of patients with Pompe disease or other inherited diseases of the same type.

Myozyme is given as an infusion of 20 mg per kilogram body weight given once every two weeks. The infusion should start slowly and then be gradually sped up as long as there are no signs of side effects caused by the infusion.

How does Myozyme work?

Myozyme is an enzyme replacement therapy. Enzyme replacement therapy provides patients with the enzyme they are lacking; in this case, alpha-glucosidase. The active substance in Myozyme, alglucosidase alfa, is a copy of human alpha-glucosidase, which is produced by a method known as ‘recombinant DNA technology’: the enzyme is made by a cell that has received a gene (DNA) that makes it able to produce the enzyme. The replacement enzyme helps to break down glycogen and stops it building up abnormally in the cells.

How has Myozyme been studied?

Myozyme has been studied in two main studies involving a total of 39 babies and children up to the age of three and a half years with infantile-onset Pompe disease. These patients were compared with a ‘historical comparison group’ of babies and young children with Pompe disease who had not received treatment and did not take part in the studies. The main measures of effectiveness were the number of patients who survived and the number who did not need a ventilator to help them breathe.

Myozyme has also been compared with placebo (a dummy treatment) in one main study involving 90 patients with late-onset disease. The main measures of effectiveness were the improvement in the distance the patients could walk in six minutes and in their ‘forced vital capacity’ (a measure of how well their lungs were working). The study lasted for up to 18 months.

What benefit has Myozyme shown during the studies?

In the first main study, which involved babies less than six months old, all 18 patients treated with Myozyme were alive at 18 months of age, and 15 of these did not need a ventilator to help them breathe. In contrast, only one of the 42 patients in the historical comparison group was alive at 18 months of age. The results were confirmed in the other study, which involved children aged between six months and three and a half years.

In late-onset disease, Myozyme was more effective than placebo at improving both the distance the patients could walk and their lung function over the course of the study. 

What is the risk associated with Myozyme?

During the studies in patients with infantile-onset Pompe disease, the most common side effects with Myozyme (seen in more than 1 patient in 10) were tachycardia (rapid heartbeat), flushing (redness), cough, tachypnoea (rapid breathing), vomiting, urticaria (itchy rash), rash, pyrexia (fever) and decreased oxygen saturation (low oxygen levels in the blood). In the study of late-onset disease, patients had many of the same side effects, but they were seen less often than in studies of infantile-onset disease. Almost all of the side effects seen with Myozyme occurred during or just after the infusion and were mild or moderate. For the full list of all side effects reported with Myozyme, see the package leaflet.

Patients who receive Myozyme may develop antibodies (proteins that are produced in response to Myozyme). The effect of these antibodies on the safety and effectiveness of Myozyme is not yet clear.

Myozyme must not be used in people who have had a life-threatening anaphylactic (severe allergic) reaction to alglucosidase alfa or any of the other ingredients, which could not be managed by giving the medicine at a slower infusion rate and reduced dose.

Why has Myozyme been approved?

The CHMP decided that Myozyme’s benefits are greater than its risks and recommended that it be given marketing authorisation.

What measures are being taken to ensure the safe use of Myozyme?

The company that makes Myozyme is putting a plan in place to ensure that Myozyme is used safely, mainly by monitoring how patients who receive Myozyme develop antibodies, by setting up a register open to all patients with Pompe disease, and by ensuring that doctors know about the reactions that patients may have to the infusion.

Other information about Myozyme

The European Commission granted a marketing authorisation valid throughout the European Union for Myozyme to Genzyme Europe B.V. on 29 March 2006. The marketing authorisation is valid for an unlimited period.

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

Name Language First published Last updated
Myozyme : EPAR - Summary for the public BG = bălgarski 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public ES = español 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public CS = čeština 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public DA = dansk 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public DE = Deutsch 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public ET = eesti keel 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public EL = elliniká 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public EN = English 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public FR = français 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public IT = italiano 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public LV = latviešu valoda 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public LT = lietuvių kalba 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public HU = magyar 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public MT = Malti 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public NL = Nederlands 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public PL = polski 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public PT = português 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public RO = română 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public SK = slovenčina 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public SL = slovenščina 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public FI = suomi 15/01/2009 23/01/2014
Myozyme : EPAR - Summary for the public SV = svenska 15/01/2009 23/01/2014

This EPAR was last updated on 23/01/2014 .

Authorisation details

Product details

Product details for Myozyme
NameMyozyme
Agency product numberEMEA/H/C/000636
Active substance

alglucosidase alfa

International non-proprietary name (INN) or common name

alglucosidase alfa

Therapeutic area Glycogen Storage Disease Type II
Anatomical therapeutic chemical (ATC) code A16AB07
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 Myozyme
Marketing-authorisation holder

Genzyme Europe B.V.

Revision11
Date of issue of marketing authorisation valid throughout the European Union29/03/2006

Contact address:

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

Product information

Product information

18/12/2013  Myozyme -EMEA/H/C/000636 -II/41

Name Language First published Last updated
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014
Myozyme : EPAR - Product Information SV = svenska 07/09/2009 23/01/2014

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
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  
Myozyme : EPAR - All Authorised presentations SV = svenska 31/05/2007  

Pharmacotherapeutic group

Other alimentary-tract and metabolism products

Therapeutic indication

Myozyme is indicated for long-term enzyme-replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid-α-glucosidase deficiency).

In patients with late-onset Pompe disease the evidence of efficacy is limited.

Assessment History

Changes since initial authorisation of medicine

Initial marketing-authorisation documents

Name Language First published Last updated
Myozyme : EPAR - Procedural steps taken before authorisation SV = svenska 19/05/2009  
Myozyme : EPAR - Scientific Discussion SV = svenska 31/05/2007  

Authorised

This medicine is approved for use in the European Union

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