Galafold

migalastat

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This is a summary of the European public assessment report (EPAR) for Galafold. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Galafold.

For practical information about using Galafold, patients should read the package leaflet or contact their doctor or pharmacist.

What is Galafold and what is it used for?

Galafold is a medicine used to treat patients aged 16 years or over with Fabry disease. This is a rare inherited disorder where patients have various mutations (changes) in the gene responsible for the production of an enzyme called alpha-galactosidase A, which normally breaks down a fatty substance called globotriaosylceramide (GL-3). In patients with Fabry disease, this enzyme does not work properly. As a result, GL-3 cannot be broken down and it builds up in various cells in the body, including in the heart and kidneys.

Because the number of patients with Fabry disease is low, the disease is considered ‘rare’ and Galafold was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 22 May 2006.

Galafold contains the active substance migalastat.

How is Galafold used?

Galafold can only be obtained with a prescription and treatment should only be started and supervised by a doctor who is experienced in the diagnosis and treatment of Fabry disease.

Galafold is available as capsules (123 mg). The recommended dose of Galafold is one capsule every other day, taken by mouth at least 2 hours before or after food.

Galafold is only for use in patients with certain mutations in the alpha-galactosidase A gene. For more information, see the summary of product characteristics (also part of the EPAR).

How does Galafold work?

The active substance in Galafold, migalastat, attaches to certain unstable forms of alpha-galactosidase A, stabilising the enzyme. This allows the enzyme to be transported into areas of the cell where it can break down GL-3.

What benefits of Galafold have been shown in studies?

Galafold has been investigated in two main studies involving a total of 127 patients with Fabry disease.

The first study, which compared Galafold with placebo (a dummy treatment) in 67 patients, looked at the proportion of patients who responded to treatment (defined as a reduction of at least 50% in GL‑3 deposits in the kidneys). Overall, Galafold was not found to be more effective than placebo at reducing GL‑3 deposits; however, additional analyses including only patients with those genetic mutations that can be treated with Galafold showed that patients responded better to Galafold than to placebo after 6 months of treatment.

The second study, in 60 patients, compared Galafold with the medicines agalsidase alfa or agalsidase beta, two treatments that replace the missing enzyme. The main measure of effectiveness was the change in patients’ kidney function after 18 months of treatment. In this study, Galafold was found to be as effective as enzyme replacement in stabilising patients’ kidney function.

What are the risks associated with Galafold?

The most common side effect with Galafold (which may affect around 1 in 10 people) is headache.

For the full list of all side effects and restrictions with Galafold, see the package leaflet.

Why is Galafold approved?

The Agency’s Committee for Medicinal Products for Human Use (CHMP) decided that Galafold’s benefits are greater than its risks and recommended that it be approved for use in the EU. The Committee noted that Galafold was studied in a limited number of patients, however the available evidence is considered sufficient for such a rare disease. The CHMP also noted that Galafold is taken by mouth and this could be an advantage compared with other authorised treatments such as enzyme replacement which are given by infusion (drip) into a vein. Regarding safety, Galafold was well-tolerated.

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

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

Other information about Galafold

The European Commission granted a marketing authorisation valid throughout the European Union for Galafold on 26 May 2016.

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

Name Language First published Last updated
Galafold : EPAR - Summary for the public BG = bălgarski 2016-06-09  
Galafold : EPAR - Summary for the public ES = español 2016-06-09  
Galafold : EPAR - Summary for the public CS = čeština 2016-06-09  
Galafold : EPAR - Summary for the public DA = dansk 2016-06-09  
Galafold : EPAR - Summary for the public DE = Deutsch 2016-06-09  
Galafold : EPAR - Summary for the public ET = eesti keel 2016-06-09  
Galafold : EPAR - Summary for the public EL = elliniká 2016-06-09  
Galafold : EPAR - Summary for the public EN = English 2016-06-09  
Galafold : EPAR - Summary for the public FR = français 2016-06-09  
Galafold : EPAR - Summary for the public IT = italiano 2016-06-09  
Galafold : EPAR - Summary for the public LV = latviešu valoda 2016-06-09  
Galafold : EPAR - Summary for the public LT = lietuvių kalba 2016-06-09  
Galafold : EPAR - Summary for the public HU = magyar 2016-06-09  
Galafold : EPAR - Summary for the public MT = Malti 2016-06-09  
Galafold : EPAR - Summary for the public NL = Nederlands 2016-06-09  
Galafold : EPAR - Summary for the public PL = polski 2016-06-09  
Galafold : EPAR - Summary for the public PT = português 2016-06-09  
Galafold : EPAR - Summary for the public RO = română 2016-06-09  
Galafold : EPAR - Summary for the public SK = slovenčina 2016-06-09  
Galafold : EPAR - Summary for the public SL = slovenščina 2016-06-09  
Galafold : EPAR - Summary for the public FI = suomi 2016-06-09  
Galafold : EPAR - Summary for the public SV = svenska 2016-06-09  
Galafold : EPAR - Summary for the public HR = Hrvatski 2016-06-09  

This EPAR was last updated on 14/12/2016 .

Authorisation details

Product details

Product details for Galafold
NameGalafold
Agency product numberEMEA/H/C/004059
Active substance

migalastat hydrochloride

International non-proprietary name (INN) or common name

migalastat

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

Publication details

Publication details for Galafold
Marketing-authorisation holder

Amicus Therapeutics UK Ltd

Revision2
Date of issue of marketing authorisation valid throughout the European Union26/05/2016

Contact address:

Amicus Therapeutics UK Ltd
Phoenix House 
Oxford Road 
Tatling End
Gerrards Cross SL9 7AP 
United Kingdom

Product information

Product information

13/10/2016  Galafold -EMEA/H/C/004059 -II/0002

Name Language First published Last updated
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14
Galafold : EPAR - Product Information HR = Hrvatski 2016-06-09 2016-12-14

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
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  
Galafold : EPAR - All Authorised presentations HR = Hrvatski 2016-06-09  

Pharmacotherapeutic group

Therapeutic indication

Galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation (see the tables in section 5.1).

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated
Galafold : EPAR - Procedural steps taken and scientific information after authorisation HR = Hrvatski 2016-10-28 2016-12-14

Initial marketing-authorisation documents

Name Language First published Last updated
Galafold : EPAR - Public assessment report HR = Hrvatski 2016-06-09  
CHMP summary of opinion for Galafold HR = Hrvatski 2016-04-01