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On 24 June 2021, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Galafold. The marketing authorisation holder for this medicinal product is Amicus Therapeutics Europe Limited.

The CHMP adopted an extension to the existing indication as follows: 1

Galafold is indicated for long-term treatment of adults and adolescents aged 16 12 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).

Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR), and will be available in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.

1 New text in bold, removed text as strikethrough.

Key facts

Agency product number
International non-proprietary name (INN) or common name
  • migalastat
Active substance
  • migalastat hydrochloride
Therapeutic area
Fabry Disease
Additional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Date opinion adopted
Company name
Amicus Therapeutics Europe Limited
Application type

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