This medicine is authorised for use in the European Union.


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

This EPAR was last updated on 20/10/2020

Authorisation details

Product details
Agency product number
Active substance
International non-proprietary name (INN) or common name
Therapeutic area (MeSH)
Mucopolysaccharidosis VI
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional 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.

Exceptional circumstancesExceptional circumstances

This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.

Publication details
Marketing-authorisation holder
BioMarin International Limited
Date of issue of marketing authorisation valid throughout the European Union
Contact address

Shanbally, Ringaskiddy
County Cork, P43 R298

Product information

27/08/2020 Naglazyme - EMEA/H/C/000640 - IB/0082


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Pharmacotherapeutic group

Other alimentary tract and metabolism products

Therapeutic indication

Naglazyme is indicated for long-term enzyme-replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis VI (MPS VI; N-acetylgalactosamine-4-sulfatase deficiency; Maroteaux-Lamy syndrome) (see section 5.1).

As for all lysosomal genetic disorders, it is of primary importance, especially in severe forms, to initiate treatment as early as possible, before appearance of non-reversible clinical manifestations of the disease.

A key issue is to treat young patients aged <5 years suffering from a severe form of the disease, even though patients <5 years were not included in the pivotal phase-3 study.

Assessment history

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