Naglazyme

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galsulfase

Authorised
This medicine is authorised for use in the European Union.

Overview

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 06/08/2018

Authorisation details

Product details
Name
Naglazyme
Agency product number
EMEA/H/C/000640
Active substance
galsulfase
International non-proprietary name (INN) or common name
galsulfase
Therapeutic area (MeSH)
Mucopolysaccharidosis VI
Anatomical therapeutic chemical (ATC) code
A16AB
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.

OrphanOrphan

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.

Publication details
Marketing-authorisation holder
BioMarin International Limited
Revision
13
Date of issue of marketing authorisation valid throughout the European Union
23/01/2006
Contact address

Shanbally, Ringaskiddy
County Cork, P43 R298
Ireland

Product information

12/07/2018 Naglazyme - EMEA/H/C/000640 - T/0072

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.

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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