EU/3/09/657: Orphan designation for the treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome)

Recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa)


This medicine is now known as elosulfase alfa.

On 24 July 2009, orphan designation (EU/3/09/657) was granted by the European Commission to BioMarin Europe Limited, United Kingdom, for recombinant human N-acetylgalactosamine-6-sulfatase for the treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome).

Recombinant human N‑acetylgalactosamine‑6-sulfatase has been authorised in the EU as Vimizim since 28 April 2014.

The sponsorship was transferred to BioMarin International Limited, Ireland, in September 2018.

Key facts

Active substance
Recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa)
Medicine name
Intended use
Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome)
Orphan designation status
EU designation number
Date of designation

BioMarin International Limited
County Cork P43 R298
Tel. +353 1479430

Review of designation

During its meeting of 11 to 12 March 2014, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/09/657 for Vimizim (elosulfase alfa, previously known as recombinant human N-acetylgalactosamine-6-sulfatase) as an orphan medicinal product for the treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome). The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other satisfactory methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained1.

1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Vimizim (elosulfase alfa) for the treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome) (PDF/77.97 KB)


    First published: 26/06/2014
    Last updated: 26/06/2014

  • Patients' organisations

    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

    • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

    • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

    EU register of orphan medicines

    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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