EU/3/09/657

About

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
Vimizim
Disease / condition
Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome)
Date of decision
24/07/2009
Outcome
Positive
Orphan decision number
EU/3/09/657

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.

Sponsor's contact details

BioMarin International Limited
Shanbally
Ringaskiddy
County Cork P43 R298
Ireland
Tel. +353 1479430
E-mail: shanbally@bmrn.com

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;

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