On 24 January 2013, orphan designation (EU/3/12/1094) was granted by the European Commission to BioMarin Europe Ltd, United Kingdom, for modified recombinant human C-type natriuretic peptide for the treatment of achondroplasia.

Key facts

Active substance
Modified recombinant human C-type natriuretic peptide
Disease / condition
Treatment of achondroplasia
Date of decision
Orphan decision number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

BioMarin Europe Ltd.
10 Bloomsbury Way
London WC1A 2SL
United Kingdom
Tel. + 44 (0)20 7420 0800
Fax + 44 (0)20 7420 0829

Patients' organisations

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

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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