EU/3/12/972

About

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in June 2018 on request of the Sponsor.

On 16 October 2015, orphan designation (EU/3/12/972) was granted by the European Commission to Ultragenyx UK Limited, United Kingdom, for sialic acid (also known as aceneuramic acid) for treatment of GNE myopathy.

The sponsorship was transferred to Ultragenyx Germany GmbH, Germany, in January 2017.

Key facts

Active substance
Sialic acid (also known as aceneuramic acid)
Disease / condition
Treatment of GNE myopathy
Date of decision
16/10/2015
Outcome
Withdrawn
Orphan decision number
EU/3/12/972

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

Ultragenyx Germany GmbH
Stadtquartier Friedrichstr. 191
10117 Berlin
Germany
Tel. +49 30 20 65 90
E-mail: Berlin.Stadtquartier@regus.com

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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