EU/3/10/752: Orphan designation for the treatment of Gaucher disease

velaglucerase alfa

Overview

On 6 June 2010, orphan designation (EU/3/10/752) was granted by the European Commission to Shire Pharmaceuticals Ireland Limited, Ireland, for velaglucerase alfa for the treatment of Gaucher disease.

Velaglucerase alfa has been authorised in the EU as Vpriv since 26 August 2016.

The sponsorship was transferred to Takeda Pharmaceuticals International AG Ireland Branch, Ireland in September 2021.

 

Key facts

Active substance
velaglucerase alfa
Medicine name
Vpriv
Intended use
Treatment of Gaucher disease
Orphan designation status
Positive
EU designation number
EU/3/10/752
Date of designation
06/06/2010
Sponsor

Takeda Pharmaceuticals International AG Ireland Branch
Block 3 Miesian Plaza
50-58 Baggott Street Lower
Dublin 2
D02 Y754
Co. Dublin
Ireland
Tel: +1 800937970
E-mail: medinfoemea@takeda.com

Review of designation

During its meeting of 7-8 July 2010, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/10/752 for Vpriv (velaglucerase alfa) as an orphan medicinal product for the treatment of Gaucher disease. 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. As other satisfactory methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained.

The 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 for Vpriv (velaglucerase alfa) for the treatment of Gaucher disease (PDF/54.85 KB)

    Adopted

    First published: 13/09/2010
    Last updated: 13/09/2010
    EMA/COMP/433831/2010

  • 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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