EU/3/13/1118: Orphan designation for the treatment of neuronal ceroid lipofuscinosis type 2

Recombinant human tripeptidyl-peptidase 1


This medicine is now known as cerliponase alfa.

On 12 March 2013, orphan designation (EU/3/13/1118) was granted by the European Commission to BioMarin Europe Ltd., UK, for recombinant human tripeptidyl-peptidase 1 for the treatment of neuronal ceroid lipofuscinosis type 2.

The sponsorship was transferred to BioMarin International Limited, Ireland, in October 2015.

Recombinant human tripeptidyl-peptidase 1 has been authorised in the EU as Brineura since 30 May 2017.

Key facts

Active substance
Recombinant human tripeptidyl-peptidase 1
Intended use
Treatment of neuronal ceroid lipofuscinosis type 2
Orphan designation status
EU designation number
Date of designation

BioMarin International Limited
County Cork
Tel. +353 21 500 8600

Review of designation

On 26 April 2017, the Committee for Orphan Medicinal Products (COMP) completed its review of the designation EU/3/13/1118 for Brineura (cerliponase alfa1) as an orphan medicinal product for the treatment of neuronal ceroid lipofuscinosis2. 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 methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained3

1 Previously known as recombinant human tripeptidyl-peptidase 1.

2During this review the original orphan indication (neuronal ceroid lipofuscinosis type 2) was expanded to include all types of neuronal ceroid lipofuscinosis.

3The 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 the same 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: Brineura (cerliponase alfa) for the treatment of neuronal ceroid lipofuscinosis (PDF/79.09 KB)

    First published: 22/06/2017
    Last updated: 22/06/2017

  • 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.

    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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