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.
Recombinant human tripeptidyl-peptidase 1
|Disease / condition||
Treatment of neuronal ceroid lipofuscinosis type 2
|Date of first decision||
|EU designation number||
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.
Sponsor's contact details
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.