Brineura
What Brineura is used for
Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Approved under exceptional circumstances: full data cannot be collected, usually because the disease is very rare.
Key facts
- Brand
- Brineura
- Active substance
- cerliponase alfa
- Disease area
- Neuronal Ceroid-Lipofuscinoses
- Medicine group
- Other alimentary tract and metabolism products
- ATC code
- A16AB
- Marketing authorisation holder
- BioMarin International Limited
- Authorised in the EU since
- 2017
Official information
In the USA
Same active substance: Brineura
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Brineura used for?
Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency
What is the active substance in Brineura?
Brineura contains cerliponase alfa.
Who makes Brineura?
The EU marketing authorisation holder is BioMarin International Limited.
Is Brineura an orphan medicine?
Yes. Brineura is designated an orphan medicine in the EU, for a rare disease (Neuronal Ceroid-Lipofuscinoses).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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