BRINEURA (cerliponase alfa)
Brineura (cerliponase alfa) is a hydrolytic lysosomal N-terminal tripeptidyl peptidase indicated for use in pediatric patients. It is specifically approved to slow the loss of ambulation in patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency. This neurodegenerative condition is characterized by a progressive decline in motor function caused by the deficiency of a specific lysosomal enzyme.
How BRINEURA Works
Cerliponase alfa is a proenzyme that is taken up by target cells in the central nervous system and translocated to the lysosomes via the Cation Independent Mannose-6-Phosphate Receptor. Once inside the lysosome, the drug is activated into a proteolytic form that cleaves tripeptides from the N-terminus of proteins. This enzymatic activity addresses the deficiency of endogenous tripeptidyl peptidase-1 (TPP1), which normally catabolizes polypeptides. By replacing the missing enzyme, the treatment helps prevent the accumulation of lysosomal storage materials that lead to progressive motor decline.
Details
- Status
- Prescription
- First Approved
- 2017-04-27
- Patent Cliff
- 2031
- Revenue
- $52M (Q4-2025)
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
BRINEURA Approval History
What BRINEURA Treats
1 indicationsBRINEURA is approved for 1 conditions since its original approval in 2017. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Neuronal Ceroid Lipofuscinosis
BRINEURA Boxed Warning
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate BRINEURA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reacti...
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate BRINEURA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue BRINEURA and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ]. WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. ( 5.1 ) Initiate BRINEURA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue BRINEURA and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )
Clinical Trial Registry
1 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05152914 | STUDY00001444 | Ph 1, Ph 2 | active not recruiting | Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2 |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
BRINEURA FDA Label Details
Indications & Usage
FDA Label (PDF)BRINEURA is indicated for the treatment of Neuronal Ceroid Lipofuscinosis.
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration...
Pro Intelligence Preview
Deep insights for BRINEURA
Revenue Insights
- • Q4-2025: $52M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2031
- • Generic/biosimilar risk
Trial Analysis
- • Clinical trial tracking
- • Development stage analysis
Competitive Landscape
- • Competitor tracking
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.