BRINEURA
(Cerliponase alfa)Phase 2Protein / enzyme
BioMarin PharmaceuticalBRINEURA is a protein / enzyme that works as a TPP1 Enzyme Replacement.[2] It is being investigated across 2 indications, with its most advanced program in Phase 2.[1]
BRINEURA is one of 3 enzyme replacement programs targeting Batten Disease. It is currently in Phase 2.
2
Indications
2
Total Trials
2
Active Trials
2
Competitors
7
FDA Applications
NDA/BLA/ANDA regulatory filings
Pipeline
Status by Indication[3][1]
Competition
Competitive Landscape
3 competitors in Disease Progression
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Timeline
Expected Milestones
Clinical
Trials by Indication[1]
Disease Progression1 trial
Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2
Active, not recruiting·—·Phase 1/2·NCT05152914·5 enrolled
Regulatory
FDA Approval Timeline[4]
Apr 27, 2017
BRINEURA
NDA BLA761052
Apr 27, 2017
BRINEURA
BLA BLA761052
Dec 11, 2018
BRINEURA
NDA BLA761052
Dec 6, 2019
BRINEURA
NDA BLA761052
Mar 27, 2020
BRINEURA
NDA BLA761052
Jul 10, 2024
BRINEURA
NDA BLA761052
Jul 24, 2024
BRINEURA
NDA BLA761052
Sources
References
- [1]
- [2]
- [3]Open Targets Platform — Drug–disease associations for Cerliponase alfa. EMBL-EBI & Wellcome Sanger Institute.
- [4]
- [5]
Details
Drug Info
Quick Facts
- Mechanism of Action
- TPP1 Enzyme Replacement[2]
- Modality
- Protein / enzyme[2]
- Highest Phase
- Phase 2[1]
- Company