BRINEURA
(Cerliponase alfa)PreclinicalProtein / enzyme
BioMarin PharmaceuticalBRINEURA is a protein / enzyme that works as a TPP1 Enzyme Replacement.[2] It is being investigated across 1 indication, with its most advanced program in Preclinical.[1]
BRINEURA is one of 3 enzyme replacement programs targeting Batten Disease. It is currently in Preclinical.
1
Indications
2
Total Trials
2
Active Trials
2
Competitors
1
FDA Application
NDA/BLA/ANDA regulatory filings
Pipeline
Status by Indication[3][1]
Indication
Competition
Competitive Landscape
2 competitors in Batten Disease
#DrugCompanyPhaseTrials
1Aromatase Inhibitor
PfizerPhase 2
RG6026
RochePhase 1
BRINEURA
BioMarin PharmaceuticalPreclinical
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
BLA 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
- Preclinical[1]
- Company