BLAINJECTIONINJECTABLEPriority Review
Approved
Apr 2017
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
1
Data completeness
5/8 — Partial
Priority Review
BRINEURA (cerliponase alfa) by BioMarin Pharmaceutical is cln2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (tpp1), which catabolizes polypeptides in the cns. Approved for neuronal ceroid lipofuscinosis type 2. First approved in 2017.
Drug data last refreshed 20h ago
Mechanism of Action
CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1), which catabolizes polypeptides in the CNS. TPP1 has no known substrate specificity. Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials…
Indications (1)
Clinical Trials (1)
Cerliponase Alfa Observational Study in the US
Started Aug 2020
35 enrolled
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.