BLAINJECTIONINJECTABLEPriority Review
Approved
Apr 2017
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
1
Data completeness
4/8 — 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. First approved in 2017.
Drug data last refreshed 13h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
BRINEURA (cerliponase alfa) is a recombinant enzyme replacement therapy for CLN2 disease, a rare lysosomal storage disorder caused by tripeptidyl peptidase-1 (TPP1) deficiency. The drug restores TPP1 activity in the central nervous system, slowing progressive neurodegeneration by enabling catabolism of accumulated polypeptides within lysosomes. Delivered via intracerebroventricular infusion, it targets the root cause of motor and cognitive decline in affected patients.
Peak
Peak lifecycle stage with limited competitive pressure (30% threat level) suggests stable, specialized commercial operations focused on rare disease patient identification and outcomes management.
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
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (1)
Cerliponase Alfa Observational Study in the US
Started Aug 2020
35 enrolled
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Career Relevance
Working on BRINEURA requires expertise in rare disease commercialization, orphan drug reimbursement, and highly specialized patient identification in a fragmented neurology market. This is a career opportunity for professionals seeking deep expertise in a defined therapeutic area with stable, long-term commercial potential and minimal competitive disruption.
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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.