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 4h ago · AI intelligence enriched 2d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
BRINEURA (cerliponase alfa) is an enzyme replacement therapy for CLN2 disease, a rare neurodegenerative lysosomal storage disorder caused by tripeptidyl peptidase-1 (TPP1) deficiency. It delivers recombinant human TPP1 directly into the CNS via intra-cerebroventricular infusion, where it is activated in lysosomes to cleave and metabolize accumulated polypeptides. This halts progressive motor decline in children with this uniformly fatal condition.
Peak
As a peak-stage orphan drug with a highly specialized patient population and intra-cerebroventricular delivery mechanism, BRINEURA supports focused, high-touch commercial teams with expertise in rare pediatric neurology and patient support infrastructure.
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
BRINEURA represents a career opportunity in high-touch, evidence-based orphan drug commercialization where success depends on deep clinical expertise, strong relationships with pediatric neurology centers, and patient/caregiver support infrastructure rather than volume-driven field sales. Working on this product builds specialized expertise in ultra-rare disease management, intra-cerebroventricular drug delivery, and value-based contracting with academic medical centers.
Worked on BRINEURA at BioMarin Pharmaceutical? Share your interview experience or compensation data (+7 days Pro)
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.