Drug updated on 11/13/2023
| Dosage Form | Injection (intraventricular: 150 mg/5 mL (30 mg/mL)) |
| Drug Class | Hydrolytic lysosomal N-terminal tripeptidyl peptidases |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Brineura (cerliponase alfa) Prescribing Information. | 2020 | BioMarin Pharmaceutical Inc., Novato, CA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Clinical review report: cerliponase alfa (Brineura). | 2019 | CADTH |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients. | 2021 | Orphanet Journal of Rare Diseases |