Drug updated on 4/29/2024
| 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 |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Study of Intraventricular Cerliponase Alfa for CLN2 Disease. | Data not availableSubjects F: null% M: null% | 2018 | The New England Journal of Medicine |
Sex Distribution:
Year:
2018
Source:The New England Journal of Medicine
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| HSE guidelines for the treatment of neuronal ceroid lipofuscinosis Type 2 (CLN2) with Cerliponase alfa (Brineura®). | 2024 | HSE Guidelines |
| Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients. | 2021 | Orphanet Journal of Rare Diseases |
| Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2. | 2019 | National Institute for Health and Care Excellence |