Drug updated on 9/4/2024
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.
Latest News
Summary
- Brineura (cerliponase alfa) is 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.
- This summary is based on the review of one randomized controlled trial(s). [1]
- The median time until a 2-point decline in motor-language score was not reached for patients treated with cerliponase alfa, whereas historical controls reached this decline in a median of 345 days.
- Treated patients exhibited a mean unadjusted rate of decline in motor-language score of 0.27 ± 0.35 points per 48-week period, compared to 2.12 ± 0.98 points per 48-week period in historical controls, with a mean difference of 1.85 points (P<0.001).
- The study focused on children aged 3 to 16 years with CLN2 disease, with no specific differences in effectiveness outcomes among subgroups within this population mentioned.
- Common adverse events in children aged 3 to 16 years with CLN2 disease treated with cerliponase alfa included convulsions, pyrexia, vomiting, hypersensitivity reactions, and failure of the intraventricular device.
- Serious adverse events involved device-related infections in 2 patients, which necessitated antibiotic treatment and device replacement.
- There is no population types or subgroups information available in the reviewed studies.
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. | 24Subjects F: 62% M: 38% | 2018 | The New England Journal of Medicine |
Sex Distribution:
F:62%
M:38%
24Subjects
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 |