Section 12 · Monograph
Cerliponase alfa
Reviewed Nov 2026FDA label
BrineuraEnzyme replacement (TPP1) · intraventricular
- Forms
- Intraventricular: 150mg/5mL vials with Intraventricular Electrolytes
- Dose
- CLN2: 300mg ICV every other week (age 2+) over about 4.5h; under 2 years lower doses (100mg birth to under 6 months; 150mg 6 months to under 1 year; 200mg x4 then 300mg for 1 to under 2 years)
- MOA
- Replaces tripeptidyl peptidase 1 (TPP1)
Black Box Warning
- Hypersensitivity reactions including anaphylaxis: life-threatening reactions have occurred with enzyme replacement therapies — appropriate medical support must be readily available during infusion
Indications
- To slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiencyFDA-labeled
Mechanism of Action
- Recombinant human TPP1 that is taken up by neurons and degrades lysosomal storage material (proenzyme activated in lysosomes)
Full Dosing
Adult / usual
- 300mg every other week by intraventricular infusion followed by Intraventricular Electrolytes, over about 4.5 hours, in patients 2 years and older
- Under 2 years: birth to under 6 months 100mg; 6 months to under 1 year 150mg; 1 to under 2 years 200mg for the first 4 doses then 300mg
- Pre-treat with antihistamines with or without antipyretics or corticosteroids 30-60 minutes before infusion; give only by trained staff via the dedicated access device
Contraindications
- Acute intraventricular access device-related complications (e.g., leakage, device failure or device-related infection)
- Patients with ventriculoperitoneal shunts
Drug Interactions
- No formal drug interaction studies
Adverse Effects
- Pyrexia, ECG abnormalities, vomiting
- CSF protein and cell count abnormalities
- Seizures and hypersensitivity
- Device-related infection and meningitis
- Bradycardia and hypotension during infusion
Monitoring Parameters
- Vital signs and ECG during infusion (especially with cardiac conduction abnormalities)
- CSF cell count and culture if infection is suspected; inspect device site
- Ambulation and clinical rating scale (CLN2 Clinical Rating Scale)
Pregnancy & Lactation
- No adequate data
- Unknown whether present in human milk