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ALIMENTARY TRACT AND METABOLISM › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › Enzymes
cerliponase alfa
A16AB17
Forms & Strengths
- Intracerebroventricular solution: 300 mg/10 mL (includes 2 mL of reconstitution buffer)
Adult Dosing
- Not applicable (indicated for a pediatric neurodegenerative condition)
Pediatric Dosing
- Pediatric patients (3 years and older): 300 mg administered once every other week via intracerebroventricular (ICV) infusion
- Each infusion should be followed by an infusion of the reconstitution buffer (2 mL)
- Administer the ICV infusion over approximately 4 hours using an infusion pump
Indications
- Treatment of symptomatic neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency
Mechanism of Action
- Recombinant human tripeptidyl peptidase 1 (rhTPP1) replaces the deficient lysosomal enzyme TPP1
- Cleaves tripeptidyl peptides from the N-terminus of proteins, reducing storage of subunit c of mitochondrial ATP synthase in the central nervous system
Contraindications
- History of acute life-threatening hypersensitivity to cerliponase alfa or any of its components
- Presence of a ventriculoperitoneal shunt or other contraindicated intracranial hardware
- Active ICV device-related infection
Adverse Reactions
- Fever
- Hypersensitivity reactions (including anaphylaxis)
- Device-related complications (infection, malfunction, leakage)
- Ventricle enlargement
- Seizures
- Pleocytosis in cerebrospinal fluid
- Vomiting
- Hypotension
- Bradycardia
Drug Interactions
- Formal clinical drug-drug interaction studies have not been performed with cerliponase alfa
- As a recombinant lysosomal enzyme, it is not metabolized by cytochrome P450 enzymes, minimizing potential for CYP-mediated interactions
Curated Content: Needs Vetting Before Put to Clinical Use