Cerliponase alfa

Cerliponase alfa Brand Name– Brineura

What is Cerliponase alfa

Cerliponase alfa is an enzyme replacement therapy for the management of patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease. CLN2 is a rare, genetic, fatal neurodegenerative disease caused by a deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1).

Cerliponase alfa is a recombinant form of TPP1 that is administered into the cerebrospinal fluid (CSF) by intraventricular infusion using a specific surgically implanted intraventricular access device.

Patients must be monitored closely during administration for cardiac adverse events and/or hypersensitivity reactions.

Indications & Dosage

• late-infantile neuronal ceroid lipofuscinosis type 2

For the treatment of patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease

NOTE: The FDA has designated cerliponase alfa as an orphan drug for this indication.

Side Effects

1. anaphylactoid reactions
2. antibody formation
3. bradycardia
4. fever
5. headache
6. hematoma
7. hypotension
8. hypoxia
9. infection
10. irritability
11. seizures
12. sinus tachycardia
13. ST-T wave changes
14. vomiting

Monitoring Parameters

• cerebrospinal fluid (CSF) assessment
• ECG

Contraindications

• bradycardia
• breast-feeding
• cardiac disease
• infection
• pregnancy
• requires an experienced clinician
• serious hypersensitivity reactions or anaphylaxis
• ventriculoperitoneal (VP) shunt

Interactions

There are no drug interactions associated with Cerliponase alfa products.