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.
- anaphylactoid reactions
- antibody formation
- sinus tachycardia
- ST-T wave changes
- cerebrospinal fluid (CSF) assessment
- cardiac disease
- requires an experienced clinician
- serious hypersensitivity reactions or anaphylaxis
- ventriculoperitoneal (VP) shunt
There are no drug interactions associated with Cerliponase alfa products.