Cerliponase Alfa
Cerliponase Alfa is an enzyme replacement therapy designed to treat a specific form of Batten disease, a rare inherited neurodegenerative disorder. It works by replacing a deficient enzyme in the brain, administered directly into the cerebrospinal fluid. This medication is specifically indicated for pediatric patients with confirmed enzyme deficiency.
Cerliponase Alfa is an enzyme replacement therapy for a rare form of Batten disease in children, administered directly into the brain.
Key facts at a glance
- RxNorm
- 1922436
- Generic
- Cerliponase Alfa
- Class
- pin-variant
- Brands
- Brineura
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Cerliponase Alfa is indicated for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as classic late-infantile Batten disease, in pediatric patients.
Cerliponase Alfa is a recombinant human tripeptidyl peptidase-1 (TPP1) enzyme that replaces the deficient TPP1 enzyme in CLN2 disease. It is administered via intracerebroventricular infusion, allowing direct delivery to the central nervous system to reduce accumulation of storage materials in neurons.
This medication requires administration by trained healthcare professionals in appropriate clinical settings. Patients should be monitored for signs of device complications, hypersensitivity reactions, and CSF parameters. Regular monitoring of neurological status and disease progression is essential during treatment.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 27, 2026. · Read our editorial policy