Cerliponase Alfa
Brineura (cerliponase alfa) is a prescription enzyme replacement therapy used to slow the loss of walking ability in pediatric and adult patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a rare inherited neurodegenerative disorder also known as Batten disease. This rare condition typically affects children ages 2-4 years and causes progressive decline in motor function, language, and vision.
Brineura is an enzyme replacement therapy for CLN2/Batten disease that slows loss of motor function in affected children and adults.
Key facts at a glance
- RxNorm
- 1922454
- Generic
- Brineura
- Class
- brand-name
- Brands
- —
- 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.
Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a rare autosomal recessive lysosomal storage disorder characterized by deficiency of the enzyme tripeptidyl peptidase 1 (TPP1). It is specifically indicated to slow loss of ambulation in symptomatic patients.
Brineura is a recombinant form of the human enzyme tripeptidyl peptidase 1 (TPP1). It replaces the deficient or absent TPP1 enzyme in CLN2 patients, helping to break down accumulating storage materials in neurons. The medication is administered via intraventricular infusion directly into the cerebrospinal fluid to reach the central nervous system.
Brineura is administered by healthcare professionals via intraventricular infusion every other week. Common side effects may include fever, vomiting, seizures, and infusion-related reactions. Patients should be monitored for signs of infection before each infusion. This medication must be stored and handled according to specific guidelines and is available only through a restricted program.
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 September 3, 2026. · Read our editorial policy