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Cerliponase Alfa

Class: Enzymes
Brands: Brineura


Cerliponase alfa is an enzyme.

Uses for Cerliponase Alfa

Cerliponase alfa has the following uses:

Cerliponase alfa is a hydrolytic lysosomal N-terminal tripeptidyl peptidase indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.1

Cerliponase Alfa Dosage and Administration


Cerliponase alfa is available in the following dosage form(s) and strength(s):

Injection: Cerliponase alfa 150 mg/5 mL (30 mg/mL) solution, two single-dose vials per carton co-packaged with Intraventricular Electrolytes Injection 5 mL in a single-dose vial.


It is essential that the manufacturer's labeling be consulted for more detailed information on dosage and administration of this drug. Dosage summary:

  • Aseptic technique must be strictly observed during preparation and administration. Cerliponase alfa should be administered by, or under the direction of a physician knowledgeable in intraventricular administration. Cerliponase alfa is administered to the cerebrospinal fluid (CSF) by infusion via a surgically implanted reservoir and catheter.1

  • Pre-treatment of patients with antihistamines with or without antipyretics or corticosteroids is recommended 30 to 60 minutes prior to the start of infusion.1

  • The recommended dosage is 300 mg administered once every other week as an intraventricular infusion followed by infusion of Intraventricular Electrolytes over approximately 4.5 hours.1

  • For complete information on preparation, specific intraventricular access device for use, and administration, see the full prescribing information.1

Cautions for Cerliponase Alfa


  • Acute intraventricular access device-related complications (e.g., leakage, device failure, device-related infection).1

  • Patients with ventriculoperitoneal shunts.1


Intraventricular Access Device-Related Complications

Cerliponase alfa must be administered using aseptic technique to reduce the risk of infection. Healthcare professionals should inspect the scalp for skin integrity to ensure the intraventricular access device is not compromised prior to each infusion. 1

Cerliponase alfa is contraindicated if there are signs of acute intraventricular access device-related complications (e.g., leakage, device failure or signs of device-related infection such as swelling, erythema of the scalp, extravasation of fluid, or bulging of the scalp around or above the intraventricular access device). In case of intraventricular access device complications, discontinue the cerliponase alfa infusion and refer to the device manufacturer’s labeling for further instructions.1

The signs and symptoms of device-related infections may not be apparent, therefore, CSF samples should routinely be sent for testing to detect subclinical device infections.1

In clinical studies with cerliponase alfa, intraventricular access device-related infections were observed in two patients. In each case, antibiotics were administered, the intraventricular access device was replaced, and the patient continued on cerliponase alfa treatment.1

Material degradation of the intraventricular access device reservoir may occur after approximately 105 perforations of the intraventricular access device. The intraventricular access device may require replacement as soon as, or prior to, 105 administrations of cerliponase alfa, equating to approximately 4.3 years of regular administrations.1

Cardiovascular Adverse Reactions

Monitor vital signs (blood pressure, heart rate) before infusion starts, periodically during infusion, and post-infusion in a healthcare setting. Upon completion of the infusion, clinically assess the patient status. Continued observation may be necessary if clinically indicated.1

Perform electrocardiogram (ECG) monitoring during infusion in patients with a history of bradycardia, conduction disorder, or with structural heart disease, as some patients with CLN2 disease may develop conduction disorders or heart disease. In patients without cardiac abnormalities, regular 12-lead ECG evaluations should be performed every 6 months.1

In the clinical studies, hypotension was reported in 2 (8%) patients, which occurred during or up to eight hours after cerliponase alfa infusion. Patients did not require alteration in treatment, and reactions resolved spontaneously or after intravenous fluid administration.1

Hypersensitivity Reactions

Hypersensitivity reactions have been reported in cerliponase alfa-treated patients during the clinical studies. A total of 11 (46%) patients experienced hypersensitivity reactions during the infusion or within 24 hours of completion of the infusion. The signs and symptoms observed concomitantly with hypersensitivity reactions included pyrexia, vomiting, pleocytosis or irritability. Patients were routinely pre-medicated with antihistamines with or without antipyretics or corticosteroids, prior to infusion of cerliponase alfa.1

Due to the potential for anaphylaxis, appropriate medical support should be readily available when cerliponase alfa is administered. If anaphylaxis occurs, immediately discontinue the infusion and initiate appropriate medical treatment. Observe patients closely during and after the infusion. Inform patients/caregivers of the signs and symptoms of anaphylaxis, and instruct them to seek immediate medical care should signs and symptoms occur.1

The management of hypersensitivity reactions should be based on the severity of the reaction and may include temporarily interrupting the infusion, and/or treatment with antihistamines, antipyretics, and/or corticosteroids. If a severe hypersensitivity reaction occurs, immediately discontinue the infusion and initiate appropriate medical treatment.1

Specific Populations


There are no available data on cerliponase alfa use in pregnant women to inform a drug-associated risk of pregnancy-related outcomes. Animal reproduction studies have not been conducted using cerliponase alfa.1

The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.1


There are no data on the presence of cerliponase alfa in human milk, the effects on the breastfed child, or the effects on milk production. The lack of clinical data during lactation precludes a clear determination of the risk of cerliponase alfa to an infant during lactation; therefore, the development and health benefits of breastfeeding should be considered along with the mother’s clinical need for cerliponase alfa and any potential adverse effects on the breastfed infant from cerliponase alfa or from the underlying maternal condition.1

Pediatric Use

Safety and effectiveness of cerliponase alfa have been established in pediatric patients 3 years of age and older. Pediatric use of cerliponase alfa to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with CLN2 is supported by a nonrandomized, single-arm, dose escalation clinical study with extension in patients with CLN2 disease and compared to untreated patients with CLN2 disease from an independent natural history cohort. Safety and effectiveness in patients less than 3 years of age have not been established.1

Common Adverse Effects

Most common adverse reactions (≥8%) are: pyrexia, ECG abnormalities, decreased CSF protein, vomiting, seizures, hypersensitivity, increased CSF protein, hematoma, headache, irritability, pleocytosis, device-related infection, bradycardia, feeling jittery, and hypotension.1

Drug Interactions

Specific Drugs

It is essential that the manufacturer's labeling be consulted for more detailed information on interactions with this drug, including possible dosage adjustments. Interaction highlights:

Please see product labeling for drug interaction information.1


Mechanism of Action

CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme TPP1, which catabolizes polypeptides in the CNS. TPP1 has no known substrate specificity. Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials normally metabolized by this enzyme in the central nervous system (CNS), leading to progressive decline in motor function.1

Cerliponase alfa (rhTTP1), a proenzyme, is taken up by target cells in the CNS and is translocated to the lysosomes through the Cation Independent Mannose-6-Phosphate Receptor (CI-MPR, also known as M6P/IGF2 receptor). Cerliponase alfa is activated in the lysosome and the activated proteolytic form of rhTPP1 cleaves tripeptides from the N-terminus of proteins.1

Advice to Patients

Patient Counseling Information

Advise patients and caregivers of the risk of device-related infections. If any signs of infection are present, instruct patients to immediately contact their healthcare provider.1

Advise patients and caregivers that hypotension and/or bradycardia may occur during and following the infusion of cerliponase alfa. Instruct patients immediately to contact their healthcare provider if these reactions occur. 1

Advise patients and caregivers that hypersensitivity reactions related to cerliponase alfa treatment, including fever, vomiting, and irritability may occur. Due to the potential for anaphylaxis, inform patients and caregivers of the signs and symptoms of anaphylaxis, and instruct them to seek immediate medical care should signs and symptoms occur. 1

Additional Information

AHFS First Release. For additional information until a more detailed monograph is developed and published, the manufacturer's labeling should be consulted. It is essential that the manufacturer's labeling be consulted for more detailed information on usual uses, dosage and administration, cautions, precautions, contraindications, potential drug interactions, laboratory test interferences, and acute toxicity.


Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.

Cerliponase Alfa


Dosage Forms


Brand Names



Injection, for intraventricular use only

150 mg/5 mL

Brineura (available as a kit containing intraventricular electrolytes injection, syringes, needles, extension line, infusion set, and port needle)

BioMarin Pharmaceutical Inc.

AHFS Drug Information. © Copyright 2017, Selected Revisions May 15, 2017. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.


1. BioMarin Pharmaceutical Inc. Brineura (cerliponase alfa) intraventricular prescribing information. 2017 Apr.