Cerliponase Alfa

Cerliponase alfa is used to slow the loss of ambulation in symptomatic pediatric patients aged 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency, a form of Batten disease.

Cerliponase alfa is available under the following different brand names: Brineura and recombinant human tripeptidyl peptidase 1 (rhtpp1).

Dosage Forms and Strengths

Injection for Intraventricular Administration Kit

• Cerliponase alfa: 150mg/5mL (2 vials)
• Intraventricular electrolytes: 5mL (1 vial)
• Also includes an administration kit
• Dosage Considerations – Should be Given as Follows:

CLN2 Disease

• Indicated to slow the loss of ambulation in symptomatic pediatric patients aged 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency, a form of Batten disease
• Children 3 years and older: 300 mg by intraventricular infusion at a rate of 2.5 mL/hour once every other week
• Children under 3 years: Safety and efficacy not established
• Follow with an intraventricular infusion of electrolytes at a rate of 2.5 mL/hour
• The total infusion time is approximately 4.5 hours for cerliponase alfa plus the electrolytes

Pretreatment

• Pretreatment with antihistamines with or without antipyretics or corticosteroids is recommended 30-60 minutes before intraventricular infusion

Common side effects of cerliponase alfa include:

• Fever
• ECG abnormalities
• Decreased CSF protein
• Vomiting
• Seizures
• Hypersensitivity
• Increased CSF protein
• Hematoma
• Headache
• Irritability
• Increased lymphocytes (pleocytosis)
• Device-related infection
• Slow heart rate
• Feeling jittery
• Low blood pressure (hypotension)
• Postmarketing side effects of cerliponase alfa reported include:
• Bacterial meningitis

This document does not contain all possible side effects and others may occur. Check with your physician for additional information about side effects.

If your doctor has directed you to use this medication, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider or pharmacist first.

• Cerliponase alfa has no listed severe interactions with other drugs.
• Cerliponase alfa has no listed serious interactions with other drugs.
• Cerliponase alfa has no listed moderate interactions with other drugs.
• Cerliponase alfa has no listed mild interactions with other drugs.
   

This document does not contain all possible interactions. Therefore, before using this product, tell your doctor or pharmacist of all the products you use. Keep a list of all your medications with you, and share the list with your doctor and pharmacist. Check with your physician if you have health questions or concerns.

Warnings
 

This medication contains cerliponase alfa. Do not take Brineura or recombinant human tripeptidyl peptidase 1 (rhtpp1) if you are allergic to cerliponase alfa or any ingredients contained in this drug.

Keep out of reach of children. In case of overdose, get medical help or contact a Poison Control Center immediately.

Contraindications

• Any sign or symptom of acute, unresolved localized infection on or around the device insertion site (e.g. cellulitis or abscess); or suspected or confirmed central nervous system (CNS) infection (e.g. cloudy cerebrospinal fluid [CSF] or positive CSF gram stain, or meningitis)
• Any acute intraventricular access device-related complication (e.g., leakage, extravasation of fluid, or device failure)
• Patients with ventriculoperitoneal shunts

Effects of Drug Abuse

• No information available

Short-Term Effects

• See "What Are Side Effects Associated with Using Cerliponase Alfa?”

Long-Term Effects

• See "What Are Side Effects Associated with Using Cerliponase Alfa?”

Cautions

• Must be administered using an 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 before each infusion
• Monitor vital signs before infusion starts, periodically during infusion, and post-infusion in a healthcare setting
• Perform ECG monitoring during infusion in patients with a history of bradycardia, conduction disorder, or 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
• Hypersensitivity reactions reported during the infusion or within 24 hours of completion; observe the patient during and after infusion; inform caregivers of signs and symptoms of anaphylaxis and instruct them to seek immediate medical care if these occur

Intraventricular access complications

• Bacterial meningitis requiring antibiotic treatment and removal of the device reported during postmarketing use; signs and symptoms of infections may not be readily apparent in patients with CLN2 disease; therapy should be administered by, or under the direction of, a physician experienced in intraventricular administration to reduce risk of infectious complications
• Obtain a sample of CSF for cell count and culture before each infusion and when clinically indicated
• Do not administer therapy if there are localized signs of infection on or around the device insertion site, such as erythema, tenderness, or discharge or suspected or confirmed CNS infection (e.g. cloudy CSF or positive CSF gram stain, or meningitis)
• Healthcare providers should be vigilant for the development of signs and symptoms of infection, including meningitis, during treatment and monitor device insertion site for signs of infection
• Consult a neurosurgeon for any complications with the implanted device; in case of device-related complication, discontinue the infusion and refer to device labeling for further instructions

Pregnancy and Lactation

There are no available data on cerliponase alfa use in pregnant women to inform a drug-associated risk of pregnancy-related outcomes. Consult your doctor.

It is unknown if cerliponase alfa is distributed in human breast milk. Consult your doctor before breastfeeding.