Usual Pediatric Dose for Neuronal Ceroid Lipofuscinosis

3 years or older: 300 mg by intraventricular infusion once every other week

Comments:

• Pretreatment with antihistamines with or without antipyretics or corticosteroids should occur 30 to 60 minutes prior to the start of infusion.
• Prior to each infusion, inspect the scalp for signs of intraventricular access device leakage, failure, or potential infection.
• Prior to each infusion, and when clinically indicated, obtain a sample of CSF for cell count and culture.
• Infusion rate 2.5 mL/hr; administer with the B Braun Perfusor Space Infusion Pump System, follow infusion with Intraventricular Electrolytes provided in the administration kit.

Use: To slow the loss of ambulation in symptomatic pediatric patients 3 years or older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Precautions

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 CNS infection (e.g. cloudy CSF or positive CSF gram stain, or meningitis)
• Any acute intraventricular access device-related complication (e.g., leakage, extravasation of fluid, or device failure)
• Ventriculoperitoneal shunts

Safety and efficacy have not been established in patients younger than 3 years.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• Administered into CSF by intraventricular infusion via a surgically implanted reservoir and catheter (intraventricular access device).
• Must only be administered by the intraventricular route, using the provided administration kit; each vial of this drug and electrolytes is for single use only.
• Each infusion consists of 10 mL of this drug followed by 2 mL of intraventricular electrolytes; administer using an infusion set with a 0.2 micron inline filter.
• Infuse both this drug and electrolytes at an infusion rate of 2.5 mL/hr; complete infusion, including required electrolytes, is about 4.5 hours.
• The Codman HOLTER RICKHAM Reservoirs should be used with the Codman Ventricular Catheter.
• To be administered with the B Braun Perfusor Space Infusion System; consult the manufacturer product information for additional information.
• The intraventricular access device must be implanted prior to the first infusion and the first dose administered at least 5 to 7 days after device implantation.
• Aseptic technique must be strictly enforced during preparation and administration.
• To be administered by, or under the direction of a physician knowledgeable in intraventricular administration.
• The manufacturer product information should be consulted for direction in the intraventricular infusion procedure.

Storage requirements:

• Store drug vials and intraventricular electrolyte injection vials upright in original carton in a freezer (-25C to -15C); protect from light
• Store administration kit separately from drug vials and intraventricular electrolytes; do not freeze
• Thawed Product: Use thawed drug and Intraventricular Electrolytes immediately; if not used immediately, store

unopened vials in the refrigerator at 2C to 8C (36F to 46F) and use within 24 hours

• Product in Syringes: Use product held in labeled syringes immediately; if not used immediately, store product held in labeled

syringes in the refrigerator at 2C to 8C (36F to 46F) up to 4 hours prior to infusion

Reconstitution/preparation techniques:

• The manufacturer product information should be consulted.

Monitoring:

• Cardiovascular: Monitor vital signs (e.g., blood pressure, heart rate) prior to infusion, 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 with structural heart disease; perform regular 12-lead ECG evaluations every 6 months in patients without cardiac abnormalities
• Hypersensitivity: Observe patients closely during and after infusion for signs/symptoms of allergic reactions, including anaphylaxis
• Nervous System: Prior to each infusion, inspect the scalp for signs of intraventricular access device leakage, failure, or potential infection; prior to each infusion, and when clinically indicated, obtain a sample of CSF for cell count and culture

Patient advice:

• Advise patients/caregivers to immediately contact their healthcare provider if any signs of infection occur.
• Advise patients/caregivers to immediately contact their healthcare provider if hypotension and/or bradycardia occur during or following infusion.
• Advise patients/caregivers to seek medical attention if any signs/symptoms of a hypersensitivity reaction (e.g., fever, vomiting, irritability) or signs/symptoms of anaphylaxis occur.

Frequently asked questions

• What condition is Brineura used to treat?