Cerliponase alfa

What is Cerliponase alfa?[edit | edit source]

Cerliponase alfa (Brineura) is a hydrolytic lysosomal N-terminal tripeptidyl peptidase used 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.

This medicine used 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.

CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (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.
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.

This medicine cannot be used in patients with:

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.

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

Recommended Dosageː

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.

Administration

Aseptic technique must be strictly observed during preparation and administration.
Brineura should be administered by, or under the direction of, a physician experienced in intraventricular administration.
Prior to each infusion, inspect the scalp for signs of intraventricular access device leakage, failure or potential infection.
Obtain a sample of CSF for cell count and culture prior to each infusion and if clinically indicated.
Brineura is administered to the cerebrospinal fluid (CSF) by infusion via a surgically implanted reservoir and catheter.
Brineura is intended to be administered with the B Braun Perfusor Space Infusion Pump System.
Administer Brineura and the Intraventricular Electrolytes using the provided Administration Kit for use with Brineura components.
Each vial of Brineura and Intraventricular Electrolytes is intended for a single dose only.
Each infusion consists of 10 mL of Brineura followed by 2 mL of Intraventricular Electrolytes.
The complete infusion must be administered using an infusion set with a 0.2 micron inline filter.
The Intraventricular Electrolytes are used to flush the infusion line, port needle, and intraventricular access device in order to fully administer Brineura and to maintain patency of the intraventricular access device.

This medicine is available in fallowing doasage form:

As Injection: Brineura 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.

This medicine is available in fallowing brand namesː

The most common side effects of this medicine include:

Bacterial meningitis requiring antibiotic treatment and removal of the device was reported during postmarketing use of Brineura. Monitor the device insertion site for signs of infection.
Intraventricular access device-related complications were reported (e.g., device leakage, device failure extravasation of CSF fluid, or bulging of the scalp around or above the intraventricular access device). Consult a neurosurgeon for any complications with the implanted device. In case of device-related complication, discontinue the infusion and refer to the device labeling for further instructions.
Monitor vital signs before, during, and post-infusion. Monitor Electrocardiogram (ECG) in patients with a history of bradycardia, conduction disorder, or with structural heart disease, during the infusion. In patients without cardiac abnormalities, perform regular 12-lead ECG evaluations every 6 months.
Hypersensitivity reactions including anaphylaxis have been reported in Brineura-treated patients. Observe patients during and after the infusion. If a severe hypersensitivity reaction occurs, immediately stop the infusion and initiate appropriate treatment.

In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

Overdose related information is also available online at poisonhelp.org/help.
In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

There are no available data on Brineura use in pregnant women to inform a drug-associated risk of pregnancy-related outcomes.

Safety and effectiveness of Brineura have been established in pediatric patients 3 years of age and older.
Safety and effectiveness in patients less than 3 years of age have not been established.

Active ingredient:

Brineura (cerliponase alfa) Injection and Intraventricular Electrolytes Injection:

Store upright in a freezer (‑25°C to ‑15°C) in original carton to protect from light.

Administration Kit for use with Brineura:

Cerliponase alfa Resources
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