>10%

Pyrexia (71%)

ECG abnormalities (71%)

Decreased CSF protein (71%)

Vomiting (63%)

Seizures (50%)

Hypersensitivity (46%)

Increased CSF protein (21%)

Hematoma (21%)

Headache (17%)

Irritability (17%)

Pleocytosis (17%)

1-10%

Device-related infection (8%)

Bradycardia (8%)

Feeling jittery (8%)

Hypotension (8%)

Brineura (cerliponase alfa) (150 mg/5 mL) injectable kit | Brineura cost  Postmarketing Reports

Bacterial meningitis

Immune system disorders: Anaphylactic reaction characterized by acute pyrexia, respiratory distress (bronchospasm, hypoxemia, perioral cyanosis), tachycardia, hypotension, diarrhea, and rash.

Description
Brineura (cerliponase alfa) (150 mg/5 mL) injectable kit | Brineura cost 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.

Policy 
Brineura (cerliponase alfa) is considered MEDICALLY NECESSARY in the treatment of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2)for ALL of the following criteria are met:

1. Diagnosis of CLN2 was confirmed by enzyme assay demonstrating a deficiency of tripeptidyl peptidase 1 (TPP1) enzyme activity or by genetic testing.
2. Member is 3 years of age or older.
3. Brineura (cerliponase alfa) (150 mg/5 mL) injectable kit | Brineura cost will be administered by, or under the direction of, a physician knowledgeable in intraventricular administration.
4. Patient does not have acute intraventricular access-related complications (e.g., leakage, device failure or device-related infections)
5. Patient does not have ventriculoperitoneal shunts
6. Prescribed by or in consultation with a neurologist with expertise in the diagnosis of CLN2/TTP1 deficiency.

Continuation

1. Patient does not have acute intraventricular access-related complications (e.g., leakage, device failure or device-related infections) AND
2. Patient does not have ventriculoperitoneal shunts AND
3. Patient has experienced a benefit from therapy (e.g., improvement in walking or crawling, or no evidence of disease progression).

Dosage

• Aseptic technique must be strictly observed during preparation and administration. Brineura should be administered by, or under the direction of, a physician knowledgeable in intraventricular administration. Brineura is administered to the cerebrospinal fluid (CSF) by infusion via a surgically implanted reservoir and catheter.
• Pre-treatment of patients with antihistamines with or without antipyretics or corticosteroids is recommended 30 to 60 minutes prior to the start of infusion.
• 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.
• For complete information on preparation, specific intraventricular access device for use, and administration, see the full prescribing information.

Coding Section 

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)

Patients with ventriculoperitoneal shunts

Cautions

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 (see Administration)

Cardiovascular adverse effects

• Monitor vital signs before infusion starts, periodically during infusion, and postinfusion in a healthcare setting
• Perform 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

Hypersensitivity reactions

• Life-threatening hypersensitivity reactions, including anaphylaxis, reported during infusion or within several hours of completion
• Observe patient during and after infusion; inform caregivers of signs and symptoms of anaphylaxis and instruct them to seek immediate medical care if these occur
• Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy; administration of therapy should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis
• Initiate therapy in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment; observe patients closely during and after the infusion
• If severe hypersensitivity reaction (eg, anaphylaxis) occurs, discontinue therapy and immediately initiate appropriate medical treatment including use of epinephrine
• Management of hypersensitivity reactions should be based on severity of reaction and may include temporarily interrupting infusion, and/or treatment with antihistamines, antipyretics, and/or corticosteroids
• Consider risks and benefits of readministration of therapy following an anaphylactic reaction; if the decision is made to readminister therapy after occurrence of anaphylaxis, ensure appropriately trained personnel and equipment for emergency resuscitation (including epinephrine and other emergency medicines) are readily available during infusion; initiate subsequent infusion at approximately one-half initial infusion rate at which anaphylactic reaction occurred

Intraventricular access complications

• Bacterial meningitis requiring antibiotic treatment and removal of 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 direction of, physician experienced in intraventricular administration to reduce risk of infectious complications
• Obtain CSF sample 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 device insertion site, such as erythema, tenderness, or discharge or suspected or confirmed CNS infection (eg, cloudy CSF or positive CSF gram stain, or meningitis)
• Clinicians should be vigilant for 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 implanted device; in case of device-related complication, discontinue infusion and refer to device labeling for further instructions

Infusion-associated reactions (IARs)

• Infusion-associated reactions (eg, vomiting, seizure, rash, pyrexia, hypersensitivity, anaphylactic reaction) have been observed
• Premedicate with antihistamines (with or without antipyretics or corticosteroids) recommended 30-60 minutes before intraventricular infusion
• If reaction occurs, decrease rate, temporarily stop infusion, and/or administer antihistamines and/or antipyretics to ameliorate symptoms
• Closely monitor patients with history or IARs when readministering