Recombinant

L01X - Other antineoplastic agents ATC L01XX02

JFDA label: Spectrila 10,000 U powder for concentrate for solution for injection

Indications

Approved

Neuronal ceroid lipofuscinosis type 2

Contraindications

Source: Lexicomp

Acute intraventricular access device-related complications (eg, leakage, device failure, device-related infection) Absolute
ventriculoperitoneal shunts Absolute

Adverse Reactions

Very Common >10%Common 1–10%Uncommon 0.1–1% Rare 0.01–0.1%Very Rare <0.01%Not Known

Cardiac disorders (3)

Very Common ECG abnormality

Common Bradycardia · hypotension

Nervous system disorders (6)

Very Common abnormal cerebrospinal fluid · Abnormal proteins in cerebrospinal fluid · headache · irritability · seizure

Common Jitteriness

Blood and lymphatic system disorders (1)

Very Common Hematoma

Immune system disorders (2)

Very Common Antibody development · Hypersensitivity reaction

Gastrointestinal disorders (1)

Very Common Vomiting

General disorders and administration site conditions (2)

Very Common Fever

Common Catheter infection

Dosing

Source: Lexicomp

(For additional information see "Recombinant human cerliponase alfa: Pediatric drug information") Note: Administer the first dose 5 to 7 days after device implantation. Pretreat with antihistamines with or without antipyretics or corticosteroids 30 to 60 minutes prior to start of infusion. Neuronal ceroid lipofuscinosis type 2: Children ≥3 years and Adolescents: Intraventricular: 300 mg (10 mL) once every other week; following cerliponase alfa infusion, administer 2 mL intraventricular electrolytes (included in administration kit)

There are no dosage adjustments provided in the manufacturer's labeling.

Warnings & Precautions

Source: Lexicomp

Cardiovascular adverse reactions

Hypotension has been reported up to 8 hours after the completion of cerliponase alfa infusion; monitor vital signs. Use with caution in patients with a history of bradycardia, conduction disorder, or structural heart disease; monitor EKG during infusion.

Device-related complications

Complications can occur with intraventricular access devices. Device-related infections have been reported with cerliponase alfa and may be subclinical. Administer using aseptic technique to minimize infection. Monitor access point skin integrity and send CSF samples routinely for signs of infection. Management of infection may include antibiotic treatment and device replacement before resumption of therapy. Monitor the device for signs of leakage or device failure. Material degradation of the intraventricular access device reservoir may occur with prolonged use (≥105 perforations), requiring replacement of the device.

Hypersensitivity reactions

Hypersensitivity reactions, including pyrexia, vomiting, pleocytosis and irritability, have been reported in patients, during and up to 24 hours after completion of cerliponase alfa infusion. Pretreat with antihistamines with or without antipyretics or corticosteroids 30 to 60 minutes prior to start of infusion. Other warnings/precautions:

Appropriate use

Administer cerliponase alfa by FDA-approved infusion pump system via intraventricular implanted catheter access (consult prescribing information for device details); health care providers should be experienced with intraventricular drug administration.

Pregnancy & Lactation

Pregnancy

Animal reproduction studies have not been conducted with cerliponase alfa.

Lactation

It is not known if cerliponase alfa is present in breast milk. According to the manufacturer, the decision to breastfeed during therapy should consider the risk of infant exposure, the benefits of breastfeeding to the infant, and benefits of treatment to the mother.

Monitoring

Clinical pearl	Vital signs (blood pressure, heart rate) prior to start of infusion, periodically during infusion and post-infusion; skin integrity (prior to infusion); routine CSF samples (to detect subclinical device infections); 12-lead EKG every 6 months or during infusion with cardiac abnormalities.

Biology & Pharmacokinetics

Pharmacokinetics

Half-life	7.7 h

Registered Products (1)

Brand	Form / strength	Pack	Agent	Citizen (JOD)
Spectrila 10,000 U powder for concentrate for solution for injection	Powder for Injection 10, 000 Units** UNT	1 vial	Pharma Care Drug Store	—