cerliponase alfa

Contraindicated in:

Acute infection around device insertion site or CNS infection;
Acute intraventricular access device-related complication;
Ventriculoperitoneal shunts.

Use Cautiously in:

Bradycardia, conduction disorder, or structural heart disease (↑ risk of conduction disorder or heart disease).

Adv. Reactions/Side Effects ⬆ ⬇

CV: ECG abnormalities, bradycardia, hypotension

GI: vomiting

Hemat: hematoma

Neuro: headache, irritability, pleocytosis, seizures, jitteriness, MENINGITIS

Misc: fever, hypersensitivity reactions (including anaphylaxis), infusion-associated reactions (including anaphylaxis), device-related complication (including device leakage, bulging of scalp around device, device failure), device-related infection

Interactions ⬆ ⬇

Drug-drug:

None reported.

Availability ⬆ ⬇

Solution for intraventricular infusion: 30 mg/mL (with 5-mL vial of intraventricular electrolyte solution for intraventricular infusion)

Route/Dosage ⬆ ⬇

Intraventricular(Children ≥2 yr): 300 mg every other wk. Follow each infusion with infusion of intraventricular electrolytes.
Intraventricular(Children 1–<2 yr): 200 mg every other wk for 1st four doses, then 300 mg every other wk. Follow each infusion with infusion of intraventricular electrolytes.
Intraventricular(Children 6 mo–<1 yr): 150 mg every other wk. Follow each infusion with infusion of intraventricular electrolytes.
Intraventricular(Children Birth–<6 mo): 100 mg every other wk. Follow each infusion with infusion of intraventricular electrolytes.

US Brand Names ⬆ ⬇

Action ⬆ ⬇

Proenzyme is activated in the lysosomes and then cleaves tripeptides from a broad range of proteins. This leads to the breakdown of lysosomal storage materials that otherwise accumulate in patients with late infantile neuronal CLN2, leading to progressive decline in motor function.

Therapeutic effects:

Slowed decline in ambulation.

Classifications ⬆ ⬇

Therapeutic Classification: none assigned

Pharmacologic Classification: enzymes

Pharmacokinetics ⬆ ⬇

Absorption: Unknown.

Distribution: Distributed into CSF following intraventricular administration.

Metabolism/Excretion: Degraded through peptide hydrolysis.

Half-Life: 6.2–7.7 hr.

Time/Action Profile ⬆ ⬇

(CSF concentrations)

ROUTE	ONSET	PEAK	DURATION
Intraventricular	unknown	4.3–4.5 hr	unknown

Patient/Family Teaching ⬆ ⬇

Explain purpose and side effects to patient and caregivers. Advise patient and caregivers to read Patient Information before starting therapy. If one or more doses are missed, restart treatment as soon as possible; maintain the 2 wk interval between infusions.
Advise patient and caregivers to notify health care professional of all Rx or OTC medications, vitamins, or herbal products being taken and to consult health care professional before taking other medications.
Instruct patient and caregivers to notify health care professional immediately if signs and symptoms of infection, hypotension, bradycardia, infusion-associated reactions, and hypersensitivity reactions occur.
Advise patients and caregivers of the risk of device-related infections. If any signs of infection are present, instruct to immediately contact a health care provider.

Pronunciation ⬆ ⬇

ser-LIP-oh-nase AL-fa

Code ⬆

NDC Code

68135- BioMarin Pharmaceutical Inc.
- 68135-502-
  - 68135-502-00- 2000 mL in 1 BOTTLE (68135-502-00)

68135- BioMarin Pharmaceutical Inc.
- 68135-811- Brineura
  - 68135-811-02- 1 KIT in 1 CARTON (68135-811-02) * 5 mL in 1 VIAL, GLASS (68135-500-00) * 5 mL in 1 VIAL, GLASS (68135-495-04)

71931- BioMarin International Limited
- 71931-820-
  - 71931-820-99- 2000 mL in 1 BOTTLE (71931-820-99)

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Indications ⬇

Contraind./Precautions ⬆ ⬇