Cerliponase Alfa｜CLN2 Disease｜HongKong DengYue Medicine

Cerliponase Alfa Application Scope

Cerliponase alfa is an enzyme replacement therapy used for:

• Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease) – A rare, fatal neurodegenerative lysosomal storage disorder caused by tripeptidyl peptidase-1 (TPP1) deficiency.
• Symptomatic children ≥3 years old – Slows the decline of motor function, particularly walking ability.

Characteristics

Ingredients

• Active: Recombinant human TPP1 enzyme (cerliponase alfa).
• Excipients: Stabilizers in the intraventricular infusion solution (exact formulation not publicly detailed).

Properties

• Mechanism: Binds to mannose-6-phosphate receptors (CI-MPR) in the CNS, delivering TPP1 to lysosomes to degrade lipofuscin deposits.
• Molecular Weight: ~65 kDa (glycosylated form).

Specification

• 30 mg/mL (intraventricular infusion solution).

Packaging Specification

• Single-dose vial (150 mg/5 mL).

Storage

• Unopened vials: 2–8°C (refrigerated) – Do not freeze.
• After reconstitution: Stable for ≤24 hours if refrigerated.

Expiry Date

• Typically 24 months when stored properly (check batch-specific labeling).

Executive Standard

• Manufactured under cGMP (FDA/EMA-approved).

Approval Number

• FDA: Approved April 2017 (first treatment for CLN2).
• EMA: Approved 2017 (EU authorization).

Date of Revision

• Varies by region (latest US label update: 2023).

Manufacturer

• BioMarin Pharmaceutical Inc. (brand name: Brineura®).

Guidelines for Use Of Cerliponase Alfa

Dosage and Administration

• Recommended dose: 300 mg every other week via intraventricular infusion (via implanted reservoir).
• Infusion protocol:
  • Premedicate with antihistamines/antipyretics ± corticosteroids (30–60 min before).
  • Administer over ~4.5 hours, followed by intraventricular electrolyte infusion.
  • Hospital administration required (due to infection/seizure risks).

Adverse Reactions

• Common (≥10%): Fever, vomiting, seizures, CSF protein increase, hypersensitivity, headache.
• Serious:
  • Device-related infections.
  • Cardiovascular events (bradycardia, hypotension).

Contraindications

• Active CNS/device-site infections.
• Acute intraventricular device complications.

Precautions

• Infusion monitoring: ECG for cardiac abnormalities; CSF sampling pre-infusion.
• Hypersensitivity: Immediate discontinuation if anaphylaxis occurs.
• Seizure risk: Monitor neurological status closely.

Interactions

Drug Interactions

• No major interactions reported, but concomitant CNS-depressants may exacerbate seizures.

 

Note:

• If there is a new packaging for the drug, the new packaging shall prevail. The above information is sourced from DengYue Medicine.
• It is only for internal discussion among medical staff and does not serve as a basis for medication. For specific medication guidelines, please consult the attending physician.