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cerliponase alfa (Brineura)
Indications:
- symptomatic pediatric patients >= 3 years of age with
- late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease
* improves motor & language function [2]
Dosage:
- 300 mg administered once every other week as an intraventricular infusion (2.5 mL/hr) followed by infusion of intraventricular electrolytes
- duration of total infusion over ~4.5 hours
single-dose vials 5 mL, 2/carton: 30 mg/mL
co-packaged with intraventricular electrolytes solution 5 mL in a single dose vial
Adverse effects:
- >= 8%
- pyrexia
- vomiting
- seizures
- hypersensitivity
- hematoma
- headache
- irritability
- pleocytosis
- failure of intraventricular device [2]
- device-related infection
- bradycardia
- jittes
- hypotension
- ECG abnormalities
- decreased or increased CSF protein
Mechanism of action:
- recombinant tripeptidyl peptidase 1
General
neurologic agent
References
- Windle ML.
FDA New Drug and Biologic Approvals -- 2017 Year-in-Review
Medscape - Jan 11, 2018.
https://reference.medscape.com/viewarticle/890871
- U.S. Food and Drug Administration.
Novel Drug Approvals for 2017.
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm
- Schulz A, Ajayi T, Specchio N et al
Study of Intraventricular Cerliponase Alfa for CLN2 Disease.
N Engl J Med 2018; 378:1898-1907. May 17, 2018
PMID: 29688815 Free full text
https://www.nejm.org/doi/full/10.1056/NEJMoa1712649
- Brineura prescribing information
http://brineura.com/downloads/Brineura_PI.pdf
- Brineura (cerliponase alfa) prescribing information.
BioMarin Pharmaceuticals, Inc. Novato, CA. April 2017.
https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/761052lbl.pdf