Cerliponase Alfa
Cerliponase Alfa Uses, Dosage, Side Effects, Food Interaction and all others data.
Cerliponase Alfa is an enzyme replacement treatment for a specific form of Batten disease. It was the first FDA-approved treatment to slow loss of walking ability (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. Intraventricular administration of the drug allows significant uptake into the brain. Cerliponase Alfa was approved in April, 2017 (as Brineura).
Cerliponase Alfa contains the active substance tripeptidyl peptidase-1 (rhTPP1), a recombinant human lysosomal exopeptidase which cleaves the N-terminal of tripeptides with a broad substrate specificity. Cerliponase Alfa slows the progressive decline in motor function caused by abnormal motor signalling in the brain by restoring the normal levels and activity of TPP1.
| Trade Name | Cerliponase Alfa |
| Generic | Cerliponase alfa |
| Cerliponase alfa Other Names | Cerliponase alfa, Immature cell growth-inhibiting gene 1 protein, Immature human tripeptidyl-peptidase 1, Immature lysosomal pepstatin-insensitive protease, Immature tripeptidyl-peptidase I |
| Weight | 150mg/5ml, |
| Type | Injectable Kit, Intracerebroventricular |
| Weight | 59.0 Da (glycosylated) |
| Groups | Approved, Investigational |
| Therapeutic Class | |
| Manufacturer | |
| Available Country | United States |
| Last Updated: | September 19, 2023 at 7:00 am |
Uses
Cerliponase Alfa is an enzyme replacement therapy used to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Cerliponase Alfa is a treatment for late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease to decelerate the progressive motor function decline in patients 3 years of age and older. CLN2 disease is a form of Batten disease, a rare inherited neurodegenerative disorder and is associated with seizures, ataxia, rapid loss of language and motor functions, blindness, and early death . It is caused by the lack the lysosomal enzyme tripeptidyl peptidase-1 (TPP1) and subsequent accumulation of lysosomal storage materials normally metabolized by this enzyme in the central nervous system.
Cerliponase Alfa is also used to associated treatment for these conditions: Neuronal ceroid lipofuscinosis type 2
How Cerliponase Alfa works
The mature form of enzyme contains 5 consensus N-glycosylation sites with high mannose, phosphorylated high mannose and complex glycosylation structures. It is taken up by LINCL fibroblasts and translocated to the lysosomes through the Cation Independent Mannose-6-Phosphate Receptor (CI-MPR, also known as M6P/IGF2 receptor). Cerliponase Alfa is activated in the lysosome under low pH conditions and the activated proteolytic form of rhTPP1 cleaves tripeptides from the N-terminus of stored proteins.
Toxicity
No data from carcinogenicity, genotoxicity, and fertility studies. Unwanted effects of cerliponase alfa treatment include pyrexia, ECG abnormalities, decreased CSF protein, seizure and hypersensitivity.
Food Interaction
No interactions found.Volume of Distribution
The estimated CSF volume of distribution of cerliponase alfa following intraventricular infusion of 300mg of Brineura (median Vss = 245 mL) exceeds the typical CSF volume (100 mL) .
Elimination Route
Refer to FDA Label
Half Life
Refer to FDA Label
Clearance
Refer to FDA Label
Innovators Monograph
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