.Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AB17_CerliponaseAlfa.CerliponaseAlfa .Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AB17_CerliponaseAlfa.CerliponaseAlfa| name: | CerliponaseAlfa | |
| ATC code: | A16AB17 | |
| route: | intraventricular | |
| compartments: | 1 | |
| dosage: | 300 | mg |
| volume of distribution: | 210 | L |
| clearance: | 4.6 | ml/min |
| other parameters in model implementation | ||
Cerliponase alfa is a recombinant human tripeptidyl peptidase 1 (TPP1) used as an enzyme replacement therapy for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2 disease, also known as Batten disease). It is approved for intraventricular administration in pediatric patients with CLN2 disease to slow the loss of ambulation.
Pharmacokinetic parameters reported in pediatric CLN2 patients (mean age 5 years) receiving recommended dosage.