Cerliponase Alfa

A recombinant human tripeptidyl peptidase 1 enzyme administered directly into the brain via intracerebroventricular infusion to treat neuronal ceroid lipofuscinosis type 2 (CLN2 disease), a rare and fatal inherited neurological disorder in children. It replaces the enzyme that is deficient in affected patients, slowing the neurological deterioration characteristic of the disease. It is the first enzyme replacement therapy approved for direct central nervous system administration.

Provides exogenous replacement of the deficient enzyme, restoring the metabolic pathway and clearing accumulated substrates from affected tissues.

Yes, Cerliponase Alfa is an approved drug. It has reached clinical phase 4. It is classified as a Enzyme.