3.1
Cerliponase alfa (Brineura, BioMarin) is an enzyme replacement therapy consisting of a recombinant form of human tripeptidyl peptidase 1. It is expected to restore deficient tripeptidyl peptidase 1 (TPP1) activity in the brain caused by the genetic mutation. Cerliponase alfa has a UK marketing authorisation granted under 'exceptional circumstances' for 'the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease', also known as TPP1 deficiency.