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.
3.2 Cerliponase alfa is administered into the cerebrospinal fluid by infusion via a surgically implanted intracerebroventricular access device (reservoir and catheter). It must only be given in a healthcare setting by a trained healthcare professional knowledgeable in intracerebroventricular infusion administration. The recommended dose is 300 mg cerliponase alfa once every other week, but lower doses are recommended in patients under 2 years.
3.3 The adverse reactions listed as very common (that is, occurring in 1 in 10 people or more) in the summary of product characteristics for cerliponase alfa include: hypersensitivity, upper respiratory tract infection, seizures, headache, irritability, cerebrospinal fluid pleocytosis, vomiting and pyrexia. For full details of adverse reactions and contraindications, see the summary of product characteristics.