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2 Information about cerliponase alfa

Marketing authorisation indication

2.1

Cerliponase alfa (Brineura, BioMarin) is indicated for 'the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency'.

Dosage in the marketing authorisation

Price

2.3

The list price of cerliponase alfa is £20,107 per 300-mg pack consisting of two 150-mg vials (excluding VAT, company submission). The recommended dosage for people over 2 is 300 mg every other week (annual cost of £522,782 per person).

2.4

The company has a commercial arrangement, which would have applied if cerliponase alfa had been recommended. The size of the discount is commercial in confidence.