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'.
2 Information about cerliponase alfa
Marketing authorisation indication
Dosage in the marketing authorisation
2.2
The dosage schedule is available in the summary of product characteristics for cerliponase alfa.
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 years is 300 mg every other week, which has an 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. The company had an additional commercial agreement as part of the managed access agreement which did not apply during the appraisal because it was a time-limited agreement.
Sustainability
2.5
For information, see BioMarin's webpage on environmental stewardship.