Brineura regulatory update

FDA approved a BLA from BioMarin for Brineura cerliponase alfa to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, in children ages ≥3. The therapy is the first approved for the indication. Brineura is a recombinant human tripeptidyl peptidase-1 (TPP1) enzyme replacement therapy.

Brineura's list price is $27,000 per biweekly infusion, or $702,000 per year. The company said the drug's net price will be $486,000 after deducting mandatory government discounts and rebates, and factoring in patient compliance. Brineura will be available in June...