FDAnews Drug Daily Bulletin
Pharmaceuticals / Submissions and Approvals

FDA Approves BioMarin's Brineura

May 3, 2017
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California–based BioMarin Pharmaceutical has gained FDA approval of Brineura (cerliponase alfa) for late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also called tripeptidyl peptidase 1 (TPP1) deficiency.

Brineura is an enzyme replacement therapy that slows the loss of ambulation in pediatric patients three years of age and older with CLN2, a type of Batten disease.

In addition to the approval, BioMarin received a rare pediatric disease priority review voucher.

The European Medicines Agency’s committee for medicinal products for human use issued a positive opinion on Brineura and a decision by the European Commission is expected by the second quarter of this year.

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