FDAnews Drug Daily Bulletin
Pharmaceuticals / Submissions and Approvals

European Commission Approves Brineura Treatment for CLN2 Disease

June 7, 2017
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BioMarin Pharmaceutical has received marketing clearance from the European Commission for Brineura for the treatment of neuronal ceroid lipofuscinosis type 2, also known as tripeptidyl peptidase 1 deficiency.

The enzyme replacement therapy is directly administered to the cerebral spinal fluid. The FDA approved Brineura to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile CLN2.

In a clinical study, 87 percent of patients did not decline in motor and language scores at 48 weeks.

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