FDAnews Drug Daily Bulletin

European Commission Approves Brineura Treatment for CLN2 Disease

June 7, 2017

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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