BioMarin announced that the Food and Drug Administration (FDA) has accepted and granted Priority Review to the Biologics License Application (BLA) of cerliponase alfa for the treatment of children with CLN2 disease, a form of Batten disease. 

CLN2 disease is a rare, rapidly progressing and fatal neurodegenerative disease caused by deficiency in tripeptidyl peptidase 1 (TPP1) enzyme activity. Initial symptoms typically present between the ages of 2 and 4, which include language delays and seizures, followed by movement disorders, motor deterioration, dementia and blindness. Death usually occurs between 8 and 12 years of age. Currently, there are no FDA-approved treatments; available options are limited to symptomatic care to preserve quality of life.

The FDA has set a Prescription Drug User Fee Act (PDUFA) target date of January 27, 2017 to make a decision on the BLA. In the meantime, BioMarin intends to implement an early access program to provide cerliponase alfa to a limited number of patients with CLN2 disease prior to approval. Overall scope and details of the program are under development, but the company expects to initiate during Q3 2016.

Cerliponase alfa is a recombinant form of human TPP1 enzyme, delivered through intracerebroventricular (ICV) infusion, to restore TPP1 activity and break down storage materials that cause CLN2 disease. The proposed brand name is Brineura.

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