Posted by Michael Wonder on 02 Aug 2016
FDA accepts BLA for BioMarin's cerliponase alfa for CLN2 disease, form of Batten disease
27 July 2016 - Potential first treatment for fatal, rare, brain disease in children.
BioMarin announced today that the U.S. FDA accepted for review the submission of a biologics license application for cerliponase alfa, an investigational therapy to treat children with CLN2 disease, a form of Batten disease. The Prescription Drug User Fee Act (PDUFA) goal date for a decision is January 27, 2017. BioMarin also has submitted a marketing authorisation application to the EMA for cerliponase alfa, and it is undergoing validation at the Agency.
The FDA granted cerliponase alfa priority review status, which is designated to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. Cerliponase alfa was previously granted orphan drug designation and breakthrough therapy designation by the FDA.
The company also announced that the preliminary approved brand name for cerliponase alfa is Brineura.
