BioMarin Reports EMA Validation Of Brineura MAA For Treatment Of CLN2 Disease

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) announced that the European Medicines Agency or EMA validated the Marketing Authorization Application or MAA for Brineura (cerliponase alfa) to treat children with CLN2 disease, a form of Batten disease. Validation of the MAA confirms that the submission is accepted and starts the formal review process by the EMA's Committee for Human Medicinal Products or CHMP.

Assuming a positive opinion from the CHMP and standard assessment timing, a decision from the European Commission is anticipated by the third quarter of 2017. The EMA previously granted Brineura Orphan Drug Designation.

On July 27, 2016, the U.S. Food and Drug Administration (FDA) accepted for review the submission of a Biologics License Application (BLA) for cerliponase alfa. The Prescription Drug User Fee Act (PDUFA) goal date for a decision is April 27, 2017. 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.