- The European Medicines Agency validates BioMarin Pharmaceutical's (NASDAQ:BMRN -1%) Marketing Authorization Application (MAA) seeking approval of enzyme replacement therapy Brineura (cerliponase alfa) for the treatment of CLN2 disease, a form of Batten disease, a rare inherited disorder in which the body's cells lose their ability to dispose of wastes. Validation means that the application is complete and the review process begins.
- The company requested accelerated review for the Orphan Drug-designated use, but the shortened time line is not guaranteed. Assuming a positive opinion from CHMP and the standard 210-day review period, a decision should be announced no later than Q3 of next year.
- The marketing application in the U.S. is currently under review with an action date of April 27, 2017.