BioMarin planning to submit Batten therapy

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) plans to submit U.S. and EU regulatory applications mid-year for cerliponase alfa ( BMN 190) to treat late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease. BioMarin announced data from a pivotal Phase I/II trial of the candidate at the WORLDSymposium meeting in San Diego.

Prior to obtaining market approval, the company intends to launch an early access program in 3Q16 to provide patients with cerliponase alfa, but said it is still determining the program's "overall scope, eligibility criteria and details." ...