BioMarin's Vimizim gets FDA approval to treat enzyme disorder The U.S. Food and Drug Administration approved Vimizim, the first FDA-approved treatment for Mucopolysaccharidosis Type IVA. Vimizim is intended to replace the missing GALNS enzyme involved in an important metabolic pathway. Vimizim was granted priority review. An FDA priority review provides for an expedited review of drugs for serious diseases or conditions that may offer major advances in treatment. Vimizim is also the first drug to receive the Rare Pediatric Disease Priority Review Voucher, a provision that aims to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Vimizim is marketed by BioMarin Pharmaceutical. Reference Link
BioMarin upgraded to Buy from Neutral at Goldman Goldman upgraded BioMarin to Buy based on increased confidence in BMN-111 for achondroplasia with expected proof-of-concept data in Q2 2015. The firm now sees a greater than 50% probability of success and peak sales of $900M. Price target raised to $104 from $78.